A Long-term trial to learn about a study drug (STAR-0215) in adults with hereditary angioedema

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Astria Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

20 Jan 2025 → ongoing

Decision date (initial)

2024-11-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2023-506540-16-01

WHO UTN

U1111-1301-0392

ClinicalTrials.gov

NCT06007677

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacodynamic, Efficacy, Pharmacokinetic

To assess long-term safety and tolerability of STAR-0215 in participants with Type 1 or Type 2 hereditary angioedema (HAE)

Secondary objectives 4

1. To assess the long-term efficacy of STAR 0215 in participants with Type 1 or Type 2 HAE
2. To characterize the pharmacokinetics (PK) of long term STAR-0215 dosing in participants with Type 1 or Type 2 HAE
3. To characterize the pharmacodynamics (PD) of long-term STAR-0215 dosing in participants with Type 1 or Type 2 HAE
4. To assess the immunogenicity of STAR-0215 dosed long-term in participants with Type 1 or Type 2 HAE

Conditions and MedDRA coding

Hereditary Angioedema

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. At least 18 years of age at the time of Screening.
2. STAR-0215-201 status was 1 of the following: a. Completed all visits through 6 months after their last dose in STAR-0215-201 b. Eligible for participation in STAR-0215-201 (met all inclusion criteria [with the exception of the criterion for the minimum number of HAE attacks during the Run-In Period] and met none of the exclusion criteria) and entered the Run-In-Period, but did not qualify for the Treatment Period because they did not meet the criterion for the minimum number of HAE attacks. c. Eligible for participation in STAR-0215-201 and entered the Run-In Period but did not complete it for reasons other than not meeting the criterion for the minimum number of HAE attacks – eligibility requires consultation with the Medical Monitor; or d. Discontinued participation in STAR-0215-201 (for personal or social reasons) after having completed at least 84 days of trial follow-up since their last dose of STAR-0215. Eligibility requires consultation with the Medical Monitor. Participants who discontinued for safety reasons are not eligible.
3. For participants who are STAR-0215-naïve and were not enrolled in STAR-0215-201: Documented diagnosis of HAE (Type 1 or Type 2). All of the following must be met: a. Documented clinical history consistent with HAE (e.g. subcutaneous [SC] or mucosal, nonpruritic swelling episodes without accompanying urticaria). b. Age at reported onset of first angioedema symptoms 30 years of age or younger, or a family history consistent with HAE Type 1 or Type 2. c. C1-esterase inhibitor protein (C1-INH) antigen or functional level less than 40% of the normal level. Participants with antigen or functional C1-INH level 40% to 50% of the normal level may be eligible if they also have a C4 level below the normal range and a family history consistent with HAE Type 1 or Type 2. Testing for C1-INH and C4 will be performed at Screening. Historical test results for C1-INH may be used to confirm eligibility if the screening C1-INH antigen test or functional level or C4 level laboratory values are inconclusive, after discussion with the Medical Monitor. If the C1-INH functional level by enzyme immunoassay (EIA) does not qualify a participant with known or clinically suspected Type 2 HAE for study eligibility, a chromogenic assay may be obtained after discussion with the Sponsor's Medical Monitor. Functional level via chromogenic assay is inclusionary if the C1-INH functional level is < 74%. In this situation, the chromogenic assay should be ordered by the site per their usual process for ordering the test.
4. For participants who are STAR-0215-naïve and were not enrolled in STAR-0215-201: At least 2 HAE attacks during the Run-In Period, as confirmed by an investigator based on protocol-specified definition of an HAE attack.

Exclusion criteria 4

1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1 INH (also known as HAE Type 3), idiopathic angioedema, or angioedema associated with urticaria
2. Any exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy) within 28 days prior to Screening. (Note: These medications are prohibited in STAR-0215-201)
3. Any exposure to androgens (e.g. stanozolol, danazol, oxandrolone, methyltestosterone, testosterone) within 7 days prior to Screening. Note: These medications are prohibited in STAR-0215-201
4. Use of therapies prescribed for the prevention of HAE attacks (with the exception of STAR-0215 for participants who received STAR-0215 in STAR-0215-201) prior to Screening: a. lanadelumab within 90 days b. berotralstat within 21 days c. all other prophylactic therapies, discuss with the Medical Monitor Note: These medications are prohibited in STAR-0215-201.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of adverse events (AEs)

Secondary endpoints 10

1. Change from baseline in monthly HAE attack rate
2. Incidence of monthly HAE attack severity (mild, moderate, and severe)
3. Duration of monthly HAE attack (shorter than 12 hours, 12 to 24 hours, 24 to 48 hours, and longer than 48 hours)
4. The number of monthly HAE attacks requiring on-demand therapy
5. Time to first HAE attack after each dose
6. Number of HAE attack-free days
7. Proportion of HAE attack-free participants (i.e., participants having 0 HAE attacks during the specified treatment period)
8. Concentration of STAR-0215 and the derived PK parameters
9. Change in plasma kallikrein activity
10. Formation of anti-drug (STAR-0215) antibodies

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Astria Therapeutics Inc.

Sponsor organisation

Astria Therapeutics Inc.

Address

22 Boston Wharf Road, Floor 10 Floor 10

City

Boston

Postcode

02210

Country

United States

Scientific contact point

Organisation

Astria Therapeutics Inc.

Contact name

Astria Therapeutics Inc

Public contact point

Organisation

Astria Therapeutics Inc.

Contact name

Astria Therapeutics Inc

Third parties 5

Locations

4 EU/EEA countries · 6 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 40 files

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

Application history

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