BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

— REDEEM-1  

Official title:

A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05116774
• Other study ID #: BCX9930-202
• Secondary ID: 2020-004438-39
• Status: Terminated
• Phase: Phase 2
• Start date: December 6, 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 PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor.

Description:

This is a randomized, active comparator-controlled, open-label, 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 oral BCX9930 monotherapy for 24 weeks versus continuing C5 inhibitor therapy in subjects with PNH with inadequate response to their current C5 inhibitor therapy. Subjects will be randomized to either discontinue C5 inhibitor therapy and start BCX9930 monotherapy or to continue C5 inhibitor therapy for the 24-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 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 C5 inhibitor therapy in Part 1 will discontinue that therapy at the Week 24 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

• Currently being treated with a stable C5 inhibitor regimen

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

• At screening: PNH clone size of = 10% and hemoglobin = 10.5 g/dL

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
Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily
• Eculizumab
Administered by intravenous infusion per current dose regimen
• Ravulizumab
Administered as intravenous infusion per current dose regimen

Locations

Country	Name	City	State
France	Investigative Site	Paris
Hungary	Investigative Site	Budapest
Italy	Investigative Site	Rome
Spain	Investigative Site	Barcelona
Spain	Investigative Site	Valencia
United Kingdom	Investigative Site	Leeds
United Kingdom	Investigative Site	London

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

France,  Hungary,  Italy,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in hemoglobin	Change from baseline in hemoglobin	mean of values at Weeks 12, 16, 20, and 24
Secondary	Proportion of subjects who are transfusion-free	Proportion of subjects who are transfusion-free	from Week 4 to Week 24
Secondary	Number of units of packed red blood cells transfused	Number of units of packed red blood cells transfused	from Week 4 to Week 24
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	mean of values at Weeks 12, 16, 20, and 24