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Palovarotene for Stone Man Syndrome

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 until 6 weeks after the end of treatment (an expected average treatment of 6 weeks). assessed until data cut-off for study termination (maximum of 35 days).

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation (heterotopic ossification or HO) in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints leading to cumulative loss of function and disability. Mouse models of FOP have demonstrated the ability of retinoic acid receptor gamma (RARγ) agonists such as palovarotene to prevent HO following injury. This 36-month study will evaluate the long-term safety and efficacy of episodic treatment with palovarotene for flare-ups in FOP subjects who successfully complete two flare-up treatment periods (6 weeks duration) and two follow-up periods (6 weeks duration) in Study PVO-1A-202.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 until 6 weeks after the end of treatment (an expected average treatment of 6 weeks). assessed until data cut-off for study termination (maximum of 35 days).

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 until 6 weeks after the end of treatment (an expected average treatment of 6 weeks). assessed until data cut-off for study termination (maximum of 35 days).

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 until 6 weeks after the end of treatment (an expected average treatment of 6 weeks). assessed until data cut-off for study termination (maximum of 35 days). for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Subjects With Treatment-emergent Adverse Events (TEAEs)

Side effects data

From 2022 Phase 3 trial • 107 Patients • NCT03312634

50%

Dry skin

50%

Alopecia

50%

Pruritus

50%

Dry Eye

50%

Rash

25%

Paronychia

25%

Hypoacusis

25%

Condition Aggravated

25%

Anemia

25%

Erythema

25%

Skin exfoliation

25%

Skin irritation

25%

Chapped Lips

25%

Nasopharyngitis

25%

Musculoskeletal Pain

25%

Skin Abrasion

25%

Headache

25%

Skin Fissures

25%

Skin Disorder

25%

Skin Burning Sensation

25%

Hypotrichosis

25%

Swelling Face

25%

Musculoskeletal Stiffness

25%

Dizziness

25%

Hyperesthesia

25%

Oropharyngeal Pain

25%

Tinnitus

25%

Pain

25%

Visual Impairment

25%

Ocular Hyperemia

25%

Alanine Aminotransferase Increased

25%

Blood Alkaline Phosphatase Increased

25%

Nausea

25%

Breast Cyst

25%

Pruritus generalized

25%

Gamma-Glutamyltransferase Increased

100%

80%

60%

40%

20%

Study treatment Arm

Flare-Up Treatment: Supplemental SS

Untreated (PVO-1A-001)

Palovarotene 20/10 mg

Untreated (PVO-1A-001)

Palovarotene 5 mg

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: PalovaroteneExperimental Treatment1 Intervention

The protocol is open only to the subjects who completed Clementia Study PVO-1A-202. Eligible subjects will receive a weight-based equivalent dose of palovarotene 10 mg once daily for 14 days, followed by 5 mg once daily for 28 days. Should treatment be extended beyond 6 weeks, a weight-based equivalent dose of 5 mg will be administered in 2-week increments.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Palovarotene

FDA approved

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