Clinical trials: Pigmentary Retinopathy
Bardet-Biedl Syndrome, POMC Deficiency
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.
- Trial name
- NCT05194124
- Testing
- Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg
- Where
- 7 sites in United States, Canada and 4 more
- Volunteers
- 19 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Retinitis Pigmentosa
Post-Market Study of the Argus® II Retinal Prosthesis System - France
This is a post-market study of the Argus II Retinal Prosthesis System. The study is being conducted in France. The objective of the study is to obtain data to further demonstrate the effectiveness and evaluate the safety of Argus…
- Trial name
- NCT02303288
- Testing
- Argus II Retinal Prosthesis System
- Where
- 3 sites in France
- Volunteers
- 18 people
- Sponsor
- Second Sight Medical Products
Retinitis Pigmentosa, Retinal Diseases
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
The purpose of the study is to evaluate the safety and tolerability of a single intravitreal injection of virally-carried Multi-Characteristic Opsin I (vMCO-I)
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration
- Trial name
- NCT04919473
- Testing
- Gene Therapy product:vMCO-I
- Where
- India
- Volunteers
- 11 people
- Sponsor
- Nanoscope Therapeutics Inc.
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- LUNA (NCT06627179)
- Testing
- Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Usher Syndrome, Retinitis Pigmentosa
Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
To evaluate the safety and tolerability of ascending doses of subretinal injections of SAR421869 in participants with Usher syndrome type 1B. To evaluate for possible biological activity of SAR421869.
- Trial name
- NCT01505062
- Testing
- SAR421869
- Where
- 2 sites in United States, France
- Volunteers
- 9 people
- Sponsor
- Sanofi
Retinitis Pigmentosa, Eye Diseases, Hereditary
Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11.
- Conditions
- Retinitis Pigmentosa, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Dystrophy Rod
- Trial name
- NCT05573984
- Where
- 7 sites in United States, Australia
- Volunteers
- 50 people
- Sponsor
- PYC Therapeutics
Retinitis Pigmentosa, Choroideremia
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
A phase I/II dose-escalating study of the safety, tolerability and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia (ABACUS).
- Conditions
- Retinitis Pigmentosa, Choroideremia, Non-Syndromic Rod-Dominant Inherited Retinal Diseases
- Trial name
- NCT05282953
- Testing
- KIO-301
- Where
- 3 sites in Australia
- Volunteers
- 32 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
Retinitis Pigmentosa
Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa
The primary objective of the study is to assess the safety of repeat injection of human retinal progenitor cells (jCell) in adult subjects with RP that have previously been treated with jCell.
- Trial name
- NCT04604899
- Testing
- human retinal progenitor cells
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- jCyte, Inc
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
- Trial name
- NCT06333249
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Leber Congenital Amaurosis, Retinitis Pigmentosa
Treatment of RP and LCA by Primary RPE Transplantation
Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients
- Trial name
- NCT03566147
- Testing
- Human primary Retinal Pigment Epithelial (HuRPE) cells
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Eyecure Therapeutics Inc.
Retinitis Pigmentosa
Long-term Safety of UC-MSC Transplantation in Patients With Retinitis Pigmentosa
The study will perform to follow-up UC-MSCs and CM transplantation. 18 patients will be called back to be examined after 5 years of UC-MSC and/or CM transplantation.
- Trial name
- NCT05786287
- Testing
- Conditioned Medium (CM), Umbilical Cord Mesenchymal Stem Cell (UC-MSC)
- Where
- 2 sites in Indonesia
- Volunteers
- 18 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
X-Linked Retinitis Pigmentosa
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
- Trial name
- NCT04312672
- Testing
- AAV5-hRKp.RPGR
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 42 people
- Sponsor
- MeiraGTx Ocular UK Ltd
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Retinitis Pigmentosa
Development of Visual Function Evaluation Method (2)
The objective of this study is to develop the comprehensive visual function evaluation method in severe visually impaired patient.
- Trial name
- NCT03626207
- Where
- Japan
- Volunteers
- 8 people
- Sponsor
- Astellas Pharma Inc