An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva. (MOVE)

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ClinicalTrials.gov Identifier: NCT03312634

Recruitment Status : Completed

First Posted : October 18, 2017

Results First Posted : March 14, 2023

Last Update Posted : March 14, 2023

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Ipsen ( Clementia Pharmaceuticals Inc. )

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition	Fibrodysplasia Ossificans Progressiva
Intervention	Drug: Palovarotene
Enrollment	107

Participant Flow

Recruitment Details

This Phase 3, open-label study conducted in adult and pediatric participants with fibrodysplasia ossificans progressiva (FOP) at 16 centers in 11 countries (Argentina, Australia, Brazil, Canada, France, Italy, Japan, Spain, Sweden, the United Kingdom, and the US). Results reported here are based on the database cut-off date (DCO) of 28-Feb-2020.

Pre-assignment Details

This study included 2 parts: Part A, the main part of the study, and Part B, the 24-month extension. A total of 107 participants were enrolled and treated in this study. Data from participants in PVO-1A-001 were used as an external control for only primary endpoint of this study. Hence, data for participants in PVO-1A-001 are reported in participant flow, baseline characteristics and adverse events section only.


Arm/Group Title	Palovarotene	Untreated (PVO-1A-001)
Arm/Group Description	Participants were administered 5 mi...	Participants from study PVO-1A-001 ...
Arm/Group Description	Participants were administered 5 milligram (mg) palovarotene orally once daily up to 24 months. Participants with flare-up symptoms or traumatic events received palovarotene 20 mg once daily for 4 weeks after the flare-up confirmation by the Investigator. Followed by palovarotene 10 mg once daily for 8 weeks.	Participants from study PVO-1A-001 (NCT02322255) were included with FOP caused by the R206H mutation and with baseline data. Participants were not administered palovarotene in this study and only compared as external control.
Period Title: Overall Study
Started	107	114
Completed	0	33
Not Completed	107	81
Reason Not Completed
Adverse Event	6	0
Sponsor request	2	0
Withdrawal by Subject	11	9
Ongoing in study at DCO	88	0
Enrolled in an interventional study	0	52
Enrolled in an interventional study at time of a flare-up	0	9
Death	0	1
Non-compliance	0	2
Lost to Follow-up	0	1
Enrolled into non-interventional study	0	5
Worsening clinical condition	0	1
Participant did not want to travel	0	1

Baseline Characteristics

Arm/Group Title		Palovarotene	Untreated (PVO-1A-001)	Total
Arm/Group Description		Participants were administered 5 mg...	Participants from study PVO-1A-001 ...	Total of all reporting groups
Arm/Group Description		Participants were administered 5 mg palovarotene orally once daily up to 24 months. Participants with flare-up symptoms or traumatic events received palovarotene 20 mg once daily for 4 weeks after the flare-up confirmation by the Investigator. Followed by palovarotene 10 mg once daily for 8 weeks.	Participants from study PVO-1A-001 (NCT02322255) were included with FOP caused by the R206H mutation and with baseline data. Participants were not administered palovarotene in this study and only compared as external control.	Total of all reporting groups
Overall Number of Baseline Participants		99	114	213
Baseline Analysis Population Description		...
Baseline Analysis Population Description		The Principal Safety Set (Principal SS) included all enrolled participants in the Principal Enrolled Population (Principal EP) set [ie, participants with the R206H activin receptor type IA (ACVR1) mutation] receiving at least 1 dose of palovarotene in study PVO-1A-301. The Full Analysis Set (FAS) included all participants in the Enrolled Analysis Set with FOP caused by the R206H mutation and who had baseline data in study PVO-1A-001 (NCT02322255).
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	99 participants	114 participants	213 participants
	<=18 years	75	70	145
	Between 18 and 65 years	24	44	68
	>=65 years	0	0	0
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	99 participants	114 participants	213 participants
	Female	46	52	98
	Male	53	62	115
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	99 participants	114 participants	213 participants
	Hispanic or Latino	19	23	42
	Not Hispanic or Latino	69	73	142
	Unknown or Not Reported	11	18	29
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	99 participants	114 participants	213 participants
	White	70	84	154
	Black or African American	1	0	1
	Asian	9	8	17
	American Indian or Alaska Native	0	1	1
	Native Hawaiian or other Pacific Islander	1	0	1
	Multiple	6	2	8
	Other	1	4	5
	Unknown	11	15	26

Outcome Measures

1.Primary Outcome


Title	Annualized New Heterotopic Ossification (HO)
Description	The annualized new HO was assessed by low-dose, whole body computed to...
Description	The annualized new HO was assessed by low-dose, whole body computed tomography (WBCT), excluding head. The weighted linear mixed effect method without square-root transformation and negatives included was used for annualized new HO analysis.
Time Frame	Baseline (within one month of screening/Day 1) and up to 24 months

Outcome Measure Data