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Report for Clinical Trial NCT03312634

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

MOVE TRIAL: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO) often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to ankyloses of major joints with cumulative and irreversible loss of movement and disability.

NCT03312634 Fibrodysplasia Ossificans Progressiva
MeSH: Myositis Ossificans

1 Interventions

Name: Palovarotene

Description: Palovarotene will be taken orally once daily at approximately the same time each day following a meal.

Type: Drug

Palovarotene Chronic/Flare-Up Regimen


Primary Outcomes

Description: Annualized change in new HO volume as assessed by low-dose, WBCT (excluding head) compared to untreated subjects from the NHS.

Measure: Change in New HO Volume

Time: Screening, every 6 months up to 2 years

Secondary Outcomes

Description: The proportion of subjects with any new HO.

Measure: Subjects with New HO

Time: Screening, every 6 months up to 2 years

Description: Change from baseline in the number of body regions with new HO.

Measure: Number of Body Regions with HO

Time: Screening, every 6 months up to 2 years

Description: The proportion of subjects reporting flare-ups.

Measure: Subjects with Flare-Ups

Time: Up to 2 years

Description: The rate of flare-ups per subject-month exposure.

Measure: Rate of Flare-Ups

Time: Up to 2 years

Description: Monitor adverse events.

Measure: Incidence of Adverse Events

Time: Up to 2 years

Description: Determination of AUC at steady-state assessed during treatment with 5, 10, and 20 mg palovarotene.

Measure: Palovarotene Area Under the Curve (AUC)

Time: Predose, and 3, 6, 10, and 24 hours postdose

Other Outcomes

Description: Change from baseline in range of motion as assessed by the Cumulative Analogue Joint Involvement Scale for FOP (CAJIS).

Measure: Range of Motion

Time: Screening, every 6 months up to 2 years

Description: Change from baseline in physical function using age-appropriate forms of the FOP-Physical Function Questionnaire (PFQ).

Measure: FOP-Physical Function Questionnaire

Time: Screening, every 6 months up to 2 years

Description: Change from baseline in physical/mental function using age-appropriate forms of the PROMIS Global Health Scale.

Measure: PROMIS Global Health Scale

Time: Screening, every 6 months up to 2 years

Purpose: Treatment

Single Group Assignment


There is one SNP

SNPs


1 R206H

- Previous participation in Clementia's natural history study (NCT02322255); clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants reported to be associated with progressive HO (who have not participated in any Clementia-sponsored study); participants in Clementia's Phase 2 studies (NCT02279095 and NCT02979769) who cannot currently receive the chronic/flare-up regimen due to country of residence or those traveling long distances to participate in the Phase 2 studies. --- R206H ---



HPO Nodes