What is the mechanism of action of this treatment?

Fibrodysplasia Ossificans Progressiva (FOP) is characterized by the abnormal formation of bone in soft tissues, leading to severe disability. Treatments like IPN60130 aim to inhibit the process of Heterotopic Ossification (HO), which is the formation of bone outside the normal skeleton. These treatments work by targeting specific pathways involved in bone formation, potentially reducing the volume of new bone growth in soft tissues. This is crucial for FOP patients as it can help manage flare-ups, reduce pain, and preserve mobility, thereby improving their quality of life.

What side effects are associated with this type of intervention?

Treatments for Fibrodysplasia Ossificans Progressiva (FOP) like IPN60130, which aim to inhibit Heterotopic Ossification (HO), often involve targeting specific pathways to prevent abnormal bone formation. Common side effects for these types of treatments can include gastrointestinal issues, such as nausea and diarrhea, as well as potential impacts on liver function and blood cell counts. Additionally, there may be risks of infections due to immunosuppressive effects. It is crucial for patients to discuss these potential side effects with their healthcare provider to manage and monitor any adverse reactions effectively.

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 severely disabling, characterized by the formation of bone in soft tissues. Current research, including the study of IPN60130, is focused on inhibiting new Heterotopic Ossification (HO) to manage the disease. Other treatments under investigation include various anti-inflammatory drugs and potential gene therapies, but none have received FDA approval yet.

What other types of research exist?

Promising alternatives to IPN60130 for treating Stone Man Syndrome (FOP) include therapies targeting the ACVR1/ALK2 pathway, which is implicated in FOP. Potential treatments could involve small molecule inhibitors, monoclonal antibodies, or gene therapies designed to correct or mitigate the effects of the ACVR1 mutation. Additionally, repurposing drugs that have shown efficacy in reducing heterotopic ossification in other conditions, such as bisphosphonates or anti-inflammatory medications, may also be considered.

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Oral Fidrisertib for Stone Man Syndrome (FALKON Trial)

Phase 2

Waitlist Available

Research Sponsored by Clementia Pharmaceuticals Inc.

Eligibility Criteria Checklist

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

Must have

Participants must be clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants associated with progressive HO

Participants must be able to undergo low-dose WBCT (excluding head) without sedation

Must not have

Participants with severe hepatic impairment

Participants with significant underlying lung disease requiring supplementary oxygen or with forced vital capacity <35% of predicted at screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up every 6 months up to month 24

Summary

This trial tests IPN60130, a drug aimed at stopping or reducing unwanted bone growth, in adults and children with FOP. FOP causes bone to form in soft tissues, leading to severe disability. The medication works by blocking the process that leads to this abnormal bone growth.

Who is the study for?

This trial is for individuals with Fibrodysplasia Ossificans Progressiva (FOP), carrying specific genetic mutations, who have experienced disease progression in the past year. Participants must be over 5 years old, weigh at least 10 kg, and able to perform certain tests without sedation. They should not be on conflicting medications or have severe organ dysfunction.

What is being tested?

The study is testing two different doses of IPN60130 against a placebo to see if they can prevent new bone growth in soft tissues. It involves scans like low dose Whole Body Computed Tomography and [18F]NaF PET-CT for some participants to measure the effectiveness.

What are the potential side effects?

While specific side effects of IPN60130 are not listed here, common risks may include reactions at the pill intake site, gastrointestinal issues, potential liver or blood abnormalities, and possible interactions with other drugs.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with FOP and have the R206H ACVR1 mutation or a related variant.

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

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I am at least 15 years old and can consent to participate.

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I am at least 5 years old.

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I can perform lung function tests without difficulty.

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My condition has worsened in the last year.

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I can sign the consent form myself, or if I'm a minor, my guardian can.

Exclusion Criteria

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

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I have severe liver problems.

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I need extra oxygen or have severe lung issues as shown in tests.

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I have a complete heart block or left bundle branch block.

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I have certain blood disorders.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ every 6 months up to month 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~every 6 months up to month 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and every 6 months up to month 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

Change from baseline in clinically significant Electrocardiogram (ECG) readings

Change from baseline in clinically significant abnormal values in laboratory parameters (haematology, biochemistry, and urinalysis)

Change from baseline in clinically significant vital signs

+1 more

Secondary study objectives

Assessment of the exposure-response relationship

Change in pain intensity

Flare-up rate and number of flare-up days in participants receiving IPN60130 compared with placebo recipients

+4 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: IPN60130 low dosageExperimental Treatment1 Intervention

Oral capsule, swallowed whole or sprinkled onto food, once daily

Group II: IPN60130 high dosageExperimental Treatment1 Intervention

Oral capsule, swallowed whole or sprinkled onto food, once daily

Group III: PlaceboPlacebo Group1 Intervention

Oral capsule, swallowed whole or sprinkled onto food, once daily

0 / 11Eligibility 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

Fibrodysplasia Ossificans Progressiva (FOP) is characterized by the abnormal formation of bone in soft tissues, leading to severe disability. Treatments like IPN60130 aim to inhibit the process of Heterotopic Ossification (HO), which is the formation of bone outside the normal skeleton. These treatments work by targeting specific pathways involved in bone formation, potentially reducing the volume of new bone growth in soft tissues. This is crucial for FOP patients as it can help manage flare-ups, reduce pain, and preserve mobility, thereby improving their quality of life.

Long-term follow-up and final height in girls with central precocious puberty treated with luteinizing hormone-releasing hormone analogue nasal spray.The effect of Cissus quadrangularis L. on delaying bone loss in postmenopausal women with osteopenia: A randomized placebo-controlled trial.St. Gallen/Vienna 2015: A Brief Summary of the Consensus Discussion.