AAV2/5-hRKp.RPGR ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
90網膜色素変性症2

90. 網膜色素変性症


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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-000425-31-GB
(EUCTR)
30/08/201814/06/2018Long term follow-up study of gene therapy trial for X-linked Retinitis PigmentosaLong term follow-up study of participants following an open label, multicentre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Long term follow-up gene therapy study for X-linked Retinitis Pigmentosa X-Linked Retinitis Pigmentosa caused by mutations in the RPGR 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 Name: AAV2/5-hRKp.RPGRMeiraGTx UK II LtdNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
36 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
2EUCTR2016-003967-21-GB
(EUCTR)
09/06/201714/09/2017Gene Therapy Trial for People with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Gene Therapy Trial for People with Retinitis Pigmentosa: RPGR X-Linked Retinitis Pigmentosa caused by mutations in the RPGR 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 Name: AAV2/5-hRKp.RPGR
INN or Proposed INN: AAV2/5-hRKp.RPGR
MeiraGTx UK II LimitedNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
71 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom