A Study of the Efficacy and Safety of Brensocatib in Adults with Moderate to Severe Hidradenitis Suppurativa

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Insmed Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

6 Mar 2025 → 8 Apr 2026

Decision date (initial)

2025-02-27

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Insmed Incorporated

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Pharmacodynamic, Efficacy

To evaluate the effect of brensocatib compared with placebo

Secondary objectives 1

1. Period 1 (Double-Blind Placebo-Controlled): To evaluate the effect of brensocatib compared with placebo; To evaluate the safety and tolerability of brensocatib; To evaluate the systemic exposure of brensocatib. Period 2 (Double-Blind Active Treatment): To assess the long-term safety and tolerability of brensocatib, To evaluate the systemic exposure of brensocatib.

Conditions and MedDRA coding

Hidradenitis Suppurativa

Study design 3 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Age 1. Adult male or female participants ≥18 to ≤80 years of age at the time of signing the informed consent.
2. Type of Participant and Disease Characteristics 2. Participants have a diagnosis of HS (confirmed by a dermatologist) with a history of signs and symptoms consistent with HS for at least 6 months before the Screening Visit. 3. Participants have moderate or severe HS defined as a total of ≥6 inflammatory (inflammatory nodules and/or abscesses) lesions for at least 8 weeks before the Baseline Visit. 4. Participants have HS lesions in at least 2 distinct anatomic areas, 1 of which must be at least Hurley Stage II or Hurley Stage III at both the Screening and Baseline Visits.
3. Sex and Contraceptive/Barrier Requirements 5. Male and female participants must use contraceptives that are consistent with local regulations regarding the methods of contraception for those participating in clinical studies. a. Male Participants: From Day 1 to at least 90 days after the last dose of study drug, male participants, who are not sterile, should refrain from donating fresh unwashed sperm and must be using effective contraception with female partners of childbearing potential. Male participants with pregnant or non-pregnant WOCBP partners must agree to use a male condom and should also be advised of the benefit for female partners to use an additional highly effective contraceptive method as defined in Section 10.4.2. b. Female Participants: Women must be postmenopausal, surgically sterile, or using highly effective contraception methods (as defined in Section 10.4.2) from Day 1 to at least 90 days after the last dose of study drug. The Investigator or designee should explain the acceptable methods of birth control to the participant and instruct the participant to follow the direction.
4. Informed Consent 6. Capable of giving signed informed consent as described in Section 10.1.5 which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.

Exclusion criteria 3

1. Medical Conditions 1. Draining tunnel count of ≥20 at the Baseline Visit. 2. Other active skin disease or condition that could interfere with HS assessments 3. Surgical or laser intervention for an HS lesion during the Screening Period. 4. Known or suspected immunodeficiency disorder, including history of invasive opportunistic infections despite infection resolution, or otherwise recurrent infections of abnormal frequency, or prolonged infections suggesting an immune compromised status, as judged by the Investigator. 5. Known history of HIV infection. 6. Established diagnosis of hepatitis B viral infection at the time of Screening, or positive for HBsAg at the time of Screening. a. Participants who have gained immunity for hepatitis B virus infection after vaccination b. Participants with positive HBcAb are eligible for the study only if hepatitis B virus DNA level is undetectable. 7. Established diagnosis of HCV infection at the time of Screening. Participants positive for hepatitis C antibody are eligible only if HCV RNA is negative. 8. History of malignancy in the past 5 years, except completely treated in situ carcinoma of the cervix and completely treated non-metastatic squamous or basal cell carcinoma of the skin. 9. Have diagnosed periodontal disease and are either: a. Under active management by a dentist for this condition or b. Expected to have periodontal disease-related procedures within the study period. 10. Clinical diagnosis of Papillon-Lefèvre Syndrome. 11. Severe concomitant illness(es) that, in the Investigator's judgment, would adversely affect the participants participation in the study. 12. History of drug or alcohol abuse within 6 months before the Screening Visit.
2. Prior/Concomitant Therapy 13. Received systemic (IV or PO) antibiotic therapy within 8 weeks before the Baseline Visit. a. PO doxycycline or minocycline up to 100 mg PO BID is permitted provided the dosing regimen has been stable for at least 8 weeks before the Baseline Visit and is expected to continue 14. Received PO or transdermal opioid analgesics (except tramadol) for any reason within 4 weeks before the Baseline Visit. 15. Permitted analgesics for HS-related pain have not been at a stable dose regimen for at least 4 weeks before the Baseline Visit. 16. Received prescription topical therapies for the treatment of HS within 2 weeks before the Baseline Visit. 17. Received any anti-TNF-α/other biologics treatment within 12 weeks or 5 elimination half-lives, whichever is longer, before the Baseline Visit. 18. Received systemic nonbiologic therapies with potential therapeutic impact for HS within 4 weeks before the Baseline Visit. 19. Received any immunomodulatory agents within 4 weeks before the Baseline Visit. 20. Received any live attenuated vaccine within 4 weeks before the Screening Visit and during the Screening Period.If a live vaccine has been administered, the participant should wait 4 weeks before Screening.
3. Prior/Concurrent Clinical Study Experience 21. Previously participated in a clinical study for brensocatib. 22. Participated in any other interventional clinical studies with an investigational medicinal product within 3 months or 5 half-lives, whichever is longer, before the Screening Visit. 23. Known history of hypersensitivity to brensocatib or any of its excipients. 24. Participant is the Investigator or any Sub-Investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the study. Diagnostic Assessments For the complete list and details of the principal exclusion criteria, please refer to section 5.2 in the Protocol v1.0 dd 12Jul2024

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percent change from Baseline in total abscess and inflammatory nodule count (AN count) at Week 16

Secondary endpoints 1

1. • Responder status for achieving HiSCR50 and HiSCR75 at Week 16 • Continuous secondary efficacy endpoints will be analyzed in the same fashion as the primary endpoint. For the detailed information on the secondary endpoint, please check the Protocol v1.0,12Jul2024, section 9.6, Secondary Endpoint Analyses

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Insmed Inc.

Sponsor organisation

Insmed Inc.

Address

700 Us Highway 202/206

City

Bridgewater

Postcode

08807-1704

Country

United States

Scientific contact point

Organisation

Insmed Inc.

Contact name

Medical Information

Public contact point

Organisation

Insmed Inc.

Contact name

Medical Information

Third parties 15

Locations

7 EU/EEA countries · 27 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 56 files

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

Application history

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