A 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

2 Apr 2024 → 14 Mar 2025

Decision date (initial)

2023-10-09

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

AstriaTherapeutics, Inc.

External identifiers

EU CT number

2022-502953-32-01

WHO UTN

U1111-1286-8704

ClinicalTrials.gov

NCT05695248

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacodynamic, Pharmacogenetic, Pharmacokinetic, Pharmacogenomic, Efficacy, Safety

To assess the safety and tolerability of subcutaneous (SC) administration of single and multiple doses of STAR-0215 in participants with Type I or Type II hereditary angioedema (HAE)

Secondary objectives 4

1. To assess the efficacy of SC administration of single and multiple doses of STAR-0215 in participants with Type I or Type II HAE
2. To characterize the pharmacokinetics (PK) of SC administration of single and multiple doses of STAR-0215 in participants with Type I or Type II HAE
3. To characterize the PD of SC administration of single and multiple doses of STAR-0215 in participants with Type I or Type II HAE
4. To assess the immunogenicity of SC administration of single and multiple doses of STAR-0215 in participants with Type I or Type II HAE

Conditions and MedDRA coding

Hereditary Angioedema (HAE)

Regulatory references

Scientific advice from competent authorities

Paul Ehrlich Institute, Food And Drug Administration

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. Willing and able to read, understand, and sign the IRB/IEC/REC approved informed consent form (ICF).
3. Documented diagnosis of HAE (Type I or II). All of the following must be met: a. Documented clinical history consistent with HAE (e.g. SC or mucosal, nonpruritic swelling episodes without accompanying urticaria); c. Age at reported onset of first angioedema symptoms 30 years of age or younger, or a family history consistent with HAE Type I or II.
4. Experienced at least 2 HAE attacks during the Run-In period, as confirmed by an investigator based on meeting the protocol-specified definition of an HAE attack.

Exclusion criteria 4

1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1inhibitor deficiency, HAE with normal C1-INH (also known as HAE Type III), idiopathic angioedema, or angioedema associated with urticaria.
2. Use of therapies prescribed for the prevention of HAE attacks prior to Screening: a.lanadelumab within 90 days; b.berotralstat within 21 days; c.all other prophylactic therapies, within 7 days.
3. Any exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy [HRT]) within 28 days prior to Screening.
4. Any exposure to androgens (e.g. stanozolol, danazol, oxandrolone, methyltestosterone, testosterone) within 7 days prior to Screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Incidence of adverse events
2. Changes in vital signs, ECG findings, physical examination findings, and clinical laboratory evaluations

Secondary endpoints 8

1. Change from baseline in monthly HAE attack rate
2. Incidence of HAE attack severity (mild, moderate, and severe)
3. Duration of HAE attack (shorter than 12 hours,12 to 24 hours, 24 to 48 hours, and longer than 48 hours)
4. The number of HAE attacks requiring on-demand therapy
5. Time to first HAE attack after first and last dosing
6. Concentration of STAR-0215 and the derived PK parameters
7. Change in plasma kallikrein activity
8. 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 12

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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 66 files

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

Application history

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