DrugEvidence

Diseases / Retinitis Pigmentosa

Retinitis Pigmentosa Clinical Trials

Disease

Retinitis pigmentosa is a group of rare inherited retinal diseases that progressively damage photoreceptor cells and lead to vision loss. Symptoms often begin with night blindness and then progressive peripheral field loss. Over time, vision can narrow substantially and may progress to severe impairment.

Trials
266
Recruiting
39
Completed
115
With results
35

Continuously tracked·last revised

Data: CSV·JSON·Browse the trials

Follow Retinitis Pigmentosa

Recruiting Phase 3

Retinitis Pigmentosa — Recruiting Phase 3
TrialPhaseStatusSponsorStarted
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa NCT06628947 Phase 2 Recruiting Kiora Pharmaceuticals, Inc. Started 2025-08

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Recently started

Retinitis Pigmentosa — Recently started
TrialPhaseStatusSponsorStarted
A Study of the Safety and Preliminary Efficacy of UGX202 in Patients With Advanced Retinitis Pigmentosa NCT07847723 Phase 1 Not yet recruiting Suzhou UgeneX Therapeutics Co., Ltd. Started 2026-09
Safety Evaluation Study of Pitavastatin PLGA Nano-particle for Retinitis Pigmentosa NCT07774975 Phase 1 Not yet recruiting Kyushu University Started 2026-09
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome NCT07710196 Phase 3 Not yet recruiting Nacuity Pharmaceuticals, Inc. Started 2026-08
9-cis Beta-Carotene-Rich Extract of Dunaliella Alga in Retinitis Pigmentosa Patients NCT07509229 Phase 1/2 Enrolling by invitation Sheba Medical Center Started 2026-07
Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment NCT07341763 Phase n/a Recruiting University of Waterloo Started 2026-07

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Results recently posted

Retinitis Pigmentosa — Results recently posted
TrialPhaseStatusSponsorResults posted
A Study in Subjects With Retinitis Pigmentosa NCT05392179 Phase 2 Completed Aldeyra Therapeutics, Inc. Results posted 2026-04-23
Sildenafil for Treatment of Choroidal Ischemia NCT04356716 Phase 2 Completed Columbia University Results posted 2025-07-04
A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa NCT00447980 Phase 2 Completed Neurotech Pharmaceuticals Results posted 2025-05-01
Environmental Localization Mapping and Guidance for Visual Prosthesis Users NCT04359108 Phase n/a Completed Johns Hopkins University Results posted 2025-03-10
Advancing Understanding of Transportation Options NCT04141891 Phase n/a Completed University of Colorado, Denver Results posted 2025-02-21

See all 35 →

Pipeline

Phase by status

Retinitis Pigmentosa — Phase by status
PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnSuspendedUnknownAvailableTotal
Early Phase 1 11——————4— 6
Phase 1 42—5121——1— 25
Phase 1/2 9218134——4— 41
Phase 2 11—2161——4— 25
Phase 2/3 —1———2——1— 4
Phase 3 —3—461———— 14
Phase 4 ————1————— 1
Not applicable 82123335—9— 63
Unknown 1643734611141 87

Trials started per year

2021
5
2022
11
2023
13
2024
14
2025
17
2026
19
2027
1

Conditions studied, by phase

Retinitis Pigmentosa — Conditions studied, by phase
ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Retinitis Pigmentosa 625412541416387 266
Retinal Degeneration 1372———614 33
Retinal Diseases —36821—49 33
Eye Diseases —2852——47 28
Usher Syndromes ——4423—112 26
Macular Degeneration —311———911 25
Retinal Dystrophies —141———310 19
Cone-Rod Dystrophies ——51———84 18
Eye Diseases, Hereditary ——442——15 16
Vision Disorders ——332——62 16

Most active sponsors

Retinitis Pigmentosa — Most active sponsors
#SponsorTrialsPhase 3
1National Eye Institute (NEI)132
2Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts90
3Johns Hopkins University81
4Second Sight Medical Products60
5Sheba Medical Center60
6Ankara Universitesi Teknokent52
7Laboratoires Thea52
8Nanoscope Therapeutics Inc.50
9Okuvision GmbH50
10Retina Implant AG50

Most studied interventions

Retinitis Pigmentosa — Most studied interventions
#InterventionTrials
1salicylhydroxamic acid5
2Intravitreal Injections4
3Ciliary Neurotrophic Factor3
4Docosahexaenoic Acids3
5Minocycline3
6retinol acetate3
7Vitamin A3
8Acetylcysteine2
9cenegermin2
10Culture Media, Conditioned2

Terminations

Terminated
18
Withdrawn
6
Suspended
1
Stopped for recruitment
1.1%3 of 266 trials

Stated reasons

Other / unclear
9
Business decision
8
Recruitment
3
Regulatory
2
Lack of efficacy
1
Funding
1
Safety
1

Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.

Recently stopped

Retinitis Pigmentosa — Recently stopped
TrialStatusStated reasonStopped
NCT03328130 Terminated Terminated lack of financing 2025-06-30
NCT04558983 Terminated Terminated The study stopped because patients dropped out of the study to participate in the interventional trials. With interventional trials present there is very little interest in participating in a Natural Histoy study. 2024-12-20
NCT04120883 Terminated Terminated Unable to recruit additional participants after multiple extensions to planned enrollment period. 2024-08-05
NCT05392751 Terminated Terminated Study terminated by the Sponsor. All study participants completed at least 12 months of safety follow-up instead of 24 mo. The reason for earlier termination was due to a corporate decision; not any safety concerns of EA-2353. 2024-04-24
NCT04295304 Terminated Terminated Lack of funds 2023-03-12
NCT05085964 Terminated Terminated The sponsor decided to terminate the study early 2022-10-18
NCT05158296 Terminated Terminated Business decision 2022-10-12
NCT05176717 Terminated Terminated Business decision 2022-08-02
NCT01490827 Terminated Terminated Sponsor discontinued the Argus II product 2020-03-31
NCT01860092 Terminated Terminated Manufacturing ceased for all Argus II and Argus 2s devices (FDA notified of and subsequently approved discontinuation of post approval study in Oct 2021) 2020-03-31

Evidence

Results reported
30.4%35 of 115 completed
Published
29.6%34 of 115 completed
Primary endpoint met
57.8%26 of 45 analyses
Median months to report
17

Documents disclosed

Protocol available
29
Statistical analysis plan
21
PubMed citations
77

Trial profile

Median enrollment
30
Median duration
36months
Countries
43
Study sites
571

Who is enrolled

Adults
266
Children
77
Older adults
241
Healthy volunteers
37

Median enrollment by phase

Early Phase 1
21
Phase 1
15
Phase 1/2
19
Phase 2
45
Phase 2/3
19
Phase 3
80
Phase 4
40
Not applicable
21
Unknown
52

Where trials run

United States
110
France
37
Germany
30
United Kingdom
24
Canada
15
China
15
Israel
12
Italy
12
Netherlands
11
Australia
10

Busiest cities

Retinitis Pigmentosa — Busiest cities
#CityCountryTrials
1ParisFrance29
2DallasUnited States26
3TübingenGermany21
4BaltimoreUnited States20
5BostonUnited States20
6LondonUnited Kingdom20
7PortlandUnited States19
8Ann ArborUnited States18
9MiamiUnited States16
10PhiladelphiaUnited States14