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Trial record 4 of 5 for:    BCX9930

BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy (REDEEM-1)

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ClinicalTrials.gov Identifier: NCT05116774
Recruitment Status : Recruiting
First Posted : November 11, 2021
Last Update Posted : November 7, 2022
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Brief 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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: BCX9930 Drug: Eculizumab Drug: Ravulizumab Phase 2

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 81 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : December 6, 2021
Estimated Primary Completion Date : August 2024
Estimated Study Completion Date : February 2025


Arm Intervention/treatment
Experimental: BCX9930 monotherapy

In Part 1, participants are randomized 2:1 to receive BCX9930 monotherapy or continue with current C5 inhibitor

In Part 2, all subjects receive BCX9930 monotherapy

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

Active Comparator: Continued C5 inhibitor therapy
In Part 1, participants are randomized 2:1 to receive BCX9930 monotherapy or continue with current C5 inhibitor
Drug: Eculizumab
Administered by intravenous infusion per current dose regimen
Other Name: Soliris

Drug: Ravulizumab
Administered as intravenous infusion per current dose regimen
Other Name: Ultomiris, ALXN1210, ravulizumab-cwvz




Primary Outcome Measures :
  1. Change from baseline in hemoglobin [ Time Frame: mean of values at Weeks 12, 16, 20, and 24 ]
    Change from baseline in hemoglobin


Secondary Outcome Measures :
  1. Proportion of subjects who are transfusion-free [ Time Frame: from Week 4 to Week 24 ]
    Proportion of subjects who are transfusion-free

  2. Number of units of packed red blood cells transfused [ Time Frame: from Week 4 to Week 24 ]
    Number of units of packed red blood cells transfused

  3. Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale score [ Time Frame: mean of values at Weeks 12, 16, 20, and 24 ]
    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



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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
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05116774


Contacts
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Contact: BioCryst Pharmaceuticals, Inc. +1 (844) 273-2328 BCX9930clinicaltrials@biocryst.com

Locations
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United States, Massachusetts
Investigative Site Not yet recruiting
Boston, Massachusetts, United States, 02114
France
Investigative Site Recruiting
Nice, France
Investigative Site Recruiting
Paris, France
Investigative Site Recruiting
Quimper, France
Hungary
Investigative Site Recruiting
Budapest, Hungary
Italy
Investigative Site Recruiting
Rome, Italy
Spain
Investigative Site Recruiting
Barcelona, Spain
Investigative Site Recruiting
Valencia, Spain
Taiwan
Investigative Site Not yet recruiting
Taipei, Taiwan
United Kingdom
Investigative Site Recruiting
Leeds, United Kingdom
Investigative Site Recruiting
London, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Austin G Kulasekararaj, MBBS, MD King's College Hospital NHS Trust
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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05116774    
Other Study ID Numbers: BCX9930-202
2020-004438-39 ( EudraCT Number )
First Posted: November 11, 2021    Key Record Dates
Last Update Posted: November 7, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by BioCryst Pharmaceuticals:
BCX9930
factor D inhibitor
proximal complement inhibitor
oral therapy
paroxysmal nocturnal hemoglobinuria
inadequate response to C5 inhibitor
C5 inhibitor
eculizumab
ravulizumab
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases
Eculizumab
Ravulizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs