INCB000928 for Stone Man Syndrome

(Progress Trial)

Recruiting at 71 trial locations

Overseen By Incyte Corporation Call Center (ex-US)

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Incyte Corporation

Must not be taking: Antibiotics, Antifungals, Antivirals

Disqualifiers: Pregnancy, HIV, HBV, HCV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 12

98 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called INCB000928 to help people with a rare condition called fibrodysplasia ossificans progressiva (FOP). FOP causes soft tissues to turn into bone, and there are few treatments available. The study will check if the drug is safe and effective in stopping or slowing this abnormal bone growth.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you have a chronic or active infectious disease requiring treatment, you may be excluded from participating.

Research Team

Amanda McBride, MD

Principal Investigator

Incyte Corporation

Eligibility Criteria

This trial is for adults and adolescents aged 12 or older with fibrodysplasia ossificans progressiva (FOP), also known as Stone Man Syndrome. Participants must be able to undergo specific imaging procedures without intubation and agree to prevent pregnancy or fathering children. Those with severe FOP, certain infections like HIV/HBV/HCV, a high CAJIS score, or other medical conditions that could affect the study can't join.

Inclusion Criteria

Further inclusion criteria apply

I am 12 or older and have been diagnosed with FOP.

You are willing to prevent pregnancy or becoming a parent based on the conditions described below.

Exclusion Criteria

Pregnant or breast-feeding

Note: Further exclusion criteria apply.

CAJIS score ≥ 24

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-blind Treatment

Participants receive either INCB000928 or placebo for 24 weeks

24 weeks

Visits at Baseline, Week 12, and Week 24

Open-label Extension

Participants who complete the double-blind period receive INCB000928 for an additional 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• INCB000928
• Placebo

Trial Overview The trial is testing INCB000928's effectiveness, safety, and how well it's tolerated in people with FOP compared to a placebo. It's a Phase 2 study where participants are randomly assigned to either receive INCB000928 or a placebo without knowing which one they're getting (double-blind).

Participant Groups

Group I: Cohort 3 Experimental Treatment 2 Interventions

Participants (2 to \< 6 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Group II: Cohort 2 Experimental Treatment 2 Interventions

Participants (6 to \< 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Group III: Cohort 1 Experimental Treatment 2 Interventions

Participants (≥ 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

INCB000928 is already approved in United States for the following indications:

Approved in United States as Zilurgisertib for:

• Fibrodysplasia ossificans progressiva (Orphan Drug Designation)

Who Is Running the Clinical Trial?

Incyte Corporation

Lead Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School