Evidence map›Paper›PMID 20937127›Full record

Trial reportBMC neurology2010

Open-label add-on treatment trial of minocycline in fragile X syndrome.

Carlo Paribello, Leeping Tao, Anthony Folino, Elizabeth Berry-Kravis, Michael Tranfaglia, Iryna M Ethell, Douglas W Ethell

Registry-linked trialOpen access · goldAbstract readRandomized Controlled Trial
In one paragraph

Trial report in BMC neurology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00858689 (Add-on Pilot Trial of Minocycline in Fragile X Syndrome), which is not on this map. Cited by 112 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
112citing papers in PubMed, 1 pooled it
15.3field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT00858689 nacompletednot on this map

Add-on Pilot Trial of Minocycline in Fragile X Syndrome

TypeinterventionalSponsorFRAXA Research FoundationRan2007 to 2009Enrolled20ConditionsFragile X SyndromeArmsMinocycline
3 · Its place in the literature

Who cites it

112 citing papers in PubMed, 1 synthesis or guideline pooled it, 218 citations in OpenAlex.

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  13. Gut microbiome-dependent IL-1 signaling is a mediator of ACVR1bioRxiv : the preprint server for biology · 2026
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  15. Targeted Treatments for Fragile X Syndrome.Advances in neurobiology · 2023
    Article
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52 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 5 institutions in 2 countries.

Carlo ParibelloSurrey Place Centre, Toronto, Ontario, Canada. fxrfc@on.aibn.com
Leeping Tao
Anthony Folino
Elizabeth Berry-Kravis
Michael Tranfaglia
Iryna M Ethell
Douglas W Ethell
Surrey Place Centre · CAFraxa Research Foundation · USRush University Medical Center · USUniversity of California, Riverside · USWestern University of Health Sciences · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundFragile X syndrome (FXS) is a disorder characterized by a variety of disabilities, including cognitive deficits, attention-deficit/hyperactivity disorder, autism, and other socio-emotional problems. It is hypothesized that the absence of the fragile X mental retardation protein (FMRP) leads to higher levels of matrix metallo-proteinase-9 activity (MMP-9) in the brain. Minocycline inhibits MMP-9 activity, and alleviates behavioural and synapse abnormalities in fmr1 knockout mice, an established model for FXS. This open-label add-on pilot trial was conducted to evaluate safety and efficacy of minocycline in treating behavioural abnormalities that occur in humans with FXS.

methodsTwenty individuals with FXS, ages 13-32, were randomly assigned to receive 100 mg or 200 mg of minocycline daily. Behavioural evaluations were made prior to treatment (baseline) and again 8 weeks after daily minocycline treatment. The primary outcome measure was the Aberrant Behaviour Checklist-Community Edition (ABC-C) Irritability Subscale, and the secondary outcome measures were the other ABC-C subscales, clinical global improvement scale (CGI), and the visual analog scale for behaviour (VAS). Side effects were assessed using an adverse events checklist, a complete blood count (CBC), hepatic and renal function tests, and antinuclear antibody screen (ANA), done at baseline and at 8 weeks.

resultsThe ABC-C Irritability Subscale scores showed significant improvement (p < 0.001), as did the VAS (p = 0.003) and the CGI (p < 0.001). The only significant treatment-related side effects were minor diarrhea (n = 3) and seroconversion to a positive ANA (n = 2).

conclusionsResults from this study demonstrate that minocycline provides significant functional benefits to FXS patients and that it is well-tolerated. These findings are consistent with the fmr1 knockout mouse model results, suggesting that minocycline modifies underlying neural defects that account for behavioural abnormalities. A placebo-controlled trial of minocycline in FXS is warranted.

trial registrationClinicalTrials.gov Open-Label Trial NCT00858689.

Indexed as

AdolescentAdultEnzyme InhibitorsFemaleFragile X SyndromeHumansMaleMinocyclinePilot ProjectsTreatment OutcomeYoung AdultEnzyme InhibitorsMinocycline

Identifiers

PMID20937127
PMCPMC2958860
OpenAlexW2153053291

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.