Clinical trials

Development of a central repository for PD-related genomic data for future research.

Investigator

Irene A Malaty

Ages

18 Years - N/A

Sexes

All

The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. Note: Due to…

Investigator

Thomas J George

Ages

12 Years - N/A

Sexes

All

This phase III trial uses the Decipher risk score to guide intensification (for higher Decipher gene risk) or de-intensification (for low Decipher gene risk) of treatment to better match therapies to an individual patient's cancer aggressiveness.…

Investigator

Brian H Ramnaraign

Ages

18 Years - N/A

Sexes

Male

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the…

Investigator

Carmen Leon Astudillo

Ages

N/A - N/A

Sexes

Male

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Investigator

Barry J Byrne

Ages

2 Years - 3 Years

Sexes

Male

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with…

Investigator

Carmen Leon Astudillo

Ages

4 Years - 7 Years

Sexes

Male

This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria…

Investigator

Roberto T Zori

Ages

18 Years - 65 Years

Sexes

All