Oral Fidrisertib for Stone Man Syndrome

(FALKON Trial)

Not currently recruiting at 37 trial locations

Overseen By Ipsen Clinical Study Enquiries

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Clementia Pharmaceuticals Inc.

Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers, Kinase inhibitors, Bisphosphonates

Disqualifiers: Heart block, Lung disease, Hepatic impairment, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called IPN60130 (also known as Fidrisertib) for individuals with Fibrodysplasia Ossificans Progressiva (FOP), a condition where bone forms in muscles or soft tissues. The trial aims to determine if two different doses of IPN60130 can reduce the growth of this abnormal bone compared to a placebo. Participants with FOP who have experienced disease progression in the past year may qualify. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires stopping certain medications, especially those that strongly affect liver enzymes or are kinase inhibitors like imatinib. If you've used specific treatments like palovarotene or garetosmab, a washout period (time without taking these medications) is needed before joining the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that IPN60130, a pill for treating Fibrodysplasia Ossificans Progressiva (FOP), has undergone earlier testing to ensure safety. These studies suggest the treatment could help manage the condition. Participants tolerated the medication well, with no serious side effects reported. The trial is now in its middle phase, indicating it has progressed beyond the initial safety tests. This suggests reasonable evidence that the treatment is generally safe, although further information will help confirm this. Prospective participants can find some reassurance in these safety findings. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Stone Man Syndrome?

Unlike standard treatments for Stone Man Syndrome, which primarily focus on managing symptoms, IPN60130 offers a new approach by targeting the underlying mechanisms of the disease. Researchers are excited about this treatment because it involves a unique oral administration method, either swallowed whole or sprinkled onto food, which can improve patient compliance. Additionally, IPN60130 is being tested at both high and low dosages to optimize its effectiveness, offering a potential breakthrough in managing this challenging condition.

What evidence suggests that this trial's treatments could be effective for Stone Man Syndrome?

Research has shown that the drug IPN60130 might help reduce unwanted bone growth in soft tissues, known as heterotopic ossification (HO), in individuals with Fibrodysplasia Ossificans Progressiva (FOP). This treatment aims to prevent the painful flare-ups and stiffness linked to this condition. In this trial, participants will receive either a high or low dosage of IPN60130, or a placebo. Early results suggest that both doses of this oral medication have shown promise in slowing the growth of unwanted bone compared to a placebo. These initial findings are encouraging and lead to more detailed studies. ^{1 2 3 6 7}

Who Is on the Research Team?

Ipsen Medical Director

Principal Investigator

Ipsen

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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

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

I can attend all treatment sessions and follow-up appointments.

Exclusion Criteria

I have severe liver problems.

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

I do not have any major health issues that are not under control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either high or low dosage of IPN60130 or placebo daily to evaluate efficacy in inhibiting new HO volume

24 months

Monthly visits for assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Long-term monitoring

Participants are monitored for changes in laboratory parameters, physical examination findings, vital signs, and ECG readings

63 months

What Are the Treatments Tested in This Trial?

Interventions

• IPN60130
• Placebo

Trial Overview 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.

How Is the Trial Designed?

Group I: IPN60130 low dosage Experimental Treatment 1 Intervention

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

Group II: IPN60130 high dosage Experimental Treatment 1 Intervention

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

Group III: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Clementia Pharmaceuticals Inc.

Lead Sponsor

Trials

11

Recruited

750+

Ipsen

Industry Sponsor

Trials

358

Recruited

74,600+

David Loew

Ipsen

Chief Executive Officer since 2020

BA in Business Administration and MBA from the University of St. Gallen, Switzerland

Sandra Silvestri

Ipsen

Chief Medical Officer since 2023

MD, PhD

Published Research Related to This Trial

In the phase I/II FIGHT-101 study involving 128 patients with advanced malignancies, pemigatinib demonstrated a manageable safety profile with no dose-limiting toxicities and a recommended phase II dose of 13.5 mg once daily.

The treatment showed preliminary efficacy, particularly in patients with FGFR fusions/rearrangements, achieving a 25% response rate, and prompting further studies in cholangiocarcinoma and other tumor types.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FIGHT-101, a first-in-human study of potent and selective FGFR 1-3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies. Subbiah, V., Iannotti, NO., Gutierrez, M., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05039515

NCT05039515 | A Study to Assess the Effectiveness and ... This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9624752/

ODP103 IPN60130 for the Treatment of Fibrodysplasia ... Results from the FALKON trial, expected to complete in August 2025, will enable evaluation of the efficacy and safety of IPN60130 in patients with FOP.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05039515?term=AREA%5BBasicSearch%5D(AREA%5BBasicSearch%5D(AREA%5BBasicSearch%5D(AREA%5BBasicSearch%5D(AREA%5BBasicSearch%5D(AREA%5BConditionSearch%5D(%22Myositis%20Ossificans%22))))))&rank=8

A Study to Assess the Effectiveness and Safety of 2 ... A group of independent scientists who monitor the safety and scientific integrity of a clinical trial. The DMC can recommend to the sponsor that the trial be ...

4. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT05039515

2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the ... This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10378717/

Novel Therapeutic Targets for Fibrodysplasia Ossificans ... This narrative review provides an overview of the disease and explores emerging strategies for FOP treatment.

6. academic.oup.com academic.oup.com/jbmrplus/advance-article/doi/10.1093/jbmrpl/ziaf150/8258639?searchresult=1

Medical guidelines for fibrodysplasia ossificans progressiva As FOP is an ultra-rare condition, evidence-based statements are generally moderate to low. If published data were unavailable or insufficient, ...

7. ifopa.org ifopa.org/ipn60130

FALKON Trial (fidrisertib) The trial is designed to evaluate the safety of 2 dosing regimens of oral fidrisertib in inhibiting new HO volume compared with placebo (a dummy treatment)