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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05741346
• Other study ID #: BCX9930-205
• Secondary ID: 2021-006776-17
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: January 18, 2023
• Est. completion date: February 2026

Study information

• Verified date: July 2023
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 28
• Est. completion date: February 2026
• Est. primary completion date: February 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• BCX9930
Taken orally at 400 mg twice daily (BID)

Locations

Country	Name	City	State
France	Investigative Site	Paris
Hungary	Investigative Site	Budapest
Korea, Republic of	Investigative Site	Daejeon
Malaysia	Investigative Site	Ampang
South Africa	Investigative Site	Bloemfontein
South Africa	Investigative Site	Cape Town
South Africa	Investigative Site	Pretoria
Spain	Investigative Site	Barcelona
Spain	Investigative Site	Valencia
United Kingdom	Investigative Site	Leeds
United Kingdom	Investigative Site	London

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

France,  Hungary,  Korea, Republic of,  Malaysia,  South Africa,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Week 96