Development of a central repository for PD-related genomic data for future research.
- Investigator
- Irene A Malaty
- Ages
- 18 Years - N/A
- Sexes
- All
Clinical trials
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PD GENE -
DoD- OA Gene Therapy -
Gene Expression Biomarkers of Wound Pain and Wound Healing -
TAPUR: Testing FDA Approved Drugs That Target Specific Abnormal Tumor Genes in Pts w/Advanced Cancer 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鈥�
- Investigator
- Thomas J George
- Ages
- 12 Years - N/A
- Sexes
- All
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Genomic-Risk Stratified Txs for Unfavorable Intermediate Risk Prostate Cancer (Guidance) This phase III trial uses the Decipher risk score to guide therapy selection. Decipher score is based on the activity of 22 genes in prostate tumor and may predict how likely it is for recurrent prostate cancer to spread (metastasize) to other parts鈥�
- Investigator
- Jonathan A Chatzkel
- Ages
- 18 Years - N/A
- Sexes
- Male
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9001-303 (ENVISION) 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鈥�
- Investigator
- Carmen Leon Astudillo
- Ages
- N/A - N/A
- Sexes
- Male
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Pfizer Early Stage GT 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
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EMBARK 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鈥�
- Investigator
- Carmen Leon Astudillo
- Ages
- 4 Years - 7 Years
- Sexes
- Male
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4D-710-C001 This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Investigator
- Cesar A Trillo-Alvarez
- Ages
- 18 Years - N/A
- Sexes
- All
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DFI17545 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鈥�
- Investigator
- Roberto T Zori
- Ages
- 18 Years - 65 Years
- Sexes
- All