We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 5 for:    BCX9930

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05116787
Recruitment Status : Recruiting
First Posted : November 11, 2021
Last Update Posted : November 4, 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 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.

Layout table for study information
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 : March 2024
Estimated Study Completion Date : December 2024


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 200 mg twice daily for the first 2 weeks, then 400 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




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 Week 4 to Week 12 ]
    Proportion of subjects who are transfusion-free

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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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
  • 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

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): NCT05116787


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

Locations
Layout table for location information
United States, Massachusetts
Investigative Site Not yet recruiting
Boston, Massachusetts, United States, 02114
Malaysia
Investigative Site Recruiting
Ampang, Malaysia
South Africa
Investigative Site Recruiting
Bloemfontein, South Africa, 9301
Investigative Site Active, not recruiting
Cape Town, South Africa
Investigative Site Recruiting
Pretoria, South Africa, 0044
Spain
Investigative Site Not yet recruiting
Barcelona, Spain
Taiwan
Investigative Site Not yet recruiting
Taipei, Taiwan
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
Layout table for investigator information
Principal Investigator: David J Kuter, MD, DPhil Massachusetts General Hospital
Layout table for additonal 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    Key Record Dates
Last Update Posted: November 4, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases