Trial Outcomes & Findings for Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome (NCT NCT00858689)

Results Overview

The 15-item Irritability Scale includes questions about aggression, self-injury, tantrums, agitation, and unstable mood on a scale of 0 to 45 with higher scores indicating greater severity. This scale has been successfully used in previous medication studies in children with autism and in patients with FXS and in a controlled trial of ampakine CX516 in FXS. All ABC subscales showed good reliability when used by parents and caregivers of individuals with FXS to assess behavior in the CX516 study NCT00054730, and yielded intraclass correlation coefficient (ICC) values of 0.7-0.9.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

20 participants

Primary outcome timeframe

Baseline and 8 weeks

Results posted on

2016-02-18

Participant Flow

Twenty subjects with FXS between 13 and 35 years of age were enrolled between December 2007 and June 2008, and after baseline testing they were started on an 8-week treatment course of minocycline added on to any other medications being administered at the time of enrollment.

Inclusion criteria included (1) diagnosis of FXS by clinical evaluation and confirmed by FMR1-DNA testing with presence of full mutation or mosaicism for the full mutation.

Participant milestones

Participant milestones
Measure	Minocyline 50 mg or 100 mg PO BID open label, single arm study of minocyline 50 mg or 100 mg PO BID
Overall Study STARTED	20
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Minocyline 50 mg or 100 mg PO BID n=20 Participants open label minocyline 50 mg or 100 mg PO BID
Age, Categorical <=18 years	11 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	18 years STANDARD_DEVIATION 5 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Region of Enrollment Canada	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 8 weeks

Population: ABC-I change in subjects completing 8 weeks of minocycline treatment

The 15-item Irritability Scale includes questions about aggression, self-injury, tantrums, agitation, and unstable mood on a scale of 0 to 45 with higher scores indicating greater severity. This scale has been successfully used in previous medication studies in children with autism and in patients with FXS and in a controlled trial of ampakine CX516 in FXS. All ABC subscales showed good reliability when used by parents and caregivers of individuals with FXS to assess behavior in the CX516 study NCT00054730, and yielded intraclass correlation coefficient (ICC) values of 0.7-0.9.

Outcome measures

Outcome measures
Measure	Minocyline 50 mg or 100 mg PO BID n=19 Participants open label minocyline 50 mg or 100 mg PO BID
Change From Baseline of ABC Irritability Subtest Score at 8 Weeks	-14.37 units on a scale Standard Deviation 7.84

PRIMARY outcome

Timeframe: 8 weeks

Population: completed 8 weeks of minocycline treatment

ABC Irritability subtest score was used

Outcome measures

Outcome measures
Measure	Minocyline 50 mg or 100 mg PO BID n=19 Participants open label minocyline 50 mg or 100 mg PO BID
ABC Irritability Subtest Score	7.74 units on a scale Standard Deviation 4.82

PRIMARY outcome

Minocyline 50 mg or 100 mg PO BID

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Minocyline 50 mg or 100 mg PO BID n=20 participants at risk open label minocyline 50 mg or 100 mg PO BID
Immune system disorders Increased serum ANA titre	10.0% 2/20 • Number of events 2 • 8 weeks

Additional Information

Michael R. Tranfaglia MD

FRAXA Research Foundation

Phone: 978-462-1990

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place