← Back to Search

Other

Oral Fidrisertib for Stone Man Syndrome (FALKON Trial)

Phase 2
Recruiting
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up every 6 months up to month 24
Awards & highlights

FALKON Trial Summary

This trial will study the efficacy of IPN60130 in inhibiting new Heterotopic Ossification (HO) volume compared to placebo in people with Fibrodysplasia Ossificans Progressiva (FOP).

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.Check my eligibility
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.See study design
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.

FALKON Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with FOP and have the R206H ACVR1 mutation or a related variant.
Select...
I can have a full-body CT scan without needing sedation.
Select...
I am at least 15 years old and can consent to participate.
Select...
I am at least 5 years old.
Select...
I can perform lung function tests without difficulty.
Select...
My condition has worsened in the last year.
Select...
I can sign the consent form myself, or if I'm a minor, my guardian can.

FALKON Trial Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Annualized change in HO volume as assessed by low-dose WBCT (excluding the head) in treated participants receiving IPN60130 compared with placebo.
Change from baseline in clinically significant Electrocardiogram (ECG) readings
Change from baseline in clinically significant abnormal values in laboratory parameters (haematology, biochemistry, and urinalysis)
+3 more
Secondary outcome measures
Assessment of the exposure-response relationship
Change from baseline in Cumulative Analogue Joint Involvement Scale for FOP (CAJIS) by treatment arm compared with placebo recipients and participants receiving the standard of care in the NHS across all available timepoints
Change from baseline in HO volume as detected by WBCT in participants receiving IPN60130 compared with placebo recipients and with participants receiving the standard of care in the Natural history study (NHS)
+11 more

FALKON 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

Find a Location

Who is running the clinical trial?

Clementia Pharmaceuticals Inc.Lead Sponsor
10 Previous Clinical Trials
637 Total Patients Enrolled
IpsenIndustry Sponsor
345 Previous Clinical Trials
73,130 Total Patients Enrolled
Ipsen Medical DirectorStudy DirectorIpsen
257 Previous Clinical Trials
55,119 Total Patients Enrolled

Media Library

IPN60130 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05039515 — Phase 2
Stone Man Syndrome Research Study Groups: IPN60130 high dosage, IPN60130 low dosage, Placebo
Stone Man Syndrome Clinical Trial 2023: IPN60130 Highlights & Side Effects. Trial Name: NCT05039515 — Phase 2
IPN60130 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05039515 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the enrollment for this clinical trial still open?

"Clinicaltrials.gov affirms that recruitment for this clinical trial is still ongoing; it was first published on December 1st 2021, and the latest edit took place on November 21st 2022."

Answered by AI

How many facilities have been identified to oversee this clinical trial?

"This medical trial is being conducted at 7 sites, including UCSF in San Francisco and the University of Alberta along with AHS in Toronto. There are also 4 additional locations participating."

Answered by AI

How many people are participants in this experiment?

"This medical research requires 90 participants who meet the pre-specified conditions. Patients can join from two different sites, such as University of California San Francisco (UCSF) in San Francisco and another at University of California San Francisco located in Edmonton, Minnesota."

Answered by AI

What has been the established safety profile of IPN60130?

"IPN60130's safety is rated at level 2 as it has yet to demonstrate efficacy in clinical trials, despite having some data supporting its safety."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).
2021. "A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05039515.
~36 spots leftby Aug 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top