KD025 in Subjects With Diffuse Cutaneous Systemic Sclerosis
Summary
This randomized, placebo-controlled phase 2 study was seeking to evaluate the efficacy and safety of belumosudil (KD025) for the treatment of diffuse cutaneous systematic sclerosis. Enrolment was terminated earlier than planned for business reasons unrelated to safety. A total of 36 participants were enrolled and randomized into 3 groups to either receive orally administered belumosudil (200 milligrams \[mg\] once daily \[QD\] and 200 mg twice daily \[BID\]) or matched placebo in 1:1:1 ratio in the double-blind (DB) period of this study. Study drug dosing was for 52 weeks: double-blinded for the first 28 weeks followed by an open-label extension of 24 weeks. After unblinding, the participants on belumosudil continued on the same belumosudil dose whereas the participants in the placebo group were re-randomized to one of the belumosudil doses in a 1:1 ratio.
Timeline
- Start
- 2019-06-26
- Primary completion
- 2022-08-09
- Completion
- 2023-02-17
Outcome
Missed primary endpoint
Stopped (Enrollment): “Sponsor decision due to slow enrollment and strategic consideration; not driven by any safety concerns.”
registry analysis (superiority test); DB Period: Belumosudil QD vs DB Period: Placebo; p = 0.9472; NCT03919799 ↗
registry analysis (superiority test); DB Period: Belumosudil BID vs DB Period: Placebo; p = 0.3078; NCT03919799 ↗
paper The primary end point, of CRISS score ≥0.60 at week 24, did not exhibit an efficacy signal in the belumosudil vs placebo groups PMID 40088930 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Belumosudil | Small molecule | 200 mg | Oral |