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Clinical Trial Results:
A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene

Summary
EudraCT number	2018-002433-38
Trial protocol	FR
Global end of trial date	14 Oct 2021

Results information
Results version number	v1(current)
This version publication date	15 Dec 2022
First version publication date	15 Dec 2022
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

PQ-421a-001

ISRCTN number

US NCT number

NCT03780257

WHO universal trial number (UTN)

Sponsor organisation name

ProQR Therapeutics

Sponsor organisation address

Zernikedreef 9, Leiden, Netherlands, 2333 CK

Public contact

Clinical Trial Manager, ProQR Therapeutics, 31 881667000, clinical@proqr.com

Scientific contact

Clinical Trial Manager, ProQR Therapeutics, 31 881667000, clinical@proqr.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

07 Dec 2021

Is this the analysis of the primary completion data?

Yes

Primary completion date

14 Oct 2021

Global end of trial reached?

Yes

Global end of trial date

14 Oct 2021

Was the trial ended prematurely?

Main objective of the trial

Primary objective: - To evaluate the safety and tolerability of QR-421a. Secondary objectives: - To evaluate the efficacy of QR-421a, as assessed by functional and structural outcome measures - To evaluate the dose-response and duration of structural and functional effects of a single dose of QR-421a - To evaluate the serum pharmacokinetics (PK) of QR-421a Exploratory objectives: - To evaluate changes in Patient-Reported Outcome (PRO) measures - To evaluate serum biomarkers

Protection of trial subjects

The study was conducted in accordance with ethical principles that have their origin in the Declaration of Helsinki and are consistent with ICH GCP. QR-421a was administered via intravitreal (IVT) injection to maximize exposure to study drug at the intended target tissue, the retina. QR-421a was administered in a unilateral manner to the subject’s worse eye, a common practice in the development of ophthalmic products. Following IVT injection of QR-421a, subjects were monitored for increases in intraocular pressure (IOP) and signs of inflammation and endophthalmitis during the post-injection period. Subjects were also seen at 1, 2, and 7 days after each injection. An independent Data Monitoring Committee (DMC) was involved to recommend on dose escalation and provided safety oversight for the study.

Background therapy

Evidence for comparator

Actual start date of recruitment

06 Mar 2019

Long term follow-up planned

Yes

Long term follow-up rationale

Safety, Efficacy

Long term follow-up duration

24 Months

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

France: 8

Country: Number of subjects enrolled

Canada: 2

Country: Number of subjects enrolled

United States: 10

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

Twenty subjects were enrolled in this study. The study plan included up to 3 cohorts. Each cohort was to have at least 4 subjects treated and 2 to receive sham-procedure (for cohort 1 and 2). Additional dose levels could have been evaluated based on ongoing safety and efficacy data monitoring, but were never utilized.

Screening details

Key inclusion criteria were: >= 18 years at Screening, a clinical diagnosis of RP with Usher syndrome type 2 or NSRP, a molecular diagnosis for 1 or more pathogenic exon 13 mutations in the USH2A gene, and impairment of VF as determined by perimetry with a continuous area of central field >= 10 degrees diameter in any axis in the treatment eye.

Period 1 title

Overall trial (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor

Blinding implementation details

Each sentinel subject was treated in an open-label fashion, as were subjects enrolled in expansion Cohort 2b (100 μg) and Cohort 3 (200 μg). All other subjects were double-masked and randomized at a ratio of 2:1 for QR-421a and sham, respectively.

Are arms mutually exclusive

Yes

Cohort 1 (50 μg)

Arm description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Arm type

Experimental

Investigational medicinal product name

Ultevursen

Investigational medicinal product code

QR-421a

Other name

Pharmaceutical forms

Concentrate for solution for injection

Routes of administration

Intravitreal use

Dosage and administration details

QR-421a solution for injection, 15 mg/mL, at a dose of 50μg. Subjects received QR-421a by a single unilateral IVT injection. Each subject received a single dose in their worse eye, as defined by the BCVA at the most recent screening visit and subsequently referred to as the TE and were assessed for safety and tolerability at follow-up visits.

Cohort 2 (100 μg)

Arm description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Arm type

Experimental

Investigational medicinal product name

Ultevursen

Investigational medicinal product code

QR-421a

Other name

Pharmaceutical forms

Concentrate for solution for injection

Routes of administration

Intravitreal use

Dosage and administration details

QR-421a solution for injection, 15 mg/mL, at a dose of 100μg. Subjects received QR-421a by a single unilateral IVT injection. Each subject received a single dose in their worse eye, as defined by the BCVA at the most recent screening visit and subsequently referred to as the TE and were assessed for safety and tolerability at follow-up visits.

Cohort 3 (200 μg)

Arm description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Arm type

Experimental

Investigational medicinal product name

Ultevursen

Investigational medicinal product code

QR-421a

Other name

Pharmaceutical forms

Concentrate for solution for injection

Routes of administration

Intravitreal use

Dosage and administration details

QR-421a solution for injection, 15 mg/mL, at a dose of 200μg. Subjects received QR-421a by a single unilateral IVT injection. Each subject received a single dose in their worse eye, as defined by the BCVA at the most recent screening visit and subsequently referred to as the TE and were assessed for safety and tolerability at follow-up visits.

Sham group

Arm description

Subjects received a sham procedure closely mimicking the active injection without globe penetration.

Arm type

Sham procedure

Investigational medicinal product name

No investigational medicinal product assigned in this arm

Number of subjects in period 1	Cohort 1 (50 μg)	Cohort 2 (100 μg)	Cohort 3 (200 μg)	Sham group
Started	4	7	3	6
Completed	4	7	3	6

Baseline characteristics

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Reporting group title

Cohort 1 (50 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Cohort 2 (100 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Cohort 3 (200 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Sham group

Reporting group description

Subjects received a sham procedure closely mimicking the active injection without globe penetration.

Reporting group values	Cohort 1 (50 μg)	Cohort 2 (100 μg)	Cohort 3 (200 μg)	Sham group	Total
Number of subjects	4	7	3	6	20
Age categorical
Units: Subjects
In utero	0	0	0	0	0
Preterm newborn infants (gestational age < 37 wks)	0	0	0	0	0
Newborns (0-27 days)	0	0	0	0	0
Infants and toddlers (28 days-23 months)	0	0	0	0	0
Children (2-11 years)	0	0	0	0	0
Adolescents (12-17 years)	0	0	0	0	0
Adults (18-64 years)	3	7	3	6	19
From 65-84 years	1	0	0	0	1
85 years and over	0	0	0	0	0
Age continuous
Units: years
median (full range (min-max))	51.5 (30 to 65)	54.0 (25 to 58)	39.0 (33 to 54)	44.5 (24 to 60)	-
Gender categorical
Units: Subjects
Female	4	3	3	2	12
Male	0	4	0	4	8

End points

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Reporting group title

Cohort 1 (50 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Cohort 2 (100 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Cohort 3 (200 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Sham group

Reporting group description

Subjects received a sham procedure closely mimicking the active injection without globe penetration.

Primary: Incidence and severity of ocular AEs (Treated Eye)

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End point title

Incidence and severity of ocular AEs (Treated Eye) ^[1]

End point description

Only the incidence is reported in a tabular format. Nine subjects in the treatment group had mild severity ocular TEAEs in the TE (3 subjects in each group) and 1 subject in the 50 μg group had a moderate severity ocular AE.

End point type

Primary

End point timeframe

Treatment emergent AEs until End of Study.

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: No statistics were planned for safety.

End point values	Cohort 1 (50 μg)	Cohort 2 (100 μg)	Cohort 3 (200 μg)	Sham group
Number of subjects analysed	4	7	3	6
Units: subjects	4	3	3	5

No statistical analyses for this end point

Primary: Incidence and severity of non-ocular AEs

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End point title

Incidence and severity of non-ocular AEs ^[2]

End point description

Only the incidence is reported in a tabular format. Five subjects in the treatment group had mild severity non-ocular TEAEs in the TE (1 subjects in each group, except for 2 subjects in the 50 μg group), 7 subjects had moderate severity non-ocular TEAEs (2 subjects in each group, except for 1 subject in the 200 μg group), and 1 subject in the sham-group experienced 2 severe non-ocular TEAEs.

End point type

Primary

End point timeframe

Treatment emergent AEs until End of Study.

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: No statistics were planned for safety.

End point values	Cohort 1 (50 μg)	Cohort 2 (100 μg)	Cohort 3 (200 μg)	Sham group
Number of subjects analysed	4	7	3	6
Units: subjects	4	3	2	4

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Information regarding occurrence of (S)AEs was captured throughout the study. Treatment Emergent Ocular Adverse Events for the Treated Eye are provided.

Adverse event reporting additional description

(S)AEs were collected from screening, and classed as medical history, non-treatment emergent AE or AE depending on relation to study drug administration. Duration (start and stop dates), severity/grade, outcome, treatment, and relationship to study drug were recorded. Pre-specified ocular related AEs were considered AEs of Special Interest.

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

21.1

Reporting group title

Cohort 1 (50 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Cohort 2 (100 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Cohort 3 (200 μg)

Reporting group description

Subjects received their dose of ultevursen (QR-421a) on Day 1 via IVT injection into the subject’s treated eye (TE).

Reporting group title

Sham group

Reporting group description

Subjects received a sham procedure closely mimicking the active injection without globe penetration.

Serious adverse events	Cohort 1 (50 μg)	Cohort 2 (100 μg)	Cohort 3 (200 μg)	Sham group
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
number of deaths (all causes)	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0
Investigations
White blood cell count increased
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Appendicitis
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0

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

Non-serious adverse events	Cohort 1 (50 μg)	Cohort 2 (100 μg)	Cohort 3 (200 μg)	Sham group
Total subjects affected by non serious adverse events
subjects affected / exposed	4 / 4 (100.00%)	4 / 7 (57.14%)	3 / 3 (100.00%)	5 / 6 (83.33%)
Investigations
Intraocular pressure increased
subjects affected / exposed	1 / 4 (25.00%)	1 / 7 (14.29%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	1	0	0
Injury, poisoning and procedural complications
Intra-ocular injection complication
subjects affected / exposed	0 / 4 (0.00%)	1 / 7 (14.29%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0
Post procedural haemorrhage
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Immune system disorders
Drug hypersensitivity
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0
Eye disorders
Eye pain
subjects affected / exposed	1 / 4 (25.00%)	1 / 7 (14.29%)	1 / 3 (33.33%)	2 / 6 (33.33%)
occurrences all number	3	1	1	2
Lacrimation increased
subjects affected / exposed	2 / 4 (50.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	2	0	1	0
Anterior chamber cell
subjects affected / exposed	0 / 4 (0.00%)	1 / 7 (14.29%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	0	2	1	0
Conjunctival haemorrhage
subjects affected / exposed	1 / 4 (25.00%)	1 / 7 (14.29%)	0 / 3 (0.00%)	2 / 6 (33.33%)
occurrences all number	1	1	0	2
Vision blurred
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	1 / 6 (16.67%)
occurrences all number	2	0	4	2
Anterior chamber flare
subjects affected / exposed	0 / 4 (0.00%)	1 / 7 (14.29%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0
Cataract subcapsular
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Conjunctival hyperaemia
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	1 / 6 (16.67%)
occurrences all number	0	0	1	1
Conjunctivitis allergic
subjects affected / exposed	0 / 4 (0.00%)	1 / 7 (14.29%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0
Cystoid macular oedema
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	0	0	4	0
Dry eye
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0
Eye irritation
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	2	0	0	0
Eye pruritus
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	1 / 6 (16.67%)
occurrences all number	0	0	2	1
Loss of visual contrast sensitivity
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Macular fibrosis
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0
Metamorphopsia
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	2	0	0	0
Ocular hyperaemia
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Ocular hypertension
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	1 / 3 (33.33%)	0 / 6 (0.00%)
occurrences all number	0	0	1	0
Posterior capsule opacification
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Retinal cyst
subjects affected / exposed	0 / 4 (0.00%)	1 / 7 (14.29%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	0	1	0	0
Retinal disorder
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Visual impairment
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	3	0	0	2
Vitreous floaters
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Vitreous opacities
subjects affected / exposed	1 / 4 (25.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	0 / 6 (0.00%)
occurrences all number	1	0	0	0
Corneal opacity
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	1
Foreign body sensation in eyes
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	1
Maculopathy
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	1
Photopsia
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	1
Swelling of eyelid
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	1
Vitreous detachment
subjects affected / exposed	0 / 4 (0.00%)	0 / 7 (0.00%)	0 / 3 (0.00%)	1 / 6 (16.67%)
occurrences all number	0	0	0	1

More information

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Were there any global substantial amendments to the protocol? Yes

24 Jul 2019

• Increase in the follow-up duration from 12 to 24 months, to enhance subject patient safety and to maximize information derived from the trial; revision of screening duration from 2-6 to 8 weeks, to decrease subject burden • Revision in the timing of when replacement of subjects can occur • Increase in time to treatment of the CE from 24 weeks to 12 months, for safety purposes • Removal of kinetic perimetry and near infrared imaging, and the addition of LLVA and FAF as endpoints • Changes to timing of repeated measures of specified visual assessments to reduce redundancy and allow more flexibility in the screening window (for practical reasons) • Updates in the description of the study population, including revision of inclusion and exclusion criteria pertaining to: o BCVA entry limits (deletion) o Kinetic VF requirements (deletion) o SD-OCT entry criteria (modification) o Nystagmus, cardiovascular disease (deletion) o Amblyopia (modification) o eGFR, LFTs entry criteria, coagulation parameters, systemic disease (deletion) o Intraocular or periocular surgery or IVT injection (modification) • The addition of the potential for re-screening of screen failures • Inclusion of more stringent birth control criteria and requirements for females of child-bearing potential • Clarification that use of general anesthesia is not allowed • Removal of physical examination by Investigators • Removal of the use of external experts to assess changes in retinal structure or visual function • Modification of and addition to AESI criteria

19 Aug 2019

• Modification in the staggering of dosing between sentinel subjects and remaining subjects for Cohorts 2+ from 4 weeks to 1 week

17 Jan 2020

• Modification of overall study duration • Addition of FST as an outcome measure • Change in study design such that expansion cohorts and additional cohorts will be open-label rather than double-masked

08 May 2020

• Removal of ERG assessment throughout study • Removal of Streetlab substudy • Removal of wording regarding treatment of the CE and/or redosing of the TE • Removal of study visits at Month 4 and Month 5 • Reduction of study assessments in the Month 9 visit to only safety assessments • Removal of FAF assessment at Month 18 • Reduced blood sampling schedules for hematology and coagulation, serum chemistry, PK, and serum biomarkers

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 First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Incidence and severity of ocular AEs (Treated Eye)

Primary: Incidence and severity of ocular AEs (Treated Eye)

Primary: Incidence and severity of non-ocular AEs

Primary: Incidence and severity of non-ocular AEs

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Clinical trials

Clinical Trial Results: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Incidence and severity of ocular AEs (Treated Eye)

Primary: Incidence and severity of ocular AEs (Treated Eye)

Primary: Incidence and severity of non-ocular AEs

Primary: Incidence and severity of non-ocular AEs

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene