What side effects are associated with this type of intervention?

Common treatments for Fibrodysplasia Ossificans Progressiva (FOP) include corticosteroids, nonsteroidal anti-inflammatory drugs (NSAIDs), and bisphosphonates. Corticosteroids can cause side effects such as weight gain, high blood pressure, and increased risk of infections. NSAIDs may lead to gastrointestinal issues, including ulcers and bleeding. Bisphosphonates can cause gastrointestinal discomfort, osteonecrosis of the jaw, and atypical femoral fractures. These treatments aim to manage symptoms and inflammation but do not cure the disease.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Fibrodysplasia Ossificans Progressiva (FOP), also known as Stone Man Syndrome. The condition is extremely rare, and treatment options are limited to managing symptoms and preventing flare-ups. Clinical trials, such as the one studying INCB000928, are ongoing to find effective therapies. Broadly, treatments under investigation include those targeting the ACVR1 gene mutation responsible for FOP, as well as anti-inflammatory and immunosuppressive drugs to manage symptoms.

What other types of research exist?

Promising alternatives to INCB000928 for treating Stone Man Syndrome (FOP) include targeting the ACVR1/ALK2 pathway, which is implicated in FOP. Drugs like palovarotene, a retinoic acid receptor gamma agonist, have shown potential in clinical trials by reducing heterotopic ossification. Additionally, monoclonal antibodies or small molecules that inhibit the aberrant signaling of the ACVR1/ALK2 receptor could be considered. Gene therapy approaches targeting the underlying genetic mutations are also being explored as potential treatments.

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INCB000928 for Stone Man Syndrome (Progress Trial)

Phase 2

Recruiting

Research Sponsored by Incyte Corporation

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Female and male adults and adolescents ≥ 12 years of age with a diagnosis of FOP

Willing and able to undergo low-dose WBCT (excluding the head) imaging without requiring intubation

Must not have

Chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment

HIV, HBV, or HCV infection

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 12 and week 24

Awards & highlights

Summary

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.

Who is the study for?

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.

What is being tested?

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).

What are the potential side effects?

While specific side effects of INCB000928 aren't listed here, common ones in trials may include reactions at the injection site, fatigue, headache, nausea. Since this medication targets FOP symptoms directly related to muscle tissue ossification; there might be unique risks involved.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I can have a full-body CT scan without needing a breathing tube.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently on treatment for a long-term or active infection.

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I am infected with HIV, HBV, or HCV.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 12 and week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 12 and week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 12 and week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Double Blind Period: Total volume of new heterotopic ossification (HO)

Secondary study objectives

Double-blind Period: Proportion of participants with a clinically meaningful improvement in the flare-related symptoms

Double-blind Period: Total number of new flares

Number of Participants with Treatment Emergent Adverse Events (TEAE)

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Group A: INCB000928Experimental Treatment2 Interventions

Participants will receive INCB000928 for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 52 weeks.

Group II: Group B: Placebo followed by INCB000928Placebo Group1 Intervention

Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive INCB000928 in the 52 week open-label extension period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

INCB000928

2021

Completed Phase 2

~70

placebo

2010

Completed Phase 4

~6580

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Stone Man Syndrome, or Fibrodysplasia Ossificans Progressiva (FOP), involves abnormal bone formation in muscles and connective tissues. Treatments like INCB000928 likely target the ACVR1 gene mutation and inhibit the BMP signaling pathway to prevent this abnormal bone growth. These mechanisms are vital for FOP patients as they help to slow disease progression, maintain mobility, and enhance quality of life.

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