Trial Outcomes & Findings for A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms (NCT NCT03421496)

NCT ID: NCT03421496

Last Updated: 2023-06-07

Results Overview

Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

2 participants

Primary outcome timeframe

Up to Day 15

Results posted on

2023-06-07

Participant Flow

This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

This study was comprised of five periods. The Screening Period lasted up to 14 days, the Initial Treatment period lasted up to 15 days, the Extended Treatment period lasted up to 75 days, followed by a 6-day Taper period and a 30 day Follow up period, with a maximum duration of approximately 140 days.

Participant milestones

Participant milestones
Measure
CBD With Vigabatrin
Participants received up to 40 mg/kg/day twice daily (BID) of CBD orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus CBD 40 mg/kg/day up to Day 75.
Placebo With Vigabatrin
Participants received a matching placebo to CBD, orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus matching placebo up to Day 75.
Overall Study
STARTED
0
0
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: Up to Day 15

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 15

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Resolution of IS was assessed by 24-hour video-EEG.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 15

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Resolution of hypsarrhythmia was assessed by 24-hour video-EEG.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 15

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Investigators will use the CGI-I scale, which is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse. Higher scores indicated worse condition.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 15

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Increase in spasm-free days will be determined by seizure diary entries.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 75

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 75

Population: This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Outcome measures

Outcome data not reported

Adverse Events

CBD With Vigabatrin

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Placebo With Vigabatrin

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Bruce Mitlak, MD; Head of Discovery Science and Chief Medical Officer

Radius Health

Phone: 617-444-1943

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place