Trial Outcomes & Findings for Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders (NCT NCT00996931)

Results Overview

Change in CSF-TNF-α from baseline to 12 weeks.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

Baseline and 12 weeks

Results posted on

2013-05-29

Participant Flow

Participant milestones

Participant milestones
Measure	Lenalidomide Six patients will receive oral 2.5 mg daily for 12 weeks
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lenalidomide n=6 Participants Six patients will receive 2.5 mg oral daily for 12 weeks
Age, Categorical <=18 years	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 12 weeks

Population: Intention to treat

Change in CSF-TNF-α from baseline to 12 weeks.

Outcome measures

Outcome measures
Measure	Lenalidomide n=6 Participants Six subjects received oral 2.5 mg daily for 12 weeks
Change in TNF-alpha Levels	57 mean % change Standard Deviation 25

SECONDARY outcome

Timeframe: Baseline and 6 weeks

Population: Intention to treat

Change in CARS value from baseline to 6 weeks. Total CARS scores range from a fifteen to 60, with a minimum score of thirty serving as the cutoff for a diagnosis of autism on the mild end of the autism spectrum.

Outcome measures

Outcome measures
Measure	Lenalidomide n=6 Participants Six subjects received oral 2.5 mg daily for 12 weeks
Change in Childhood Autism Rating Scale (CARS)Value From Baseline to 6 Weeks	-2.08 mean change in units on scale Standard Deviation 1.94

Adverse Events

Lenalidomide

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

Carol Parise, PhD

Sutter Institute for Medical Research

Phone: 916-733-4413

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place