Clinical Trials Register

Summary
EudraCT Number:	2020-004403-14
Sponsor's Protocol Code Number:	BCX9930-203
National Competent Authority:	Lithuania - SMCA
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-08-18
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Lithuania - SMCA

A.2

EudraCT number

2020-004403-14

A.3

Full title of the trial

A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

Atsitiktinių imčių, dvigubai koduotas, daugiacentris, placebu kontroliuojamas, lygiagrečių grupių tyrimas, skirtas įvertinti vartojamo per burną BCX9930 monoterapijos veiksmingumą, saugumą bei toleravimą gydant paroksizminę naktinę hemoglobinuriją

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

BCX9930 compared to placebo in the treatment of PNH

A.3.2

Name or abbreviated title of the trial where available

REDEEM 2

A.4.1

Sponsor's protocol code number

BCX9930-203

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT05116787

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	BioCryst Pharmaceuticals Inc
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	BioCryst Pharmaceuticals Inc
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AMS Advanced Medical Services
B.5.2	Functional name of contact point	Project Management
B.5.3	Address:
B.5.3.1	Street Address	26-28 Hammersmith Grove
B.5.3.2	Town/ city	London
B.5.3.3	Post code	W67BA
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	+44208834 1144
B.5.5	Fax number	+44208834 1156
B.5.6	E-mail	operations@ams-europe.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	BCX9930
D.3.2	Product code	BCX9930
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	BCX9930
D.3.9.2	Current sponsor code	BCX9930
D.3.9.3	Other descriptive name	BCX9930
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	250
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	BCX9930
D.3.2	Product code	BCX9930
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	BCX9930
D.3.9.2	Current sponsor code	BCX9930
D.3.9.3	Other descriptive name	BCX9930
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Paroxysmal Nocturnal Hemoglobinuria

E.1.1.1

Medical condition in easily understood language

PNH

E.1.1.2

Therapeutic area

Body processes [G] - Immune system processes [G12]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10034042
E.1.2	Term	Paroxysmal nocturnal haemoglobinuria
E.1.2	System Organ Class	10038359 - Renal and urinary disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

• To determine the efficacy of oral BCX9930 monotherapy administered for 12 weeks, as compared to placebo, in subjects with paroxysmal nocturnal hemoglobinuria (PNH)
• To evaluate the long-term safety and tolerability of oral BCX9930 monotherapy administered for up to 52 weeks in subjects with PNH

E.2.2

Secondary objectives of the trial

• To evaluate the safety and tolerability of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, in subjects with PNH
• To characterize the effects of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, using clinical and laboratory measurements, including complement and thrombosis biomarkers, and PNH clone size
• To evaluate the effects of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, on FACIT-Fatigue scale and other PROs
• To assess the effectiveness of BCX9930 monotherapy administered for up to 52 weeks
• To characterize the effects of BCX9930 monotherapy administered for up to 52 weeks using clinical and laboratory measurements, including complement and thrombosis biomarkers, and PNH clone size
• To evaluate the effects of BCX9930 monotherapy administered for up to 52 weeks on FACIT-Fatigue scale and other PROs
• To characterize BCX9930 plasma concentrations and pharmacokinetic (PK) parameters

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

PK/PD substudy - within the same protocol

E.3

Principal inclusion criteria

1. Male or female, aged ≥ 18 years old.
2. Body weight ≥ 40 kg.
3. Documented diagnosis of PNH confirmed by flow cytometry with a PNH granulocyte or monocyte clone size of ≥ 10% during screening.
4. Are either: (a) naïve to treatment with a complement inhibitor, or (b) have received no treatment with a complement inhibitor for at least 12 months prior to the screening visit.
5. Do not have access to or have a contraindication (ie, have had a serious adverse reaction) to approved complement, C5 or C3, inhibitor therapies
6. Recorded the following results during screening:
a. Hb ≤ 105 g/L (≤ 10.5 g/dL).
b. LDH ≥ 2 × upper limit of normal reference range (ULN)
c. ARC of ≥ 100 × 109 cells/L (≥ 100,000 cells/µL; ≥ 100 G/L).
d. Absolute neutrophil count (ANC) of ≥ 0.75 × 109 cells/L (≥ 750 cells/µL; ≥ 0.75 G/L).
e. Platelet count of ≥ 30 × 109/L (≥ 30,000/µL; ≥ 30 G/L).
f. Estimated glomerular filtration rate of ≥ 60 mL/min/1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Levey and Stevens 2010) and no evidence of clinically relevant abnormal renal function unrelated to underlying PNH disease.
7. Contraception requirements:
Female participants must meet at least one of the following requirements:
a. Be a woman of nonchildbearing potential.
b. Be a woman of childbearing potential who agrees to use a highly effective contraceptive method throughout the study and for a duration of 30 days after the last dose of study drug.
c. Alternatively, true abstinence is acceptable for women of childbearing potential when it is in line with the subject’s preferred and usual lifestyle.
Male participants must meet at least one of the following requirements:
a. Males with a female partner of childbearing potential must use condoms throughout the study and for a duration of 90 days after the dose of study drug unless their partner is using a highly effective contraceptive method independent of the study.
b. Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle
8. Documentation of current vaccinations against Neisseria meningitidis types A, C, W, and Y, and Streptococcus pneumoniae, or willingness to start vaccination series at least 14 days prior to Day 1.
9. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study, including compliance with the BID dosing schedule for BCX9930.
10. Willing and able to provide written informed consent

E.4

Principal exclusion criteria

1. Known history of or existing diagnosis of hereditary complement deficiency.
2. History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
3. Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
4. History of malignancy within 5 years prior to the screening visit, with exception of adequately treated non-melanoma skin or superficial bladder cancer, curatively treated carcinoma in situ of the cervix, or other curatively treated solid tumor deemed by the investigator and medical monitor to be at low risk for recurrence.
5. Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening.
(Note: Suspected or confirmed coronavirus disease [COVID-19]; persistent or recurrent positive test(s) for severe acute respiratory syndrome coronavirus 2 [SARS CoV 2] nucleic acids or antigens; and worsening of dyspnea not due to PNH, vasculitic rash, and persistent fever or other symptoms consistent with multisystem inflammatory syndrome in adults [MIS A] are exclusionary.)
6. Current participation in any other investigational drug study or participation in an investigational drug study within 30 days prior to the screening visit, or 5.5 half-lives of the investigational drug, whichever is longer.
7. Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
8. Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit.
(Note: Treatment with these medications initiated > 28 days prior to the screening visit is not exclusionary, if the dose is stable and there is a reasonable expectation that treatment will be continued.)
9. Receiving iron with an unstable dose in the 28 days prior to the screening visit.
10. Clinically significant abnormal electrocardiogram (ECG) at the screening visit.
(Note: This includes, but is not limited to, a QT interval corrected using Fridericia’s method [QTcF] of > 450 msec in males or > 470 msec in females, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping.)
11. Subjects with any of the following results at the screening visit:
a. Alanine aminotransferase (ALT; also serum glutamic-pyruvic transaminase [SGPT]) > 3 × ULN.
b. AST (SGOT) > 3 × ULN.
(Note: Subjects may be enrolled with AST > 3 × ULN if explained by hemolysis.)
c. Total serum bilirubin > 2 × ULN
(Note: Subjects may be enrolled with total serum bilirubin > 2 × ULN if explained by hemolysis or Gilbert’s syndrome. In the case of hemolysis, total serum bilirubin must be < 5 × ULN and in the case of Gilbert’s syndrome, total serum bilirubin must be < 11 × ULN.)
12. Current use of a prohibited concomitant medication within 7 days prior to Day 1
13. Positive serology for human immunodeficiency virus, or active infection with hepatitis B virus or hepatitis C virus, unless receiving antiviral therapy and viral load is undetectable.
14. Positive drugs of abuse screen, unless by prescription.
15. Pregnant, planning to become pregnant, or breastfeeding.
16. Known hypersensitivity to BCX9930 or any of its formulation excipients.
17. History of severe hypersensitivity to any medicinal product, which was associated with swelling, severe rash requiring treatment/hospitalization, or anaphylaxis.
18. Any other clinically significant medical or psychiatric condition that, in the opinion of the investigator or sponsor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject.

E.5 End points

E.5.1

Primary end point(s)

Part1
• Change from baseline (CFB) in hemoglobin (Hb)
Part2
• Number and proportion of subjects with a TEAE
• Number and proportion of subjects who discontinue due to a TEAE
• Number and proportion of subjects who experience a TESAE
• Number and proportion of subjects who experience a CTCAE Grade 3 or Grade 4 TEAE
• Number and proportion of subjects who experience a treatment-emergent CTCAE Grade 3 or Grade 4 laboratory abnormality

E.5.1.1

Timepoint(s) of evaluation of this end point

Part 1 - Week 12
Part 2 - week 52

E.5.2

Secondary end point(s)

Part 1
1. Proportion of subjects who are transfusion-free
2. Number of units of packed red blood cells (pRBCs) transfused
3. Percent CFB in lactate dehydrogenase (LDH)
4. CFB in FACIT-Fatigue scale score
Part 2
• CFB in Hb
• Proportion of subjects with Hb ≥ 12 g/dL
• Proportion of subjects achieving Hb stabilization (avoidance of a > 2 g/dL decrease in the absence of transfusion)
• Proportion of subjects who are transfusion-free
• Percent reduction in rate of pRBCs transfused
• Percent CFB in LDH
• CFB in ARC
• Proportion of subjects with ARC in the normal range
• CFB in haptoglobin
• Proportion of subjects with haptoglobin ≥ LLN
• CFB in total PNH RBC clone size
• CFB in total PNH RBC clone size relative to PNH WBC clone size

E.5.2.1

Timepoint(s) of evaluation of this end point

Part 1 - Week 12
Part 2 - week 52

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Argentina

Brazil

Canada

China

South Africa

United States

European Union

Albania

Azerbaijan

Turkey

Serbia

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Subjects who are experiencing clinical benefit will be offered the opportunity to continue to receive BCX9930 via an long term access protocol

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2021-10-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-09-09

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-09-18

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