Palovarotene for Stone Man Syndrome

(PIVOINE Trial)

Recruiting at 12 trial locations

Overseen By Ipsen Recruitment Enquiries

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Ipsen

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 3 years

63 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called palovarotene to help people with a rare condition called Fibrodysplasia Ossificans Progressiva (FOP). FOP causes muscles and other soft tissues to turn into bone. Palovarotene aims to stop this abnormal bone growth by blocking certain signals in the body. The study includes both adults and children.

Research Team

Ipsen Medical Director

Principal Investigator

Ipsen

Eligibility Criteria

This trial is for males and females aged 14 or older with Fibrodysplasia Ossificans Progressiva (FOP) who have finished previous related studies. Participants under 18 must be fully skeletally mature, while those over 18 are presumed so. They should not have any conditions that could risk their safety or affect the study.

Inclusion Criteria

Participant has completed the EOS or End of Treatment Visit of Study PVO-1A-301 or PVO-1A-202 (PVO-1A-202 Parts C and D correspond to Study PVO-1A-204 in France) and did not previously withdraw consent from any of the parent studies to be eligible for Study CLIN-60120-452.

I am 14 or older and my bones have fully grown.

Exclusion Criteria

Suicidal ideation (type 4 or 5) or any suicidal behaviour at the Inclusion Visit as defined by the Columbia-Suicide Severity Rating Scale (C-SSRS).

I have a painful broken bone in my spine.

I am not taking drugs that strongly affect liver enzyme CYP450 3A4 or drugs like imatinib.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive chronic and flare-up treatment with palovarotene

3 years

Regular visits every six months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Palovarotene

Trial Overview The trial is testing the continued use of Palovarotene capsules to assess its long-term safety and effectiveness in treating FOP. It's designed for patients from prior parent studies who might benefit from ongoing treatment.

Participant Groups

Group I: Palovarotene Chronic/Flare-Up Regimen Experimental Treatment 1 Intervention

Chronic treatment: participants will receive 5 mg palovarotene or the dose received during participation in the parent study at the time of transition to Study CLIN-60120-452 or prior to interrupting/stopping palovarotene treatment. Flare-up treatment: at the time of a flare-up (or substantial high-risk traumatic event likely to lead to a flare-up) participants will receive 20 mg palovarotene for 28 days, followed by 10 mg palovarotene for 56 days.

Who Is Running the Clinical Trial?

Ipsen

Lead 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