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

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status

Active

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

BCX9930 monotherapy

Placebo

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH) focused on measuring BCX9930, factor D inhibitor, proximal complement inhibitor, oral therapy, paroxysmal nocturnal hemoglobinuria

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Investigative Site
Investigative Site
Investigative Site
Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

BCX9930 monotherapy

Placebo

Arm Description

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

In Part 1, participants are randomized to 2:1 to receive BCX9930 monotherapy or placebo under double-blind conditions

Outcomes

Primary Outcome Measures

Change from baseline in hemoglobin

Secondary Outcome Measures

Proportion of subjects who are transfusion-free

Number of units of packed red blood cells transfused

Percent change from baseline in lactate dehydrogenase

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

Full Information

NCT ID

NCT05116787

First Posted

November 2, 2021

Last Updated

December 15, 2022

Sponsor

BioCryst Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT05116787

Brief Title

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

Acronym

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

Study Type

Interventional

2. Study Status

Record Verification Date

December 2022

Overall Recruitment Status

Active, not recruiting

Study Start Date

October 26, 2021 (Actual)

Primary Completion Date

March 2024 (Anticipated)

Study Completion Date

December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

BioCryst Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

57 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

BCX9930 monotherapy

Arm Type

Experimental

Arm Description

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

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

In Part 1, participants are randomized to 2:1 to receive BCX9930 monotherapy or placebo under double-blind conditions

Intervention Type

Drug

Intervention Name(s)

BCX9930 monotherapy

Intervention Description

Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Administered orally twice daily

Primary Outcome Measure Information:

Title

Change from baseline in hemoglobin

Description

Change from baseline in hemoglobin

Time Frame

at Week 12

Secondary Outcome Measure Information:

Title

Proportion of subjects who are transfusion-free

Description

Proportion of subjects who are transfusion-free

Time Frame

from Week 4 to Week 12

Title

Number of units of packed red blood cells transfused

Description

Number of units of packed red blood cells transfused

Time Frame

from Week 4 to Week 12

Title

Percent change from baseline in lactate dehydrogenase

Description

Percent change from baseline in lactate dehydrogenase

Time Frame

at Week 12

Title

Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale score

Description

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

Time Frame

at Week 12

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David J Kuter, MD, DPhil

Organizational Affiliation

Massachusetts General Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Investigative Site

City

Ampang

Country

Malaysia

Facility Name

Investigative Site

City

Bloemfontein

ZIP/Postal Code

9301

Country

South Africa

Facility Name

Investigative Site

City

Cape Town

Country

South Africa

Facility Name

Investigative Site

City

Pretoria

ZIP/Postal Code

0044

Country

South Africa

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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