Oral Fidrisertib for Stone Man Syndrome

(FALKON Trial)

Recruiting at 32 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

113 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

How is the drug Fidrisertib unique in treating Stone Man Syndrome?

Fidrisertib is unique because it is an oral drug that targets fibroblast growth factor receptors (FGFRs), which are involved in various conditions, including cancers. This mechanism of action is different from other treatments for Stone Man Syndrome, as there are no standard treatments specifically targeting FGFRs for this condition. ^{1 2 3 4 5}

Research Team

Ipsen Medical Director

Principal Investigator

Ipsen

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. England 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. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Rogaratinib in patients with advanced cancers selected by FGFR mRNA expression: a phase 1 dose-escalation and dose-expansion study. [2020]

3. Netherlands pubmed.ncbi.nlm.nih.gov

The role of fibroblast growth factor receptor (FGFR) protein-tyrosine kinase inhibitors in the treatment of cancers including those of the urinary bladder. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Multicenter Phase I Study of Erdafitinib (JNJ-42756493), Oral Pan-Fibroblast Growth Factor Receptor Inhibitor, in Patients with Advanced or Refractory Solid Tumors. [2020]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Hyperphosphatemia Secondary to the Selective Fibroblast Growth Factor Receptor 1-3 Inhibitor Infigratinib (BGJ398) Is Associated with Antitumor Efficacy in Fibroblast Growth Factor Receptor 3-altered Advanced/Metastatic Urothelial Carcinoma. [2021]