DDrare: Database of Drug Development for Rare Diseases

90. 網膜色素変性症
［臨床試験数：103，薬物数：158（DrugBank：42），標的遺伝子数：52，標的パスウェイ数：107］

Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 100 µg Brimonidine Tartrate Implant; 200 µg Brimonidine Tartrate Implant; 400 µg Brimonidine Tartrate Implant; 4D-125 IVT Injection; 9715X; 9741X; 9742X; AAV-RPGR; AAV2-REP1; AAV2/5-hPDE6B; AAV2/5-hRKp.RPGR; AAV8-RPGR; ALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED; Acetate; Acetazolamide; Aflibercept; Alga Dunaliella Bardawil; Alga Dunaliella Bardawil powder; Alga Dunaliella Bardawill; Antioxidants; BMMNCs; BS01; Betamethasone; Brimonidine; Brimonidine Tartrate; Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System; CD34+ bone marrow stem cells intravitreal; CIRCADIN 2 mg comprimidos de liberación prolongada; CNS10-NPC implantation; CNTO 2476; COQUN; CPK850; Cannabis; Carbidopa; Cholesterol; Ciliary Neurotrophic Factor Implant NT-501; Ciliary neurotrophic factor; Circadin; Circadin 2 mg; Combination Product: PRP combined Magnovision; Compound thrombosis capsule sig: 1.5g/tid; Conditioned Medium (CM); Continuous oxygen; Cornstarch; Cornstarch control; Cyclosporine; Cysteine; DAC; DDS; Device: Medmont Darl Adapted Chromatic (DAC); Device: MonCvONE electrophysiology; Device: RetMap, RM Electrode; Device: blue light-absorbing sunglasses; Device: dexamethasone injection; Dexamethasone; Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A); Docosahexaenoic acid; Dorzolamide; Eylea; Fluocinolone; Fluocinolone Acetonide; Fluocinolone acetonide; GS030-DP; Ginkgo; Ginkgo biloba; Ginkgo biloba pills sig: 300mg/tid; HORA-PDE6B (AAV2/5.hPDE6B); HRPC; HRPC DP; Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE); Human Retinal Progenitor Cells Drug Product; Human primary Retinal Pigment Epithelial (HuRPE) cells; Human retinal progenitor cells; Hydroxychloroquine; Hydroxychloroquine higher dose; Hydroxychloroquine lower dose; Iluvien; Intravitreal injection of Autologous bone marrow Stem Cell; Intravitreal injection of autologous bone marrow stem cells; Lentiviral vector containing MYO7A gene; Lentiviral vector containing the human MY07A gene; Levodopa; Levodopa-carbidopa; Lutein; Lutein (10 or 30 mg/day) capsules; Lycium Barbarum; MELATONIN; Melatonin; Minocycline; N-Acetyl Cysteine (NAC); NAC; NAC effervescent tablets; NPI-001; NT-501; Nerve Growt Factor - 2.5S; Nerve Growth Factor; Nilvadipine; Null; Nutritional Supplement; Other: Mock injection; Other: Observational; Other: Sham (no implant); Other: Sham procedure; Other: Sham-procedure (dose cohort 1&2 only); Other: Standard of Care Sildenafil; Other: Subconjunctival injection of saline; Other: Sugar pill; Other: Visual Acuity (VA); Oxygen; PRP; PS; Phosphate; Platelet rich plasma; Procedure: Sham; Procedure: hyperbaric oxygen therapy; QLT091001; QR-1123; QR-421a; RAAV.hPDE6A vector; RAAV2tYF-GRK1-RPGR; RECOMBINANT HUMAN NERVE GROWTH FACTOR (RHNGF); RST-001; Recombinant adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene; Recombinant human Nerve Growth Factor (rhNGF); Retinal pigment epitheliums transplantation; RhNGF; RhNGF 180 µg/ml eye drops solution; RhNGF 60 µg/ml eye drops solution; SAR421869; Sildenafil; Sodium valproate; Stem Cell Transplantation; Stem/progenitor cells transplantation; Subretinal injection of rAAV.hPDE6A; Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP); Timrepigene emparvovec; Tocoferol; Triamcinolone; Twice-daily dosage with 0.5% cyclosporine-A eyedrops; UF-021; UF-021 (isopropyl unoprostone); UF-021(isopropyl unoprostone); Umbilical Cord Mesenchymal Stem Cell (UC-MSC); Unoprostone; UshStat; UshStat (SAR421869); VA; Valproate; Valproic Acid; Valproic acid; Vehicle eye drops; Vitamin A; Vitamin AD sig: 1 tablet/tid; Vitamin B sig: 10mg/tid; Vitamin E; Wharton's jelly derived mesenchymal stem cell; Zuretinol Acetate; Zuretinol acetate

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02609165 (ClinicalTrials.gov)	May 2015	12/10/2015	Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema	Nerve Growth Factor Eye Drops as a Novel Treatment for Vision Loss in Patients With Retinitis Pigmentosa: From Preclinical to Clinical Phase II Trial	Retinitis Pigmentosa;Cystoid Macular Edema	Drug: rhNGF 180 µg/ml eye drops solution;Drug: vehicle eye drops	Ospedale San Raffaele	Dompé Farmaceutici S.p.A	Completed	18 Years	N/A	All	45	Phase 2	Italy
2	NCT02110225 (ClinicalTrials.gov)	January 2014	8/4/2014	A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)	A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP.	Retinitis Pigmentosa	Drug: rhNGF 60 µg/ml eye drops solution;Drug: rhNGF 180 µg/ml eye drops solution;Drug: Placebo	Dompé Farmaceutici S.p.A	NULL	Completed	18 Years	N/A	All	50	Phase 1;Phase 2	Italy

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT02609165
(ClinicalTrials.gov)

May 2015

12/10/2015

Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema

Nerve Growth Factor Eye Drops as a Novel Treatment for Vision Loss in Patients With Retinitis Pigmentosa: From Preclinical to Clinical Phase II Trial

Retinitis Pigmentosa;Cystoid Macular Edema

Drug: rhNGF 180 µg/ml eye drops solution;Drug: vehicle eye drops

Ospedale San Raffaele

Dompé Farmaceutici S.p.A

Completed

18 Years

N/A

All

Phase 2

Italy

NCT02110225
(ClinicalTrials.gov)

January 2014

8/4/2014

A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)

A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP.

Retinitis Pigmentosa

Drug: rhNGF 60 µg/ml eye drops solution;Drug: rhNGF 180 µg/ml eye drops solution;Drug: Placebo

Dompé Farmaceutici S.p.A

NULL

Completed

18 Years

N/A

All

Phase 1;Phase 2

Italy