Clinical trial NCT05741346: Long-term Safety of BCX9930 in Participants 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: BCX9930
Factor D inhibitor
Proximal complement inhibitor
Oral therapy
Paroxysmal nocturnal hemoglobinuria
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2023-02-23
Enrollment: 28
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is designed to provide continued access to BCX9930 for participants 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.


Eligibility criteria: Inclusion Criteria:

* Male or non-pregnant, non-lactating female participants
* 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 participant's ability to participate in the study or increase the risk of participation for that participant
* An ongoing adverse event, including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the participant to continue study-specific procedures or it is considered not to be in the participant's best interest to continue or benefit-risk assessment is no longer in favor of the participant's continued treatment

Primary outcomes:
measure: Proportion of Participants with Treatment Emergent Adverse Events (TEAEs)
timeFrame: Week 96
Secondary outcomes:
Other outcomes:
Locations:
city: Paris
country: France
facility: Investigative Site
city: Budapest
country: Hungary
facility: Investigative Site
city: Daejeon
country: Korea, Republic of
facility: Investigative Site
city: Ampang
country: Malaysia
facility: Investigative Site
city: Bloemfontein
country: South Africa
facility: Investigative Site
city: Cape Town
country: South Africa
facility: Investigative Site
city: Pretoria
country: South Africa
facility: Investigative Site
city: Barcelona
country: Spain
facility: Investigative Site
city: Valencia
country: Spain
facility: Investigative Site
city: Leeds
country: United Kingdom
facility: Investigative Site
city: London
country: United Kingdom
facility: Investigative Site
Study officials:
affiliation: Beneficência Portuguesa de São Paulo
name: Phillip Scheinberg, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: