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Clinical Trial Results:
A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele

Summary
EudraCT number	2018-000966-12
Trial protocol	DE
Global end of trial date	05 Apr 2022

Results information
Results version number	v1(current)
This version publication date	27 May 2023
First version publication date	27 May 2023
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

EL-004

ISRCTN number

US NCT number

NCT04126473

WHO universal trial number (UTN)

Sponsor organisation name

Eloxx Pharmaceuticals, Inc.

Sponsor organisation address

480 Arsenal Way, Suite 130, Watertown, MA, United States, 02472

Public contact

Tim Kachmar, Eloxx Pharmaceuticals, Inc., +1 6176459926, tim.kachmar@eloxxpharma.com

Scientific contact

Tim Kachmar, Eloxx Pharmaceuticals, Inc., +1 6176459926, tim.kachmar@eloxxpharma.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

05 Apr 2022

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

05 Apr 2022

Was the trial ended prematurely?

Main objective of the trial

To assess the safety and tolerability of different dose levels and exposures of ELX-02 administered subcutaneously (SC), as monotherapy and in combination with ivacaftor in subjects with Cystic Fibrosis (CF) carrying at least one G542X or phenotypically similar Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) nonsense allele and to study the pharmacokinetics (PK) of multiple SC doses of ELX-02 as monotherapy and in combination with ivacaftor, in subjects with CF.

Protection of trial subjects

The study was conducted in compliance with the study protocol, with the International Standard of Good Clinical Practice (GCP) procedures, and with the principles of the Declaration of Helsinki (1964) and relevant amendments.

Background therapy

Evidence for comparator

Actual start date of recruitment

05 Nov 2019

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Israel: 12

Country: Number of subjects enrolled

Germany: 5

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

The study was conducted at 6 centers: 4 in Israel and 2 in Germany from 05 November 2019 to 05 April 2022.

Screening details

A total of 17 subjects were enrolled in this study. The study consisted of 3 periods for each subject; a screening period of up to 6 weeks, a treatment period (TP) of up to 10 weeks and a safety follow up period of 4 weeks after the last treatment.

Period 1 title

Overall trial (overall period)

Is this the baseline period?

Yes

Allocation method

Not applicable

Blinding used

Not blinded

ELX-02: All Subjects (At least one TP from TP 1 to TP 5)

Arm description

Subjects received ELX-02 0.3, 0.75 and 1.5 milligram per kilograms (mg/kg) subcutaneously (SC) daily for one week each in Treatment Periods 1,2 and 3 as monotherapy and ELX-02 up to 3.0 mg/kg SC daily for 2 weeks in Treatment Period 4 (individualized dose 2.8 mg/kg to 3.0 mg/kg to target a total exposure of approximately 190 microgram-hours per milliliter (mcg*h/mL). Subjects received 5 ELX-02 1.5 mg/kg for 1 week, followed by ELX-02 at 1.5 mg/kg SC daily, together with one ivacaftor 150 mg tablet orally every 12 hours for 4 weeks in Treatment Period 5.

Arm type

Experimental

Investigational medicinal product name

ELX-02

Investigational medicinal product code

Other name

Pharmaceutical forms

Concentrate for solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

ELX-02 0.3, 0.75 or 1.5 mg/kg SC daily for one week in Treatment Periods 1, 2 and 3, respectively. ELX-02 was dosed daily for 2 weeks in Treatment Period 4 with an individualized dose 2.8 mg/kg to 3.0 mg/kg to target a total exposure of approximately 190 mcg*h/mL. In Treatment Period 5, ELX-02 was dosed at 1.5 mg/kg daily for one week followed by ELX-02 at 1.5 mg/kg daily in combination with one ivacaftor 150 mg tablet every 12 hours for 4 weeks.

Investigational medicinal product name

Ivacaftor

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Ivacaftor administered orally in Treatment Period 5 as 150 mg tablet every 12 hours together with ELX-02 at 1.5 mg/kg SC daily for 4 weeks after administration of ELX-02 as monotherapy at a dose of 1.5 mg/kg (as in Treatment Period 3) for 1 week.

Number of subjects in period 1	ELX-02: All Subjects (At least one TP from TP 1 to TP 5)
Started	17
Completed	11
Not completed	6
Consent withdrawn by subject	3
Adverse event, non-fatal	2
Lost to follow-up	1

Baseline characteristics

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Reporting group title

Overall trial

Reporting group description

Reporting group values	Overall trial	Total
Number of subjects	17	17
Age categorical
Units: Subjects
Age continuous
Units: years
arithmetic mean (standard deviation)	29.4 ± 8.95	-
Gender categorical
Units: Subjects
Female	5	5
Male	12	12
Race
Units: Subjects
Black or African American	0	0
White	17	17
Ethnicity
Units: Subjects
Hispanic or Latino	0	0
Not Hispanic or Latino	17	17

End points

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Reporting group title

ELX-02: All Subjects (At least one TP from TP 1 to TP 5)

Reporting group description

Subject analysis set title

Treatment Period 1: ELX-02 0.3 mg/kg

Subject analysis set type

Safety analysis

Subject analysis set description

Subjects received ELX-02 0.3 mg/kg SC daily for 1 week (about 0.4 mL/day, total dose not to exceed 2.1 mg/kg for this 7-day period) in Treatment Period 1. Safety population consisted of all treated subjects who received at least one dose of study medication, including subjects prematurely withdrawn from the study.

Subject analysis set title

Treatment Period 2: ELX-02 0.75 mg/kg

Subject analysis set type

Safety analysis

Subject analysis set description

Subjects received ELX-02 0.75 mg/kg SC daily for 1 week (about 0.9 mL/day, total dose not to exceed 5.25 mg/kg for this 7-day period) in Treatment Period 2.

Subject analysis set title

Treatment Period 3: ELX-02 1.5 mg/kg

Subject analysis set type

Safety analysis

Subject analysis set description

Subjects received ELX-02 1.5 mg/kg SC daily for 1 week (two daily injections of about 0.9 mL, total dose not to exceed 10.5 mg/kg for this 7-day period) in Treatment Period 3.

Subject analysis set title

Treatment Period 4: An individualized dose up to 3 mg/kg

Subject analysis set type

Safety analysis

Subject analysis set description

Subjects received ELX-02 individualized doses 2.8 mg/kg to 3.0 mg/kg SC daily for 2 weeks (up to 3 or 4 injections of about 1.2 mL) to target a total exposure of approximately 190 mcg*h/mL in Treatment Period 4.

Subject analysis set title

Treatment Period 5: ELX-02 + Ivacaftor

Subject analysis set type

Safety analysis

Subject analysis set description

Subjects received ELX-02 1.5 mg/kg (as in Treatment Period 3) for 1 week, followed by 4 weeks of therapy with ELX-02 1.5 mg/kg SC daily, together with Ivacaftor 150 mg tablet taken orally every 12 hours in Treatment Period 5.

Primary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs), Severe TEAEs, Serious TEAEs, Related TEAEs, Related Serious TEAE, TEAE Leading to Study Drug Discontinuation and Death

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End point title

Number of Subjects With Treatment-emergent Adverse Events (TEAEs), Severe TEAEs, Serious TEAEs, Related TEAEs, Related Serious TEAE, TEAE Leading to Study Drug Discontinuation and Death ^[1]

End point description

TEAE was defined as any AE with onset after the first administration of study medication through the end of the study, or any event that was present at baseline but worsened in intensity or was subsequently considered drug-related by the Investigator through the end of the study. Severe TEAE is a TEAE with CTCAE Grade 3 or above. Related TEAE is defined as a TEAE with a certain, probable/likely, or possible relationship to the study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening. The Safety population comprised of all subjects who received 1 or more doses of study medication. Serious TEAE subject identified 3 months after completing treatment period 3 during dosing hiatus.

End point type

Primary

End point timeframe

From baseline up to 29 months

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics were performed; no inferential statistical analyses were performed.

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	13	10	8	4	6
Units: subjects
All TEAE	7	6	4	4	5
Severe TEAE	0	0	0	0	1
Serious TEAE	0	0	1	0	0
Related TEAE	6	5	4	2	1
Related Serious TEAE	0	0	0	0	0
TEAE Leading to Study Drug Discontinuation	1	1	0	0	1
TEAE Leading to Death	0	0	0	0	0

No statistical analyses for this end point

Primary: AUC(0-24h): Area Under the Plasma Concentration-time Curve from Time 0 to 24 Hours Postdose for ELX-02

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End point title

AUC(0-24h): Area Under the Plasma Concentration-time Curve from Time 0 to 24 Hours Postdose for ELX-02 ^[2]

End point description

AUC(0-24h) was defined as the area under the plasma concentration-time curve from zero to the time of the last measured concentration that is 24 hours above the limit of quantification post dose. The PK population included all safety subjects who had analyzable PK data without relevant deviation interfering with the PK evaluations. Here, overall number of subjects analyzed signifies subjects who were evaluable for this endpoint. '99999' indicates that data could not be estimated for specified Treatment Period where data is not available due to either no value(s) within the detectable range or no sample(s) were collected.

End point type

Primary

End point timeframe

Treatment Period 1-4 (Day 1) and Treatment Period 5 (Day 8): Pre-dose, and at 15 minutes, 30 minutes, 1,3,4,6,8, and 24 hours post-dose

Notes
[2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics were performed; no inferential statistical analyses were performed.

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	13	10	2	2	4
Units: nanogramhour per milliliter (ngh/mL)
arithmetic mean (standard deviation)	99999 ± 99999	99999 ± 99999	26729.645 ± 8280.6357	31301.081 ± 5781.1191	99999 ± 99999

No statistical analyses for this end point

Primary: Ae24h: Amount Excreted in Urine from Time 0 to 24 Hours Postdose ELX-02

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End point title

Ae24h: Amount Excreted in Urine from Time 0 to 24 Hours Postdose ELX-02 ^[3]

End point description

Ae24h was the amount of study drug excreted in urine from Time 0 to 24 Hours post dose. The PK population included all safety subjects who had analyzable PK data without relevant deviation interfering with the PK evaluations. Here, overall number of subjects analyzed signifies subjects who were evaluable for this endpoint. Urine PK was not collected during Treatment Period 5.

End point type

Primary

End point timeframe

Treatment Period 1-4: pre-dose, and at 0-8 hours, and 8-24 hours post-dose

Notes
[3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics were performed; no inferential statistical analyses were performed.

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	10	10	8	3	0 ^[4]
Units: milligram (mg)
arithmetic mean (standard deviation)	22.043 ± 10.8882	49.499 ± 14.6477	106.350 ± 59.3726	173.822 ± 90.9374	±

Notes
[4] - Urine PK was not collected during Treatment Period 5.

No statistical analyses for this end point

Primary: Cmax: Maximum Observed Plasma Concentration for ELX-02

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End point title

Cmax: Maximum Observed Plasma Concentration for ELX-02 ^[5]

End point description

Cmax was defined as the maximum observed plasma concentration. PK population included all safety subjects who had analyzable PK data without relevant deviation interfering with the PK evaluations. Here, overall number of subjects analyzed signifies subjects who were evaluable for this endpoint.

End point type

Primary

End point timeframe

Treatment Period 1- 4 (Day 1) and Treatment Period 5 (Day 8): pre-dose and at 15 minutes, 30 minutes, 1, 3, 4, 6, 8 and 24 hours post-dose

Notes
[5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics were performed; no inferential statistical analyses were performed.

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	13	10	8	4	4
Units: nanogram per milliliter (ng/mL)
arithmetic mean (standard deviation)	1066.7 ± 187.71	2286.0 ± 551.53	4775.0 ± 1099.73	8632.5 ± 243.91	5265.0 ± 777.88

No statistical analyses for this end point

Primary: Cpeak: Maximum (Peak)- Concentration in Plasma at Expected Time of Maximum Concentration (Cpeak) for ELX-02

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End point title

Cpeak: Maximum (Peak)- Concentration in Plasma at Expected Time of Maximum Concentration (Cpeak) for ELX-02 ^[6]

End point description

Cpeak was defined as the highest level of drug concentration in plasma at expected time of maximum concentration (peak) at defined timepoints. The PK population included all safety subjects who had analyzable PK data without relevant deviation interfering with the PK evaluations. Here, "n" refers to the subjects who were evaluable for specified categories of this endpoint. Cpeak was not evaluated at treatment period 5. '99999' indicates that data could not be estimated for specified Treatment Period where data is not available due to either no value(s) within the detectable range or no sample(s) were collected.

End point type

Primary

End point timeframe

Treatment Period 1-4 (Day 7): at 30 minutes and 1 hour post dose, Treatment Period 4 (Day 14): at 30 minutes and 1 hour post dose

Notes
[6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics were performed; no inferential statistical analyses were performed.

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	13	10	8	4	0 ^[7]
Units: ng/mL
arithmetic mean (standard deviation)
Cpeak at Day 7 (n=12,9,7,4,0)	1014.2 ± 164.81	2632.2 ± 562.49	3978.6 ± 1113.39	8092.5 ± 1937.34	±
Cpeak at Day 14 (n=0,0,0,4,0)	99999 ± 99999	99999 ± 99999	99999 ± 99999	7187.5 ± 3079.91	±

Notes
[7] - Cpeak was not evaluated at treatment period 5.

No statistical analyses for this end point

Primary: Cpredose: Pre-dose Observed Plasma Concentrations Over Time for ELX-02

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End point title

Cpredose: Pre-dose Observed Plasma Concentrations Over Time for ELX-02 ^[8]

End point description

Cpredose was defined as Trough plasma concentration observed at the end of the dosing interval (that is (i.e.), at each predose starting from second dose) at defined timepoints. The PK population included all safety subjects who had analyzable PK data without relevant deviation interfering with the PK evaluations. Here, number of subjects analyzed signifies subjects who were evaluable for this endpoint. Here, "n" refers to the subjects who were evaluable for specified categories of this endpoint. '99999' indicates that data could not be estimated for specified Treatment Period where data is not available due to either no value(s) within the detectable range or no sample(s) were collected.

End point type

Primary

End point timeframe

Treatment Period 1-4: pre-dose on Days 1 and 7; Treatment Period 4: pre-dose on Day 14; Treatment Period 5: pre-dose on Days 8,14,21 and 35

Notes
[8] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics were performed; no inferential statistical analyses were performed.

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	13	10	8	4	4
Units: nanogram per milliliter (ng/mL)
arithmetic mean (standard deviation)
Predose at Day 1 (n=13,9,8,4,0)	0.00 ± 0.000	0.00 ± 0.000	427.50 ± 1209.153	0.00 ± 0.000	99999 ± 99999
Predose at Day 7 (12,10,8,4,0)	0.00 ± 0.000	0.00 ± 0.000	6.90 ± 7.793	17.65 ± 5.606	99999 ± 99999
Predose at Day 8 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	27.53 ± 15.250
Predose at Day 14 (n=0,0,0,4,3)	99999 ± 99999	99999 ± 99999	99999 ± 99999	20.42 ± 2.502	13.60 ± 11.789
Predose at Day 21 (n=0,0,0,0,3)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	34.13 ± 34.240
Predose at Day 35 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	18.05 ± 27.093

No statistical analyses for this end point

Secondary: Change from Baseline in Sweat Chloride Concentration

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End point title

Change from Baseline in Sweat Chloride Concentration

End point description

Change from Baseline in Sweat Chloride Concentration were calculated from defined timepoints and reported in this endpoint. Here, "number of subjects analysed signifies" subjects who were evaluable for this endpoint and "n" refers to the subjects who were evaluable for specified categories of this endpoint. '99999' indicates no samples were collected.

End point type

Secondary

End point timeframe

Baseline, Treatment Period 1 to 5 (Day 1): pre-dose, Treatment Period 1 to 5 (Day 7): 6 hours post-dose, Treatment Period 4 to 5 (Day 14): 6 hours post-dose, Treatment Period 5 (Days 8, 21, 28, and 35): 6 hours post-dose

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	10	8	7	4	4
Units: millimoles per liter (mmol/L)
arithmetic mean (standard deviation)
Change at Day 1 (n=0,8,7,4,1)	99999 ± 99999	-4.86 ± 14.725	-3.84 ± 8.474	-2.27 ± 5.132	1.50 ± 99999
Change at Day 7 (n=10,7,7,4,4)	1.03 ± 6.362	-1.70 ± 9.141	-4.99 ± 10.230	3.85 ± 7.341	0.55 ± 2.505
Change at Day 8 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-1.70 ± 2.482
Change at Day 14 (n=0,0,0,4,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	4.23 ± 5.616	4.80 ± 4.774
Change at Day 21 (n=0,0,0,0,3)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	1.57 ± 2.113
Change at Day 28 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.82 ± 9.378
Change at Day 35 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.20 ± 7.445

No statistical analyses for this end point

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Volume (ppFEV1)

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End point title

Change from Baseline in Percent Predicted Forced Expiratory Volume (ppFEV1)

End point description

FEV1 was measured as the volume of air that was forcibly blown out in one second, after full inspiration. These were preferably performed pre-bronchodilator, ideally at the same time at every study visit. If not performed pre-bronchodilator, it was to be performed consistently post-bronchodilator at each study visit. Here, "number of subjects analysed signifies" subjects who were evaluable for this endpoint and "n" refers to the subjects who were evaluable for specified categories of this endpoint. '99999' indicates no samples were collected.

End point type

Secondary

End point timeframe

Baseline, Treatment Period 1-5: Day 1, Day 7, Treatment Period 4-5: Day 14 and Treatment Period 5: Days 8, 21, 28 and 35

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	13	10	8	4	6
Units: Percentage of predicted FEV1
arithmetic mean (standard deviation)
Change at Day 1 (n=0,10,8,4,1)	99999 ± 99999	1.1 ± 4.48	-0.5 ± 6.91	3.8 ± 5.91	1.0 ± 99999
Change at Day 7 (n=13,9,8,4,1)	0.2 ± 4.25	0.4 ± 6.44	0.1 ± 5.99	3.5 ± 5.92	-0.2 ± 9.09
Change at Day 8 (n=0,0,0,0,5)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-1.8 ± 3.03
Change at Day 14 (n=0,0,0,4,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	46.8 ± 18.89	0.0 ± 5.89
Change at Day 21 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-4.8 ± 10.47
Change at Day 28 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-2.8 ± 6.24
Change at Day 35 (n=0,0,0,0,6)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-1.3 ± 2.50

No statistical analyses for this end point

Secondary: Change from Baseline in Percent Predicted Forced Vital Capacity (ppFVC)

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End point title

Change from Baseline in Percent Predicted Forced Vital Capacity (ppFVC)

End point description

FVC was pulmonary function test and was conducted as per the study Pulmonary Function Manual, based on the American Thoracic Society/European Respiratory Society (ATS/ERS) Consensus Statement. FVC was the maximum amount of air exhaled from the lungs after taking the deepest breath possible. The highest value was recorded into a spirometer. Here, "n" refers to the subjects who were evaluable for specified categories of this endpoint. '99999' indicates no samples were collected.

End point type

Secondary

End point timeframe

Baseline, Treatment Period 1-5: Day 1, Day 7, Treatment Period 4-5: Day 14 and Treatment Period 5: Days 8, 21, 28 and 35

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	12	10	8	4	6
Units: Percentage of predicted FCV
arithmetic mean (standard deviation)
Change at Day 1 (n=0,10,8,4,1)	99999 ± 99999	1.2 ± 8.07	1.3 ± 9.13	3.8 ± 3.77	0.0 ± 99999
Change at Day 7 (n=13,9,8,4,5)	-0.2 ± 3.67	1.1 ± 8.68	0.5 ± 7.05	1.5 ± 2.52	-2.4 ± 6.91
Change at Day 8 (n=0,0,0,0,5)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-1.6 ± 3.36
Change at Day 14 (n=0,0,0,4,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	-1.5 ± 7.59	0.0 ± 5.35
Change at Day 21 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-4.8 ± 8.92
Change at Day 28 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-2.0 ± 3.92
Change at Day 35 (n=0,0,0,0,6)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.5 ± 2.66

No statistical analyses for this end point

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Flow at 25-75% (ppFEF25-75)

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End point title

Change from Baseline in Percent Predicted Forced Expiratory Flow at 25-75% (ppFEF25-75)

End point description

Forced expiratory flow at 25-75% [FEF25-75]) was defined as how much air a person exhaled during a forced breath, measured using standard spirometry techniques. Here, "number of subjects analysed signifies" subjects who were evaluable for this endpoint and "n" refers to the subjects who were evaluable for specified categories of this endpoint. '99999' indicates no samples were collected.

End point type

Secondary

End point timeframe

Baseline, Treatment Period 1-5: Day 1, Day 7, Treatment Period 4-5: Day 14 and Treatment Period 5: Days 8, 21, 28 and 35

End point values	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: ELX-02 1.5 mg/kg	Treatment Period 4: An individualized dose up to 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Number of subjects analysed	12	9	7	3	6
Units: Percentage of predicted FEF25-75
arithmetic mean (standard deviation)
Change at Day 1 (n=0,9,7,3,1)	99999 ± 99999	0.113 ± 0.438	0.001 ± 0.364	0.280 ± 0.305	0.550 ± 99999
Change at Day 7 (n=12,8,7,3,5)	-0.015 ± 0.287	0.063 ± 0.234	0.036 ± 0.294	0.340 ± 0.344	0.142 ± 0.608
Change at Day 8 (n=0,0,0,0,5)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.120 ± 0.206
Change at Day 14 (n=0,0,0,3,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	0.300 ± 0.249	-0.047 ± 0.396
Change at Day 21 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.215 ± 0.680
Change at Day 28 (n=0,0,0,0,4)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.198 ± 0.650
Change at Day 35 (n=0,0,0,0,6)	99999 ± 99999	99999 ± 99999	99999 ± 99999	99999 ± 99999	-0.203 ± 0.460

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

From baseline up to 29 months

Adverse event reporting additional description

Safety population consisted of all treated subjects who received at least one dose of study medication, including subjects prematurely withdrawn from the study.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

25.0

Reporting group title

Treatment Period 1: ELX-02 0.3 mg/kg

Reporting group description

Reporting group title

Treatment Period 2: ELX-02 0.75 mg/kg

Reporting group description

Subjects received ELX-02 0.75 mg/kg SC daily for 1 week (about 0.9 mL/day, total dose not to exceed 5.25 mg/kg for this 7-day period) in Treatment Period 2.

Reporting group title

Treatment Period 3: 1.5 mg/kg

Reporting group description

Subjects received ELX-02 1.5 mg/kg SC daily for 1 week (two daily injections of about 0.9 mL, total dose not to exceed 10.5 mg/kg for this 7-day period) in Treatment Period 3.

Reporting group title

Treatment Period 4: 3 mg/kg

Reporting group description

Reporting group title

Treatment Period 5: ELX-02 + Ivacaftor

Reporting group description

Serious adverse events	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: 1.5 mg/kg	Treatment Period 4: 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
number of deaths (all causes)	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma gastric
Additional description: Serious TEAE subject identified 3 months after completing treatment period 3 during dosing hiatus.
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Gastric cancer
Additional description: Serious TEAE subject identified 3 months after completing treatment period 3 during dosing hiatus.
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 0%

Non-serious adverse events	Treatment Period 1: ELX-02 0.3 mg/kg	Treatment Period 2: ELX-02 0.75 mg/kg	Treatment Period 3: 1.5 mg/kg	Treatment Period 4: 3 mg/kg	Treatment Period 5: ELX-02 + Ivacaftor
Total subjects affected by non serious adverse events
subjects affected / exposed	7 / 13 (53.85%)	6 / 10 (60.00%)	4 / 8 (50.00%)	4 / 4 (100.00%)	5 / 6 (83.33%)
General disorders and administration site conditions
Injection site pain
subjects affected / exposed	3 / 13 (23.08%)	1 / 10 (10.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	5	1	2	0	0
Injection site erythema
subjects affected / exposed	0 / 13 (0.00%)	2 / 10 (20.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	4	0	0	0
Injection site swelling
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	0	0	2	1	0
Injection site oedema
subjects affected / exposed	0 / 13 (0.00%)	1 / 10 (10.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	3	1	0	0
Injection site induration
subjects affected / exposed	0 / 13 (0.00%)	1 / 10 (10.00%)	1 / 8 (12.50%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	0	1	1	1	0
Injection site haematoma
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	1	0
Fatigue
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Injection site dryness
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0	0
Injection site pruritus
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Injection site reaction
subjects affected / exposed	0 / 13 (0.00%)	1 / 10 (10.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0	0
Injection site vesicles
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Non-cardiac chest pain
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Immune system disorders
Allergy to animal
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	0	0	0	1	0
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	1	0	1
Haemoptysis
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1
Rales
subjects affected / exposed	0 / 13 (0.00%)	1 / 10 (10.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0	0
Sputum increased
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Psychiatric disorders
Depression
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0	0
Investigations
Blood creatine phosphokinase increased
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	0	0	0	1	0
Tympanometry abnormal
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1
Nervous system disorders
Dizziness
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Presyncope
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1
Blood and lymphatic system disorders
Anaemia
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0	0
Ear and labyrinth disorders
Tinnitus
subjects affected / exposed	3 / 13 (23.08%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	4	0	0	0	2
Ear pain
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	1	0	0
Vertigo
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0	0
Gastrointestinal disorders
Vomiting
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	1	0	0
Abdominal pain
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0	0
Diarrhoea
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0	0
Skin and subcutaneous tissue disorders
Erythema
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	0	3	0	0
Skin induration
subjects affected / exposed	0 / 13 (0.00%)	1 / 10 (10.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0	0
Musculoskeletal and connective tissue disorders
Back pain
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	0	0	0	1	0
Myalgia
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
subjects affected / exposed	3 / 13 (23.08%)	0 / 10 (0.00%)	1 / 8 (12.50%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	6	0	1	0	0
Bacterial disease carrier
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	2 / 4 (50.00%)	0 / 6 (0.00%)
occurrences all number	0	0	0	2	0
Nasopharyngitis
subjects affected / exposed	1 / 13 (7.69%)	1 / 10 (10.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	1	0	0	0
Influenza
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1
Respiratory tract infection
subjects affected / exposed	0 / 13 (0.00%)	1 / 10 (10.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0	0
Upper respiratory tract infection
subjects affected / exposed	1 / 13 (7.69%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0	0
Viral infection
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	0 / 4 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	0	1
Metabolism and nutrition disorders
Hyperglycaemia
subjects affected / exposed	0 / 13 (0.00%)	0 / 10 (0.00%)	0 / 8 (0.00%)	1 / 4 (25.00%)	0 / 6 (0.00%)
occurrences all number	0	0	0	1	0

More information

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Were there any global substantial amendments to the protocol? Yes

04 Sep 2019

Amendment 1.0: 1) Updated the nonclinical and clinical safety information (based on studies that had been completed since the original version was submitted). 2) Further defined and clarified the objectives and endpoints. 3) Updated the description of the study design to more clearly delineate how the subjects will progress through the study. 4) Provided a clear justification on the rationale for the recommended doses, duration of study drug administration, and the modification of the dosing regimen from twice weekly to daily. 5) Modified number of subjects to permit intra-subject dose escalation. 6) Adjusted the inclusion/exclusion criteria to provide clarity as to the nonsense alleles most appropriate for treatment with the investigational product under study, increased age of entry from 16 to 18 in Germany, as well as more clearly defining the original criteria. 7) Enhanced and provided additional guidance on the protocol defined stopping rules. 8) Reduced subject burden by simplifying procedures, modifying the PK schedule, decrease in-patient hospital stays, as well as offering an optional hiatus between treatment periods. 9) Additional changes to provide clarity, correct errors, and additional detail for certain sections were also made in order to increase the understanding of the protocol.

14 Feb 2020

Amendment 2.0: 1) Modified number of homozygous subjects to reflect actual availability of these subjects and to further define the type of heterozygous subjects to be allowed in the study. 2) Clarified the return from hiatus procedures. 3) Enhanced and provided additional guidance on the protocol defined stopping rules. 4) Reduced subject burden by simplifying procedures. 5) Removed exploratory objectives/endpoints that were no longer to be evaluated in this study. 6) Additional changes to provide clarity, correct errors, and additional detail for certain sections were also made in order to increase the understanding of the protocol.

06 Oct 2020

Amendment 3.0: Permitted subjectss to be enrolled into the study with F508del mutation on the second allele only if not receiving any CFTR modulators or potentiators for 2 months prior to study treatment.

12 Nov 2020

Amendment 4.0: Included measures taken to accommodate COVID-19 pandemic situation in the protocol.

27 Jan 2021

Amendment 4.1: Israel specific changes: 1) Added Treatment Period 5 – ELX-02 1.5mg/kg in combination with ivacaftor 150 mg twice daily for 28 days. 2) Provided rationale for the combined therapy in Treatment Period 5. 3) Provided support for 28 days administration of ELX-02. 3) Amended the study objective/endpoints to include the combined therapy in Treatment period 5. 4) Additional changes to provide clarity, correct errors, and additional detail for certain sections were also made in order to increase the understanding of the protocol.

15 Jul 2021

Amendment 5.0: 1) Included subjects with CF who have phenotypically similar disease to G542X mutations. 2) Addition of a 1-week monotherapy run-in period for Treatment Period 5. 3) One week of monotherapy run-in allowed the generation of contemporaneous monotherapy and combination therapy data making the interpretation of pharmacodynamic effect of ELX-02 more robust. 4) Made Treatment Periods 1 to 4 (TP1 to TP4) optional.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results:
A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs), Severe TEAEs, Serious TEAEs, Related TEAEs, Related Serious TEAE, TEAE Leading to Study Drug Discontinuation and Death

Primary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs), Severe TEAEs, Serious TEAEs, Related TEAEs, Related Serious TEAE, TEAE Leading to Study Drug Discontinuation and Death

Primary: AUC(0-24h): Area Under the Plasma Concentration-time Curve from Time 0 to 24 Hours Postdose for ELX-02

Primary: AUC(0-24h): Area Under the Plasma Concentration-time Curve from Time 0 to 24 Hours Postdose for ELX-02

Primary: Ae24h: Amount Excreted in Urine from Time 0 to 24 Hours Postdose ELX-02

Primary: Ae24h: Amount Excreted in Urine from Time 0 to 24 Hours Postdose ELX-02

Primary: Cmax: Maximum Observed Plasma Concentration for ELX-02

Primary: Cmax: Maximum Observed Plasma Concentration for ELX-02

Primary: Cpeak: Maximum (Peak)- Concentration in Plasma at Expected Time of Maximum Concentration (Cpeak) for ELX-02

Primary: Cpeak: Maximum (Peak)- Concentration in Plasma at Expected Time of Maximum Concentration (Cpeak) for ELX-02

Primary: Cpredose: Pre-dose Observed Plasma Concentrations Over Time for ELX-02

Primary: Cpredose: Pre-dose Observed Plasma Concentrations Over Time for ELX-02

Secondary: Change from Baseline in Sweat Chloride Concentration

Secondary: Change from Baseline in Sweat Chloride Concentration

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Volume (ppFEV1)

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Volume (ppFEV1)

Secondary: Change from Baseline in Percent Predicted Forced Vital Capacity (ppFVC)

Secondary: Change from Baseline in Percent Predicted Forced Vital Capacity (ppFVC)

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Flow at 25-75% (ppFEF25-75)

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Flow at 25-75% (ppFEF25-75)

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Outside EU/EEA

Clinical trials

Clinical Trial Results: A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs), Severe TEAEs, Serious TEAEs, Related TEAEs, Related Serious TEAE, TEAE Leading to Study Drug Discontinuation and Death

Primary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs), Severe TEAEs, Serious TEAEs, Related TEAEs, Related Serious TEAE, TEAE Leading to Study Drug Discontinuation and Death

Primary: AUC(0-24h): Area Under the Plasma Concentration-time Curve from Time 0 to 24 Hours Postdose for ELX-02

Primary: AUC(0-24h): Area Under the Plasma Concentration-time Curve from Time 0 to 24 Hours Postdose for ELX-02

Primary: Ae24h: Amount Excreted in Urine from Time 0 to 24 Hours Postdose ELX-02

Primary: Ae24h: Amount Excreted in Urine from Time 0 to 24 Hours Postdose ELX-02

Primary: Cmax: Maximum Observed Plasma Concentration for ELX-02

Primary: Cmax: Maximum Observed Plasma Concentration for ELX-02

Primary: Cpeak: Maximum (Peak)- Concentration in Plasma at Expected Time of Maximum Concentration (Cpeak) for ELX-02

Primary: Cpeak: Maximum (Peak)- Concentration in Plasma at Expected Time of Maximum Concentration (Cpeak) for ELX-02

Primary: Cpredose: Pre-dose Observed Plasma Concentrations Over Time for ELX-02

Primary: Cpredose: Pre-dose Observed Plasma Concentrations Over Time for ELX-02

Secondary: Change from Baseline in Sweat Chloride Concentration

Secondary: Change from Baseline in Sweat Chloride Concentration

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Volume (ppFEV1)

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Volume (ppFEV1)

Secondary: Change from Baseline in Percent Predicted Forced Vital Capacity (ppFVC)

Secondary: Change from Baseline in Percent Predicted Forced Vital Capacity (ppFVC)

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Flow at 25-75% (ppFEF25-75)

Secondary: Change from Baseline in Percent Predicted Forced Expiratory Flow at 25-75% (ppFEF25-75)

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele