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Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BCX9930
Sponsored by
BioCryst Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria 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 non-pregnant, non-lactating female subjects Are receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, have benefited from treatment with BCX9930 and would benefit from continued treatment with BCX9930, and who do not have access to other treatment options Exclusion Criteria: Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the subject to continue study-specific procedures or it is considered not to be in the subject's best interest to continue or benefit-risk assessment is no longer in favor of the subject's continued treatment

Sites / Locations

  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative SiteRecruiting
  • Investigative Site
  • Investigative Site
  • Investigative Site
  • Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BCX9930

Arm Description

All subjects receive BCX9930 for 96 weeks

Outcomes

Primary Outcome Measures

Proportion of subjects with treatment emergent adverse events (TEAEs)
Proportion of subjects with TEAEs. Safety assessments include graded TEAEs / serious TEAEs, lab abnormalities, changes to vital signs, and physical examination findings.

Secondary Outcome Measures

Full Information

First Posted
February 14, 2023
Last Updated
October 3, 2023
Sponsor
BioCryst Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05741346
Brief Title
Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Official Title
An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst-sponsored Study
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 18, 2023 (Actual)
Primary Completion Date
February 2026 (Anticipated)
Study Completion Date
February 2026 (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
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This study is designed to provide continued access to BCX9930 for subjects currently receiving treatment with BCX9930 in a BioCryst-sponsored clinical study for paroxysmal nocturnal hemoglobinuria (PNH) who, in the opinion of the investigator, would benefit from continued treatment with BCX9930 and who do not have access to other treatment options.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
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
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
BCX9930
Arm Type
Experimental
Arm Description
All subjects receive BCX9930 for 96 weeks
Intervention Type
Drug
Intervention Name(s)
BCX9930
Intervention Description
Taken orally at 400 mg twice daily (BID)
Primary Outcome Measure Information:
Title
Proportion of subjects with treatment emergent adverse events (TEAEs)
Description
Proportion of subjects with TEAEs. Safety assessments include graded TEAEs / serious TEAEs, lab abnormalities, changes to vital signs, and physical examination findings.
Time Frame
Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or non-pregnant, non-lactating female subjects Are receiving treatment with BCX9930 in another clinical study of PNH and, in the opinion of the investigator, have benefited from treatment with BCX9930 and would benefit from continued treatment with BCX9930, and who do not have access to other treatment options Exclusion Criteria: Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the subject to continue study-specific procedures or it is considered not to be in the subject's best interest to continue or benefit-risk assessment is no longer in favor of the subject's continued treatment
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
BioCryst Pharmaceuticals, Inc.
Phone
+1 (844) 273-2328
Email
BCX9930clinicaltrials@biocryst.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Phillip Scheinberg, MD, PhD
Organizational Affiliation
Beneficência Portuguesa de São Paulo
Official's Role
Principal Investigator
Facility Information:
Facility Name
Investigative Site
City
Paris
Country
France
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Budapest
Country
Hungary
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Daejeon
Country
Korea, Republic of
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Ampang
Country
Malaysia
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Bloemfontein
Country
South Africa
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Cape Town
Country
South Africa
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Pretoria
Country
South Africa
Individual Site Status
Recruiting
Facility Name
Investigative Site
City
Barcelona
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Valencia
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
Leeds
Country
United Kingdom
Individual Site Status
Active, not recruiting
Facility Name
Investigative Site
City
London
Country
United Kingdom
Individual Site Status
Active, not recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria

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