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Trial Outcomes & Findings for Minocycline to Treat Childhood Regressive Autism (NCT NCT00409747)

NCT ID: NCT00409747

Last Updated: 2015-12-11

Results Overview

The Mann-Whitney U-test was used to compare pre-/post-treatment differences in analyte concentrations in serum, plasma and CSF. The Mann-Whitney U test generates a z-score test statistic with an associated p value. A negative z-statistic reflects a decrease in analyte level from pre- to post-treatment. Statistical significance level was set at 0.05. There is one test statistic (z-score) per analyte, reflecting the pre-post comparison across all subjects. Pre and post treatment measurements of csf analytes: TNF alpha, Il-6, CCL-2(MCP-1), CCL3 (MIP-1alpha), CCL5(RANTES), CXCL(IL-8), BDNF, CD40L, GDNF, HGF, Leptin

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

11 participants

Primary outcome timeframe

Pre and post treatment with minocyline at 6 months for 10 subjects

Results posted on

2015-12-11

Participant Flow

Referrals accepted from community physicians and self(parent) referrals.

Three subjects did not start the drug due to abnormal MRI,initial elevated liver enzymes, and and initial elevated amylase. 11 subjects began taking drug. One subject withdrew after starting drug.

Participant milestones

Participant milestones
Measure
Minocycline
Open-label minocycline treatment at 1.4 mg/kg/day
Overall Study
STARTED
11
Overall Study
COMPLETED
10
Overall Study
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Minocycline
Open-label minocycline treatment at 1.4 mg/kg/day
Overall Study
Withdrawal by Subject
1

Baseline Characteristics

Minocycline to Treat Childhood Regressive Autism

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Minocycline
n=11 Participants
Open-label minocycline treatment at 1.4 mg/kg/day
Age, Categorical
<=18 years
11 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
Age, Continuous
7.2 years
STANDARD_DEVIATION 2.9 • n=5 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
Sex: Female, Male
Male
9 Participants
n=5 Participants
Region of Enrollment
United States
11 participants
n=5 Participants

PRIMARY outcome

Timeframe: Pre and post treatment with minocyline at 6 months for 10 subjects

Population: 10 subjects completed six months of minocycline and have pre-/post-minocycline CSF samples for analysis.

The Mann-Whitney U-test was used to compare pre-/post-treatment differences in analyte concentrations in serum, plasma and CSF. The Mann-Whitney U test generates a z-score test statistic with an associated p value. A negative z-statistic reflects a decrease in analyte level from pre- to post-treatment. Statistical significance level was set at 0.05. There is one test statistic (z-score) per analyte, reflecting the pre-post comparison across all subjects. Pre and post treatment measurements of csf analytes: TNF alpha, Il-6, CCL-2(MCP-1), CCL3 (MIP-1alpha), CCL5(RANTES), CXCL(IL-8), BDNF, CD40L, GDNF, HGF, Leptin

Outcome measures

Outcome measures
Measure
Minocycline
n=10 Participants
Open-label minocycline treatment at 1.4 mg/kg/day
z Score
GDNF
-0.37 Z-score
z Score
HGF
-2.19 Z-score
z Score
Leptin
-1.48 Z-score
z Score
TNFalpha
-0.45 Z-score
z Score
IL-6
-0.36 Z-score
z Score
CCL2(MCP-1)
-1.78 Z-score
z Score
CCL3(MIP-1alpha)
-0.18 Z-score
z Score
CCL5(Rantes)
-0.73 Z-score
z Score
CXCL8(IL-8)
-0.45 Z-score
z Score
BDNF
-2.03 Z-score
z Score
CD40L
-0.89 Z-score

SECONDARY outcome

Timeframe: Baseline and 6 months

Population: All children completing 6 months of drug and participating in final evaluation

This instrument has two scales - Severity (CGI-S). The CGI-S is a seven point scale with a minimum score of 1 and a maximum score as 7 as follows: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; and 7 = among the most extremely ill patients.

Outcome measures

Outcome measures
Measure
Minocycline
n=10 Participants
Open-label minocycline treatment at 1.4 mg/kg/day
Clinical Global Impressions Scale-Severity (CGI). (Connors & Barkley, 1985)
Baseline
4.4 mean scores on global impression scale
Standard Deviation 0.8
Clinical Global Impressions Scale-Severity (CGI). (Connors & Barkley, 1985)
6 months
4.6 mean scores on global impression scale
Standard Deviation .81

Adverse Events

Overall Study/Minocycline

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Overall Study/Minocycline
n=11 participants at risk
Gastrointestinal disorders
Vomiting and fever
9.1%
1/11 • Number of events 1 • 6 months of minocycline administration

Additional Information

Dr. Susan Swedo

NIH/NIMH/PDN Branch

Phone: 301-496-5323

Results disclosure agreements

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