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Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies (APeX-A)

Primary Purpose

Hereditary Angioedema, HAE

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
berotralstat
Sponsored by
BioCryst Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Hereditary Angioedema focused on measuring prophylaxis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Males and females currently enrolled in BioCryst-sponsored Study 302 or 204
  • Able to provide written informed consent.
  • Would benefit from continued berotralstat treatment
  • Acceptable effective contraception

Key Exclusion Criteria:

  • Pregnancy or breast-feeding
  • Any condition or situation, including medical history that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study
  • Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study.
  • Use of any other investigational medicinal product at the Baseline visit or any time during the study.

Sites / Locations

  • Study Center
  • Study Center
  • Study Center
  • Study Center
  • Study Center
  • Study Center
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  • Study Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BCX7353 Capsules 150 mg once daily

Arm Description

Berotralstat (BCX7353) 150 mg capsule orally administered once daily.

Outcomes

Primary Outcome Measures

Number and proportion of subjects with a treatment-related TEAE
Number and proportion of subjects who experience a serious adverse event (SAE)
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
Number and proportion of subjects who discontinue due to a TEAE

Secondary Outcome Measures

Full Information

First Posted
June 14, 2021
Last Updated
May 17, 2023
Sponsor
BioCryst Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04933721
Brief Title
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
Acronym
APeX-A
Official Title
An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 23, 2021 (Actual)
Primary Completion Date
August 2026 (Anticipated)
Study Completion Date
August 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
No

5. Study Description

Brief Summary
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
Detailed Description
APeX-A, 7353-312, is a single-arm, open-label, multicenter study. The study will be conducted in countries where berotralstat is not available either commercially or via another mechanism. All enrolled subjects will receive the planned marketed dose of berotralstat, 150 mg QD. The study will assess the long term safety and tolerability of berotralstat. Eligible subjects will be enrolled directly into Study BCX7353-312 (Study 312) from Studies 302 and 204. Subjects will receive berotralstat 150 mg administered orally once daily (QD). Subjects will return to the clinic every 24 weeks for drug dispensation and safety monitoring for up to 240 weeks (approximately 5 years). Safety and tolerability will be evaluated through assessment of serious adverse events (SAEs) and treatment-related, treatment-emergent adverse events (TEAEs). Up to 110 subjects are planned to enroll.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema, HAE
Keywords
prophylaxis

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open-label berotralstat (BCX7353) 150 mg orally administered QD.
Masking
None (Open Label)
Masking Description
No masking as this is open-label.
Allocation
N/A
Enrollment
110 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
BCX7353 Capsules 150 mg once daily
Arm Type
Experimental
Arm Description
Berotralstat (BCX7353) 150 mg capsule orally administered once daily.
Intervention Type
Drug
Intervention Name(s)
berotralstat
Other Intervention Name(s)
Orladeyo
Intervention Description
BCX7353 capsules administered orally once daily
Primary Outcome Measure Information:
Title
Number and proportion of subjects with a treatment-related TEAE
Time Frame
240 weeks
Title
Number and proportion of subjects who experience a serious adverse event (SAE)
Time Frame
240 weeks
Title
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
Time Frame
240 weeks
Title
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
Time Frame
240 weeks
Title
Number and proportion of subjects who discontinue due to a TEAE
Time Frame
240 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Males and females currently enrolled in BioCryst-sponsored Study 302 or 204 Able to provide written informed consent. Would benefit from continued berotralstat treatment Acceptable effective contraception Key Exclusion Criteria: Pregnancy or breast-feeding Any condition or situation, including medical history that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study. Use of any other investigational medicinal product at the Baseline visit or any time during the study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Vesna Grivcheva-Panoska, MD, PhD
Organizational Affiliation
PHI University Clinic of Dermatology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Study Center
City
Brno
Country
Czechia
Facility Name
Study Center
City
Plzen
Country
Czechia
Facility Name
Study Center
City
Grenoble
Country
France
Facility Name
Study Center
City
Paris
Country
France
Facility Name
Study Center
City
Daegu
Country
Korea, Republic of
Facility Name
Study Center
City
Gwangju
Country
Korea, Republic of
Facility Name
Study Center
City
Seoul
Country
Korea, Republic of
Facility Name
Study Center
City
Skopje
Country
North Macedonia
Facility Name
Study Center
City
Kraków
Country
Poland
Facility Name
Study Center
City
Martin
Country
Slovakia
Facility Name
Study Center
City
Cape Town
Country
South Africa

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

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