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Found 168 clinical trials
Featured trial
An MRI-Study to Characterize Cerebrospinal Fluid Geometry and Dynamics in Individuals with Rett Syndrome

Knowledge of the CSF geometry and dynamics in RTT will allow for customized intrathecal (IT) drug delivery for subjects with RTT and may help improve gene therapy distribution and exposure

  • 15 Feb, 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 carcinoma
renal cell cancer
primary cancer
  • 22 Dec, 2020
  • 1 location
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease (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

  • 30 Apr, 2022
  • 3 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
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 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
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
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
Gene Therapy Study for Children With CLN5 Batten Disease (CLN5-200)

This is a prospective, non-randomized, open-label study of a single dose administration of gene therapy in children who are 3 to 8 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype

  • 10 Mar, 2022
  • 1 location
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

  • 26 Jan, 2022
  • 8 locations