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Found 173 clinical trials
Featured trial
Family and Caregiver Perspective on Gene Therapy for Rett Syndrome

>The purpose of this study is to assess the attitudes and opinions of parents, siblings, caregivers, and legal guardians of individuals with Rett syndrome about gene therapy. The study is

Online studies
  • 23 Aug, 2022
  • 1 location
  • Online study
Featured trial
Safety and Efficacy of TSHA-102 in Adult Females With Rett Syndrome (REVEAL Adult Study)

The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety

  • 17 Nov, 2022
  • 1 location
Featured trial
HERV-E TCR Transduced Autologous T Cells in People With Metastatic Clear Cell Renal Cell Carcinoma

Gene transfer is a new cancer therapy takes white blood cells from a person and grows them in a lab. The cells are changed with a virus to attack tumor cells, then returned to the person. Researchers want to see if this therapy fights kidney cancer cells. Metastatic renal cell …

metastatic cancer
clear cell renal cell carcinoma
renal cell cancer
clear cell carcinoma
  • 22 Dec, 2020
  • 1 location
A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy) (GALax-C)

PBKR03 is a gene therapy for Krabbe Disease (Globoid cell leukodystrophy) intended to deliver a functional copy of the GALC gene to the brain and peripheral tissues. This study will evaluate the

  • 22 Oct, 2022
  • 8 locations
PDE6A Gene Therapy for Retinitis Pigmentosa

The PDE6A gene encodes a subunit of the rod phosphodiesterase. The loss of this enzyme function leads to a chronically elevated cGMP level which causes an increased calcium inflow into the cell and thereby the hyperactivation of cell death pathways. The goal of the PIGMENT study is to develop, produce …

other disease
gene therapy
  • 26 Jan, 2021
  • 1 location
Gene Therapy for Pyruvate Kinase Deficiency (PKD)

This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).

  • 17 Feb, 2022
  • 3 locations
Gene Therapy Clinical Study in Adult PKU (pheNIX)

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.

gene therapy
pah gene
  • 09 Feb, 2022
  • 12 locations
Gene Therapy for Severe Crigler Najjar Syndrome (CareCN)

approximately 12 months (48 weeks) a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene

  • 28 Feb, 2022
  • 4 locations
GNT0006 Gene Therapy Trial in Patients With LGMDR9

Phase 1-2 study including a dose escalation safety and proof of concept phase (Stage 1, open label), followed by a double-blind, randomized, placebo-controlled confirmatory phase (Stage 2)

  • 04 Oct, 2022
  • 2 locations
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

have much worse overall outcomes from HSCT. This study will investigate whether patients with SCID-X1 without a fully matched related donor may benefit from gene therapy. To do this the

  • 03 Feb, 2022
  • 1 location