Disease · MONDO_0010383
fragile X syndrome
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. fragile X syndrome. Diseases, record MONDO_0010383. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010383
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
- FRAXA syndrome, FXS, FraX syndrome, Fragile X syndrome, X-linked dominant, Martin-Bell syndrome, fragile X intellectual disability syndrome, marker X syndrome
- Open Targets
- Highest stage of its drugs
- Phase 3, for 5 of its 30 drugs, from ClinicalTrials.gov and TTD
- Open Targets
Drugs30
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| ARBACLOFEN | CHEMBL301742 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| CANNABIDIOL | CHEMBL190461 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| ZATOLMILAST | CHEMBL4541964 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 4 | 4 | Drugs and targets |
| BACLOFEN | CHEMBL701 | Small molecule | Phase 3 | TTD | 3 | 2 | 2 | Drugs and targets |
| ARBACLOFEN PLACARBIL | CHEMBL2107312 | Small molecule | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets |
| MAVOGLURANT | CHEMBL3087515 | Small molecule | Phase 2/3 | ClinicalTrials.gov and TTD | 8 | 7 | 7 | Drugs and targets |
| METFORMIN | CHEMBL1431 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| MINOCYCLINE | CHEMBL1434 | Small molecule | Phase 2 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| BASIMGLURANT | CHEMBL3301626 | Small molecule | Phase 2 | ClinicalTrials.gov and TTD | 4 | 3 | 3 | Drugs and targets |
| GABOXADOL | CHEMBL312443 | Small molecule | Phase 2 | ClinicalTrials.gov and TTD | 4 | 3 | 3 | Drugs and targets |
1 to 10 of 30
See all 30 drugsTargets103
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| GABBR1 | GABA-B receptor | 3 | GABA-B receptor agonist | Phase 3 | https://conductscience.org/library/genes/2550 | Drugs and targets |
| GABBR2 | GABA-B receptor | 3 | GABA-B receptor agonist | Phase 3 | https://conductscience.org/library/genes/9568 | Drugs and targets |
| GABRA2 | GABA-A receptor; anion channel | 3 | GABA receptor alpha-2 subunit positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator | Phase 2 | https://conductscience.org/library/genes/2555 | Drugs and targets |
| GABRA3 | GABA-A receptor; anion channel | 3 | GABA receptor alpha-3 subunit positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator | Phase 2 | https://conductscience.org/library/genes/2556 | Drugs and targets |
| GABRA4 | GABA-A receptor; anion channel | 3 | GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist | Phase 2 | https://conductscience.org/library/genes/2557 | Drugs and targets |
| GABRB3 | GABA-A receptor; anion channel | 3 | GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist | Phase 2 | https://conductscience.org/library/genes/2562 | Drugs and targets |
| GABRD | GABA-A receptor; anion channel | 3 | GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist | Phase 2 | https://conductscience.org/library/genes/2563 | Drugs and targets |
| GRM5 | Metabotropic glutamate receptor 5 | 3 | Metabotropic glutamate receptor 5 antagonist; Metabotropic glutamate receptor 5 modulator; Metabotropic glutamate receptor 5 negative allosteric modulator | Phase 2/3 | https://conductscience.org/library/genes/2915 | Drugs and targets |
| DRD2 | D(2) dopamine receptor | 2 | Dopamine D2 receptor partial agonist; Dopamine receptor agonist | Phase 2 | https://conductscience.org/library/genes/1813 | Drugs and targets |
| GABRA1 | GABA-A receptor; anion channel | 2 | GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator | Phase 2 | https://conductscience.org/library/genes/2554 | Drugs and targets |
1 to 10 of 103
See all 103 targetsTrials52
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03614663 | Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) | Phase 2/3 | 212 | 2022-06-16 | Fragile X Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
| NCT01348087 | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome | Phase 2 | 148 | 2016-05-25 | Fragile X Syndrome | MAVOGLURANT | Its record names the disease | ClinicalTrials.gov |
| NCT01357239 | Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome | Phase 2 | 139 | 2015-05-12 | Fragile X Syndrome | MAVOGLURANT | Its record names the disease | ClinicalTrials.gov |
| NCT01433354 | Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label) | Phase 2/3 | 119 | 2015-10-12 | Fragile X Syndrome | MAVOGLURANT | Its record names the disease | ClinicalTrials.gov |
| NCT02920892 | AFQ056 for Language Learning in Children With FXS | Phase 2 | 110 | 2023-10-10 | Fragile X Syndrome | MAVOGLURANT | Its record names the disease | ClinicalTrials.gov |
| NCT01053156 | Trial of Minocycline to Treat Children With Fragile X Syndrome | Not applicable | 66 | 2014-07-30 | Fragile X Syndrome | MINOCYCLINE | Its record names the disease | ClinicalTrials.gov |
| NCT01725152 | Ganaxolone Treatment in Children With Fragile X Syndrome | Phase 2 | 59 | 2023-04-10 | Fragile x Syndrome | GANAXOLONE | Its record names the disease | ClinicalTrials.gov |
| NCT01474746 | Trial of Sertraline to Treat Children With Fragile X Syndrome | Phase 2 | 57 | 2017-09-25 | Fragile X Syndrome | SERTRALINE | Its record names the disease | ClinicalTrials.gov |
| NCT01120626 | Randomized Controlled Study of Donepezil in Fragile X Syndrome | Phase 2 | 45 | 2016-03-17 | Fragile X Syndrome | DONEPEZIL | Its record names the disease | ClinicalTrials.gov |
| NCT03697161 | A Study of OV101 in Individuals With Fragile X Syndrome | Phase 2 | 36 | 2024-01-23 | Fragile X Syndrome (FXS) | GABOXADOL | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 52
See all 52 trialsComparisons in its trials14
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| ZYN002 - Cannabidiol Transdermal Gel vs Placebo Transdermal Gel: deaths from any cause | 0 of 109 | 0 of 102 | 211 | NCT03614663 | Trial comparisons |
| ZYN002 - Cannabidiol Transdermal Gel vs Placebo Transdermal Gel: serious adverse events | 0 of 109 | 0 of 102 | 211 | NCT03614663 | Trial comparisons |
| ZYN002 - Cannabidiol Transdermal Gel vs Placebo Transdermal Gel: Number of Participants With Any Improvement - Clinical Global Impressions- Improvement (CGI-I) - Full Analysis Set | 46 of 109 | 37 of 101 | 210 | NCT03614663 | Trial comparisons |
| ZYN002 - Cannabidiol Transdermal Gel vs Placebo Transdermal Gel: Number of Participants With Any Improvement - Clinical Global Impressions- Improvement (CGI-I) - Ad Hoc Analysis | 43 of 91 | 25 of 76 | 167 | NCT03614663 | Trial comparisons |
| Minocycline vs Placebo: serious adverse events | 0 of 66 | 1 of 66 | 132 | NCT01053156 | Trial comparisons |
| Ganaxolone vs Placebo: deaths from any cause | 0 of 59 | 0 of 54 | 113 | NCT01725152 | Trial comparisons |
| Ganaxolone vs Placebo: serious adverse events | 0 of 59 | 0 of 54 | 113 | NCT01725152 | Trial comparisons |
Placebo Comparator: Double-Blind Placebo With Language Intervention vs Experimental: Double-Blind AFQ056 With Language Intervention: Number of Participants With a Positive Response as Defined by Improvement in Clinical Global Impression - Improvement (CGI-I) Overall Function Scores | 10 of 49 | 8 of 50 | 99 | NCT02920892 | Trial comparisons |
| BPN14770 vs Placebo: deaths from any cause | 0 of 30 | 0 of 30 | 60 | NCT03569631 | Trial comparisons |
| BPN14770 vs Placebo: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 11 of 30 | 8 of 30 | 60 | NCT03569631 | Trial comparisons |
1 to 10 of 14
See all 14 comparisons in its trialsPapers from its trials16
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it | Source |
|---|---|---|---|
| 21862226 | RESULT | 1 | PubMed |
| 20937127 | RESULT | 1 | PubMed |
| 42104256 | DERIVED | 1 | PubMed |
| 41254489 | DERIVED | 1 | PubMed |
| 39483619 | DERIVED | 1 | PubMed |
| 39248107 | DERIVED | 1 | PubMed |
| 36434514 | DERIVED | 1 | PubMed |
| 36167501 | DERIVED | 1 | PubMed |
| 34690787 | DERIVED | 1 | PubMed |
| 33927413 | DERIVED | 1 | PubMed |
1 to 10 of 16
See all 16 papers from its trialsData dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0010383
GET https://conductscience.org/api/diseases/v1/MONDO_0010383/drugs?page=2