Disease · MONDO_0019200

retinitis pigmentosa

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. retinitis pigmentosa. Diseases, record MONDO_0019200. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019200

On this page

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: retinitis pigmentosa; Open Targets
Open Targets ID: MONDO_0019200; Open Targets
Other names: Rod cone dystrophy; Open Targets
Disease Ontology: DOID:10584; Disease Ontology
MeSH: D012174; MeSH
NCI Thesaurus: C85045; NCI Thesaurus
Orphanet: ORPHA:791; Orphanet
Highest stage of its drugs: Approval, for 1 of its 31 drugs, from EMA Human Drugs; Open Targets

Drugs31

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
VORETIGENE NEPARVOVEC	CHEMBL3707275	Gene	Approval	EMA Human Drugs	2	0	0	Drugs and targets
ACETYLCYSTEINE	CHEMBL600	Small molecule	Phase 3	ClinicalTrials.gov	4	4	1	Drugs and targets
MINOCYCLINE	CHEMBL1434	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
RETINOL	CHEMBL986	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
UNOPROSTONE ISOPROPYL	CHEMBL1200661	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
VITAMIN E	CHEMBL3989727		Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
CILIARY NEUROTROPHIC FACTOR	CHEMBL4298063		Phase 2/3	TTD	5	4	4	Drugs and targets
ZURETINOL ACETATE	CHEMBL4297637	Small molecule	Phase 2/3	ClinicalTrials.gov	5	4	4	Drugs and targets
ULTEVURSEN	CHEMBL5095297	Oligonucleotide	Phase 2/3	ClinicalTrials.gov	3	3	3	Drugs and targets
LARUPARETIGENE ZOVAPARVOVEC	CHEMBL5095428	Gene	Phase 2	ClinicalTrials.gov	4	3	1	Drugs and targets

1 to 10 of 31

See all 31 drugs

Targets17

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
ADRA2A	Adrenergic receptor alpha-2	1	Adrenergic receptor alpha-2 agonist	Phase 1/2	https://conductscience.org/library/genes/150	Drugs and targets
ADRA2B	Adrenergic receptor alpha-2	1	Adrenergic receptor alpha-2 agonist	Phase 1/2	https://conductscience.org/library/genes/151	Drugs and targets
ADRA2C	Adrenergic receptor alpha-2	1	Adrenergic receptor alpha-2 agonist	Phase 1/2	https://conductscience.org/library/genes/152	Drugs and targets
ALDH5A1	Succinate-semialdehyde dehydrogenase, mitochondrial	1	Succinate semialdehyde dehydrogenase inhibitor	Phase 2	https://conductscience.org/library/genes/7915	Drugs and targets
DHFR	Dihydrofolate reductase	1	Dihydrofolate reductase inhibitor	Phase 2	https://conductscience.org/library/genes/1719	Drugs and targets
DRD3	D(3) dopamine receptor	1	Dopamine D3 receptor agonist	Phase 2	https://conductscience.org/library/genes/1814	Drugs and targets
NTRK1	Neurotrophic tyrosine kinase receptor	1	Neurotrophic tyrosine kinase receptor agonist	Phase 1/2	https://conductscience.org/library/genes/4914	Drugs and targets
NTRK2	Neurotrophic tyrosine kinase receptor	1	Neurotrophic tyrosine kinase receptor agonist	Phase 1/2	https://conductscience.org/library/genes/4915	Drugs and targets
NTRK3	Neurotrophic tyrosine kinase receptor	1	Neurotrophic tyrosine kinase receptor agonist	Phase 1/2	https://conductscience.org/library/genes/4916	Drugs and targets
PDE5A	cGMP-specific 3',5'-cyclic phosphodiesterase	1	Phosphodiesterase 5A inhibitor	Phase 2	https://conductscience.org/library/genes/8654	Drugs and targets

1 to 10 of 17

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Trials39

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. 6 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01233609	Trial of Oral Valproic Acid for Retinitis Pigmentosa	Phase 2	90	2017-12-02	Retinitis Pigmentosa	VALPROIC ACID	Its record names the disease	ClinicalTrials.gov
NCT00100230	DHA and X-Linked Retinitis Pigmentosa	Phase 2	78	2015-03-17	Retinitis Pigmentosa, X-linked Genetic Diseases	DOCONEXENT	Its record names the disease	ClinicalTrials.gov
NCT00447980	A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa	Phase 2	73	2025-05-01	Retinitis Pigmentosa	CILIARY NEUROTROPHIC FACTOR	Its record names the disease	ClinicalTrials.gov
NCT00447993	A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa	Phase 2	67	2024-08-13	Retinitis Pigmentosa	CILIARY NEUROTROPHIC FACTOR	Its record names the disease	ClinicalTrials.gov
NCT02110225	A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)	Phase 1/2	50	2019-07-11	Retinitis Pigmentosa	CENEGERMIN	Its record names the disease	ClinicalTrials.gov
NCT02661711	Aflibercept for Macular Oedema With Underlying Retinitis Pigmentosa (AMOUR) Study	Phase 2	30	2024-05-06	Macular Oedema, Retinitis Pigmentosa	AFLIBERCEPT	Its record names the disease	ClinicalTrials.gov
NCT01530659	Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa	Phase 2	22	2023-02-14	Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3	CILIARY NEUROTROPHIC FACTOR	Its record names the disease	ClinicalTrials.gov
NCT04356716	Sildenafil for Treatment of Choroidal Ischemia	Phase 2	22	2025-07-04	Choroidal Ischemia, Vitelliform Macular Dystrophy, Age-related Macular Degeneration, Central Serous Retinopathy, Retinitis Pigmentosa	SILDENAFIL	Its record names the disease	ClinicalTrials.gov
NCT00661479	An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis Pigmentosa	Phase 1/2	21	2013-04-24	Retinitis Pigmentosa	BRIMONIDINE TARTRATE	Its record names the disease	ClinicalTrials.gov
NCT05085964	An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa	Phase 2	21	2022-12-27	Retinitis Pigmentosa, Usher Syndrome Type 2	ULTEVURSEN	Its record names the disease	ClinicalTrials.gov

1 to 10 of 39

See all 39 trials

Comparisons in its trials7

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
Valproic Acid vs Placebo: deaths from any cause	1 of 46	0 of 44	90	NCT01233609	Trial comparisons
Valproic Acid vs Placebo: serious adverse events	3 of 46	2 of 44	90	NCT01233609	Trial comparisons
Low Dose vs High Dose: deaths from any cause	0 of 20	0 of 48	68	NCT00447980	Trial comparisons
Low Dose vs High Dose: serious adverse events	1 of 20	4 of 48	68	NCT00447980	Trial comparisons
Low Dose vs High Dose: deaths from any cause	0 of 22	0 of 43	65	NCT00447993	Trial comparisons
1. Docosahexaenoic Acid (DHA) Arm vs Corn/Soy Oil Placebo Arm: serious adverse events	0 of 33	0 of 27	60	NCT00100230	Trial comparisons
Low Dose vs High Dose: serious adverse events	1 of 22	0 of 22	44	NCT00447993	Trial comparisons

Papers from its trials22

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
36882562	RESULT	1	PubMed
25015354	RESULT	1	PubMed
24805262	RESULT	1	PubMed
41981307	DERIVED	1	PubMed
40339601	DERIVED	1	PubMed
39627217	DERIVED	1	PubMed
37261916	DERIVED	1	PubMed
32573764	DERIVED	1	PubMed
32041720	DERIVED	1	PubMed
31805012	DERIVED	1	PubMed

1 to 10 of 22

See all 22 papers from its trials

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_0019200

GET https://conductscience.org/api/diseases/v1/MONDO_0019200/drugs?page=2

API reference For AI assistants