Disease · MONDO_0012589

Pitt-Hopkins syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Pitt-Hopkins syndrome. Diseases, record MONDO_0012589. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0012589

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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.

Name: Pitt-Hopkins syndrome; Open Targets
Open Targets ID: MONDO_0012589; Open Targets
Disease Ontology: DOID:0060488; Disease Ontology
MeSH: C537403; MeSH
NCI Thesaurus: C129872; NCI Thesaurus
Orphanet: ORPHA:2896; Orphanet
Highest stage of its drugs: Phase 2, for 2 of its 3 drugs, from ClinicalTrials.gov; Open Targets

Drugs3

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
VANCOMYCIN	CHEMBL262777	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
NNZ-2591	CHEMBL4802144	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
VORINOSTAT	CHEMBL98	Small molecule	Phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets4

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
HDAC1	Histone deacetylase 1	1	Histone deacetylase 1 inhibitor	Phase 1	https://conductscience.org/library/genes/3065	Drugs and targets
HDAC2	Histone deacetylase 2	1	Histone deacetylase 2 inhibitor	Phase 1	https://conductscience.org/library/genes/3066	Drugs and targets
HDAC3	Histone deacetylase 3	1	Histone deacetylase 3 inhibitor	Phase 1	https://conductscience.org/library/genes/8841	Drugs and targets
HDAC6	Protein deacetylase HDAC6	1	Histone deacetylase 6 inhibitor	Phase 1	https://conductscience.org/library/genes/10013	Drugs and targets

Trials4

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
NCT05025332	An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)	Phase 2	28	2025-05-18	Pitt Hopkins Syndrome	NNZ-2591	Its record names the disease	ClinicalTrials.gov
NCT04132427	MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders	Phase 2	6	2024-08-26	Pitt Hopkins Syndrome	VANCOMYCIN	Its record names the disease	ClinicalTrials.gov
NCT06321796	Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders	Phase 2	20		Pitt Hopkins Syndrome	VANCOMYCIN	Its record names the disease	ClinicalTrials.gov
NCT07150026	An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome	Phase 1	5		Pitt Hopkins Syndrome	VORINOSTAT	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials2

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
Group A: Treatment vs Group B: Placebo: deaths from any cause	0 of 3	0 of 3	6	NCT04132427	Trial comparisons
Group A: Treatment vs Group B: Placebo: serious adverse events	0 of 3	0 of 3	6	NCT04132427	Trial comparisons

Papers from its trials0

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

None in this release.

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0012589

GET https://conductscience.org/api/diseases/v1/MONDO_0012589/drugs

API reference For AI assistants