BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy (REDEEM-2)

• ClinicalTrials.gov Identifier: NCT05116787
• Recruitment Status: Active, not recruiting
• First Posted: November 11, 2021
• Last Update Posted: May 6, 2022
• Sponsor: BioCryst Pharmaceuticals
• Information provided by (Responsible Party): BioCryst Pharmaceuticals

Study Description

Brief 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.

Condition or disease	Intervention/treatment	Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH)	Drug: BCX9930 monotherapy; Drug: Placebo	Phase 2

Detailed 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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 57 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: October 26, 2021
• Estimated Primary Completion Date: November 2022
• Estimated Study Completion Date: August 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: BCX9930 monotherapy; In Part 1, participants are randomized to 2:1 to receive BCX9930 monotherapy or placebo under double-blind conditions In Part 2, all participants receive open-label BCX9930 monotherapy	Drug: BCX9930 monotherapy; Administered orally at a dose of 500 mg twice daily
Placebo Comparator: Placebo; In Part 1, participants are randomized to 2:1 to receive BCX9930 monotherapy or placebo under double-blind conditions	Drug: Placebo; Administered orally twice daily

Outcome Measures

Primary Outcome Measures :

1. Change from baseline in hemoglobin [ Time Frame: at Week 12 ]
   Change from baseline in hemoglobin

Secondary Outcome Measures :

1. Proportion of subjects who are transfusion-free [ Time Frame: from Day 14 to Week 12 ]
   Proportion of subjects who are transfusion-free
2. Number of units of packed red blood cells transfused [ Time Frame: from Day 14 to Week 12 ]
   Number of units of packed red blood cells transfused
3. Percent change from baseline in lactate dehydrogenase [ Time Frame: at Week 12 ]
   Percent change from baseline in lactate dehydrogenase
4. Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale score [ Time Frame: at Week 12 ]
   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

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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
• 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

Contacts and Locations

Locations

United States, Massachusetts

Investigative Site

Boston, Massachusetts, United States, 02114

Malaysia

Investigative Site

Ampang, Malaysia

South Africa

Investigative Site

Bloemfontein, South Africa, 9301

Investigative Site

Cape Town, South Africa

Investigative Site

Pretoria, South Africa, 0044

Spain

Investigative Site

Barcelona, Spain

Taiwan

Investigative Site

Taipei, Taiwan

Sponsors and Collaborators

BioCryst Pharmaceuticals

Investigators

• Principal Investigator: David J Kuter, MD, DPhil; Massachusetts General Hospital

More Information

• Responsible Party: BioCryst Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT05116787
• Other Study ID Numbers: BCX9930-203; 2020-004403-14 ( EudraCT Number )
• First Posted: November 11, 2021
• Last Update Posted: May 6, 2022
• Last Verified: January 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by BioCryst Pharmaceuticals:

BCX9930; factor D inhibitor; proximal complement inhibitor; oral therapy; paroxysmal nocturnal hemoglobinuria

Additional relevant MeSH terms:

Hemoglobinuria; Hemoglobinuria, Paroxysmal; Proteinuria; Urination Disorders; Urologic Diseases; Urological Manifestations; Anemia, Hemolytic; Anemia; Hematologic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases