Long term access to berotralstat for HAE subjects from previous clinical trials.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biocryst Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

31 Aug 2021 → ongoing

Decision date (initial)

2024-07-17

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

BioCryst Pharmaceuticals Inc

External identifiers

EU CT number

2024-511285-37-00

EudraCT number

2020-004230-37

ClinicalTrials.gov

NCT04933721

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Prophylaxis, Safety

To monitor the long-term safety of berotralstat

Secondary objectives 2

1. To provide continued access to berotralstat to subjects who have participated in Studies BCX7353-302 (Study 302) and BCX7353-204 (Study 204), are expected to continue benefiting from treatment with berotralstat, and do not have other means of access to berotralstat
2. To provide continued access to berotralstat to patients who have participated in BCX7353-304 (Study 304) and are expected to continue to benefit from treatment with berotralstat

Conditions and MedDRA coding

Hereditary Angioedema

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002449-PIP02-18

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Males and non-pregnant, non-lactating females who are currently enrolled as a subject or were previously enrolled and did not discontinue due to an adverse event or due to noncompliance in BioCryst-sponsored Study 302, 204, or 304.
2. Participant or parent/ legally designated representative (for participants < 18 years of age) able to provide written informed consent. For participants < 18 years of age, based on the child’s age and local regulatory requirements, participant assent will be collected as appropriate.
3. Would benefit from continued berotralstat treatment in the opinion of the investigator
4. Female subjects must meet at least 1 of the following requirements: a. Be a WOCBP (defined as a female following menarche and prior to becoming post-menopausal who has not had a hysterectomy or bilateral salpingectomy and bilateral oophorectomy) who agrees to use at least an acceptable effective contraceptive method during the study. b. Be a woman of nonchildbearing potential (defined as postmenopausal [no menses for at least 12 months without an alternative medical cause] or has had a hysterectomy or a bilateral salpingectomy and bilateral oophorectomy).
5. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures

Exclusion criteria 8

1. Any condition or situation, including medical history or changes in medical history, which, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study
2. Dementia, altered mental status, or any psychiatric condition that would prohibit the understanding or rendering of informed consent.
3. Hypersensitivity to the active substance or to any of the excipients in the IMP
4. Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study. These include: C1-INH, tranexamic acid, androgens, or lanadelumab
5. Use of any other investigational medicinal product at the Baseline visit or any time during the study.
6. Pregnancy or breastfeeding
7. Subjects with an immediate family relationship to either sponsor employees, the investigator, or employees of the study site who are named on the delegation log.
8. Subjects who are held in an institution by a governmental or judicial order.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

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

Secondary endpoints 1

1. Another mechanism is available to provide drug to the subject(eg, market access), or until the sponsor discontinues all global commercialization and development of berotralstat for the prevention of angioedema attacks, whichever comes first

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biocryst Pharmaceuticals Inc.

Sponsor organisation

Biocryst Pharmaceuticals Inc.

Address

4505 Emperor Boulevard Suite 200

City

Durham

Postcode

27703-8457

Country

United States

Scientific contact point

Organisation

Biocryst Pharmaceuticals Inc.

Contact name

Biocryst Pharmaceuticals Inc.

Public contact point

Organisation

Biocryst Pharmaceuticals Inc.

Contact name

Biocryst Pharmaceuticals Inc.

Third parties 2

Locations

7 EU/EEA countries · 9 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 43 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

7 submissions — initial application plus substantial / non-substantial modifications