BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

— REDEEM-2  

Official title:

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 PNH

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05116787
• Other study ID #: BCX9930-203
• Secondary ID: 2020-004403-14
• Status: Terminated
• Phase: Phase 2
• Start date: October 26, 2021
• Est. completion date: September 14, 2023

Study information

• Verified date: December 2022
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult patients with PNH not currently receiving complement inhibitor therapy.

Description:

This is a randomized, placebo-controlled, double-blind, parallel-group, 2-part study. Parts 1 and 2 will be conducted in the same subjects.

Part 1 of the study is designed to evaluate the efficacy, safety, and tolerability of treatment with oral BCX9930 monotherapy for 12 weeks versus placebo in subjects with PNH who are not currently receiving treatment with complement inhibitor therapy. Subjects will be randomized to receive BCX9930 or placebo under double blind conditions for the 12-week randomized treatment period. The primary efficacy and safety analyses will be based on Part 1.

Part 2 of the study is designed to evaluate the long-term safety, tolerability, and effectiveness of open-label BCX9930 monotherapy when administered through Week 52. All subjects in Part 2 will receive BCX9930. Subjects who are randomized to BCX9930 monotherapy in Part 1 will continue to receive BCX9930 in Part 2. Subjects who are randomized to placebo in Part 1 will discontinue that therapy at the Week 12 visit and receive BCX9930 in Part 2.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 12
• Est. completion date: September 14, 2023
• Est. primary completion date: September 14, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Male or female, aged = 18 years old

• Body weight = 40 kg

• Documented diagnosis of PNH

• No complement inhibitor therapy for = 12 months prior to screening

• Contraindication to or no access to approved (C3 or C5) complement inhibitor therapies

• Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series

• At screening: PNH clone of = 10%, hemoglobin = 10.5 g/dL and lactate dehydrogenase = 2 × upper limit of normal

Exclusion Criteria:

• Known history of or existing diagnosis of hereditary complement deficiency

• History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation

• Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition

• History of malignancy within 5 years prior to the screening visit

• Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening

• Treatment with anti-thymocyte globulin within 180 days prior to screening

• Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening

• Receiving iron supplementation with an unstable dose in the 28 days prior to screening

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention

Drug:
• BCX9930 monotherapy
Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily
• Placebo
Administered orally twice daily

Locations

Country	Name	City	State
Korea, Republic of	InvestigativeSite	Daejeon
Malaysia	Investigative Site	Ampang
South Africa	Investigative Site	Bloemfontein
South Africa	Investigative Site	Cape Town
South Africa	Investigative Site	Pretoria

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

Korea, Republic of,  Malaysia,  South Africa, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in hemoglobin	Change from baseline in hemoglobin	at Week 12
Secondary	Proportion of subjects who are transfusion-free	Proportion of subjects who are transfusion-free	from Week 4 to Week 12
Secondary	Number of units of packed red blood cells transfused	Number of units of packed red blood cells transfused	from Week 4 to Week 12
Secondary	Percent change from baseline in lactate dehydrogenase	Percent change from baseline in lactate dehydrogenase	at Week 12
Secondary	Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale score	Change from baseline in FACIT-Fatigue scale score; FACIT-Fatigue total scale score ranges from 0 to 52, with a higher score indicating less fatigue	at Week 12