A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis
Summary
This is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial. Participants will be screened within 6 weeks prior to the Baseline (Day 1) Visit. Approximately 300 participants who meet the trial eligibility criteria will be randomized on Day 1 in a 1:1:1 ratio to receive HZN-825 300 mg QD, HZN-825 300 mg BID or placebo for 52 weeks. The trial will include up to a 42-day Screening Period and a 52-week Double-blind Treatment Period. Participants will take their first dose of trial drug at the clinic and will participate in trial visits at Week 4 and every 6 weeks thereafter until Week 52. All participants who complete the Double-blind Treatment Period (Week 52) will be eligible to enter a 52-week extension trial (HZNP-HZN-825-302, NCT05626751). Participants not entering the extension trial will participate in a Safety Follow-up Visit 4 weeks after the last dose of trial drug.
Timeline
- Start
- 2021-11-05
- Primary completion
- 2025-02-24
- Completion
- 2025-02-24
Outcome
Missed primary endpoint
Stopped: “The study was terminated due to meeting pre-defined criteria for futility.”
registry analysis (superiority test); Fipaxalparant 300 mg QD vs Placebo; p = 1.000 NCT04781543 ↗
registry analysis (superiority test); Fipaxalparant 300 mg BID vs Placebo; p = 0.525 NCT04781543 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | FIPAXALPARANT | Small molecule | 300 mg | Oral |