Trial Outcomes & Findings for In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects (NCT NCT02521792)

Results Overview

The primary endpoint was the safety of palovarotene as assessed by the incidence of TEAEs (including those known to be associated with retinoids) and serious adverse event (SAEs) monitored throughout the treatment period. TEAEs were adverse events reported during treatment with palovarotene or within 6 weeks after the end of treatment. Day 1 was the first day that study drug was administered for a flare-up. The number of subjects experiencing at least one TEAE or treatment-emergent SAE are presented.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

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).

Results posted on

2020-10-19

Participant Flow

Fibrodysplasia Ossificans Progressiva (FOP) subjects treated with palovarotene for 2 flare-ups in Study PVO-1A-202/Part A were enrolled. During enrolment, the dosage regimen for Study PVO-1A-202/Part B was optimized. Study PVO-1A-203 was terminated and participating subjects enrolled into Study PVO-1A-202/Part B to receive the modified regimen.

The screening visit for this study (PVO-1A-203) was the same day as the end of study visit for Study PVO-1A-202/Part A. Subjects experiencing an eligible flare-up received open-label palovarotene (treatment period of at least 6 weeks). Subjects were to be followed for 36 months with telephone/video-conferencing assessments performed every 6 months.

Participant milestones

Participant milestones
Measure	Palovarotene Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 milligram (mg) orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Study STARTED	6
Overall Study Received Treatment	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Palovarotene Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 milligram (mg) orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Study Study Terminated	6

Baseline Characteristics

In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Palovarotene n=6 Participants Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Sex: Female, Male Female	1 Participants n=99 Participants
Sex: Female, Male Male	5 Participants n=99 Participants
Region of Enrollment United States	6 participants n=99 Participants

PRIMARY outcome

Timeframe: 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).

Population: The safety population included all subjects who received at least 1 dose of study drug.

The primary endpoint was the safety of palovarotene as assessed by the incidence of TEAEs (including those known to be associated with retinoids) and serious adverse event (SAEs) monitored throughout the treatment period. TEAEs were adverse events reported during treatment with palovarotene or within 6 weeks after the end of treatment. Day 1 was the first day that study drug was administered for a flare-up. The number of subjects experiencing at least one TEAE or treatment-emergent SAE are presented.

Outcome measures

Outcome measures
Measure	Palovarotene n=4 Participants Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Number of Subjects With Treatment-emergent Adverse Events (TEAEs) TEAE	4 Participants
Number of Subjects With Treatment-emergent Adverse Events (TEAEs) Treatment-emergent SAE	1 Participants

SECONDARY outcome

Timeframe: Every 6 weeks while on study drug, at the end of treatment (should treatment be extended beyond 6 weeks), and 6 weeks after the end of treatment.