90. 網膜色素変性症 Retinitis pigmentosa Clinical trials / Disease details


臨床試験数 : 147 薬物数 : 176 - (DrugBank : 43) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 110

  
2 trials found
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PhaseCountries
1NCT03374657
(ClinicalTrials.gov)
August 22, 201811/12/2017A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis PigmentosaAn Open-label First-in-human Single Ascending Dose Study to Explore Safety, Tolerability and Efficacy of Subretinal Administration of CPK850 Gene Therapy in Patients With Retinitis Pigmentosa Due to Mutations in the Retinaldehyde Binding Protein 1 (RLBP1) GeneRetinitis PigmentosaBiological: CPK850Novartis PharmaceuticalsNULLActive, not recruiting18 Years70 YearsAll12Phase 1/Phase 2Sweden
2EUCTR2016-002696-10-SE
(EUCTR)
09/05/201821/12/2017Explore the safety and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 geneAn open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Code: CPK850
INN or Proposed INN: TBD
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 1;Phase 2Sweden