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Conditions: Choroideremia
Interventions: 4D-110; Biological
Lead sponsor: 4D Molecular Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 2
States / cities: Dallas, Texas • Salt Lake City, Utah

Conditions: Metastatic Melanoma
Interventions: F5 TCR transgenic cells and MART-1 peptide pulsed dendritic cells, non-myeloablative conditioning chemotherapy; Biological · Drug
Lead sponsor: Jonsson Comprehensive Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2019
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Intermittent Claudication, Peripheral Vascular Disease, Atherosclerosis
Interventions: Ad2/HIF-1α/VP16, Saline (Placebo Control); Biological · Other
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 40 Years to 80 Years
Enrollment: 289 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2010
U.S. locations: 35
States / cities: Birmingham, Alabama • Palo Alto, California • Sacramento, California + 28 more

Conditions: Multiple Myeloma
Interventions: Stemgen; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Alzheimer Disease
Interventions: LX1001; Biological
Lead sponsor: Lexeo Therapeutics; Industry
Eligibility: 50 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 2
States / cities: Orlando, Florida • Durham, North Carolina

Conditions: Fanconi Anemia Complementation Group A
Interventions: RP-L102; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 1 Year and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 2
States / cities: Stanford, California • Minneapolis, Minnesota

Conditions: Frontotemporal Dementia, FTD, FTD-GRN, Dementia, Frontotemporal
Interventions: Intrathalamic AAV.PGRN administration, Intrathalamic AVB-101; Procedure · Genetic
Lead sponsor: AviadoBio Ltd; Industry
Eligibility: 30 Years to 75 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 3
States / cities: Columbus, Ohio • Nashville, Tennessee • Houston, Texas

Conditions: Hematologic Neoplasm, HIV Infection
Interventions: GCSF Mobilized Allogeneic PBSC Cultured w/Cytokines; Transduced w/RV; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hemophilia B
Interventions: PF-06838435 (formerly SPK-9001); Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 17
States / cities: Sacramento, California • Metairie, Louisiana • New Orleans, Louisiana + 4 more

Conditions: Beta-Thalassemia
Interventions: LentiGlobin BB305 Drug Product; Genetic
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: 0 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania

Conditions: Retinoschisis, X-Linked
Interventions: Not listed
Lead sponsor: National Eye Institute (NEI); NIH
Eligibility: 18 Years and older · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Acute Coronary Syndrome
Interventions: dalcetrapib, Placebo; Drug
Lead sponsor: DalCor Pharmaceuticals; Industry
Eligibility: 45 Years and older
Enrollment: 6,147 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 122
States / cities: Alexander City, Alabama • Huntsville, Alabama • Mobile, Alabama + 119 more

Conditions: Pyruvate Kinase Deficiency
Interventions: RP-L301; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years to 55 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Prostate Cancer, Liver Cancer, Breast Cancer, Colon Cancer, Lung Cancer
Interventions: Not listed
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 750 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: Started 2004
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Malignant Glioma, Recurrent Ependymoma
Interventions: AdV-tk, valacyclovir, Radiation; Biological · Drug · Radiation
Lead sponsor: Candel Therapeutics, Inc.; Industry
Eligibility: 3 Years to 21 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2021
U.S. locations: 2
States / cities: Chicago, Illinois • Boston, Massachusetts

Conditions: Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
Interventions: Not listed
Lead sponsor: Spur Therapeutics; Industry
Eligibility: 16 Years and older · Male only
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 17
States / cities: Phoenix, Arizona • Los Angeles, California • Sacramento, California + 14 more

Conditions: Open Angle Glaucoma (OAG), NAION( Non-arteritic Anterior Ischemic Optic Neuropathy)
Interventions: ER-100 epigenetic therapy; Genetic
Lead sponsor: Life Biosciences Inc.; Industry
Eligibility: 40 Years to 85 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 4
States / cities: Glendale, California • Boston, Massachusetts • New York, New York + 1 more

Conditions: Muscular Dystrophy, Duchenne
Interventions: delandistrogene moxeparvovec, placebo; Genetic
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 4 Years to 7 Years · Male only
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 2
States / cities: Los Angeles, California • Columbus, Ohio

Conditions: X-linked Severe Combined Immunodeficiency (XSCID)
Interventions: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 2 Years to 50 Years · Male only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2032
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Fanconi Anemia
Interventions: Retrovirus Construct; Genetic
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 1 Year to 35 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2007
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Solid Tumor, Adult, Colorectal Cancer, Non-Small Cell Lung, NSCLC (Non-small Cell Lung Cancer), Cancer, Colon Cancer, Rectal Cancer, Lung Cancer, CRC, Head and Neck Squamous Cell Cancer, HNSCC, Renal Cell Carcinoma, RCC, Kidney Cancer, Triple Negative Breast Cancer, TNBC, Colorectal Adenocarcinoma
Interventions: A2B395, xT CDx with HLA-LOH assay; Biological · Diagnostic Test
Lead sponsor: A2 Biotherapeutics Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 240 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 10
States / cities: Gilbert, Arizona • La Jolla, California • Los Angeles, California + 7 more

Conditions: Ovarian Cancer
Interventions: MOv-gamma chimeric receptor gene, aldesleukin, therapeutic allogeneic lymphocytes; Biological
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older · Female only
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1997
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: CD40L-HyperIgM Syndrome
Interventions: Base-edited hematopoietic stem and progenitor cells, Alemtuzumab, Sirolimus, Palifermin, Busulfan, Base-edited T lymphocyte cells; Biological · Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 37 Years to 120 Years · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Bone Marrow Suppression, Brain and Central Nervous System Tumors, Drug/Agent Toxicity by Tissue/Organ
Interventions: filgrastim, gene therapy, lomustine, procarbazine hydrochloride, vincristine sulfate, in vitro-treated peripheral blood stem cell transplantation; Procedure · Biological · Drug
Lead sponsor: Indiana University; Other
Eligibility: 5 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2011
U.S. locations: 2
States / cities: Indianapolis, Indiana • Boston, Massachusetts

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Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

Study of Gene Modified Immune Cells in Patients With Advanced Melanoma

Safety and Efficacy Study of Ad2/Hypoxia Inducible Factor (HIF)-1α/VP16 Gene Transfer in Patients With Intermittent Claudication

Comparing Treatments for Multiple Myeloma

Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease

Gene Therapy for Fanconi Anemia, Complementation Group A

A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)

Stem Cell (Modified Bone Marrow) Transplantation in HIV-Infected Patients With Blood Cancer

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

People s Expectations When Enrolling in a Phase I/II RS1 Ocular Gene Transfer Clinical Trial

Effect of Dalcetrapib vs Placebo on CV Risk in a Genetically Defined Population With a Recent ACS

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Follow-Up Study of Subjects Previously Enrolled in Poxviral Vector Gene Transfer Studies

A Phase I Study of AdV-tk + Prodrug Therapy in Combination With Radiation Therapy for Pediatric Brain Tumors

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)

Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions)

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

A Study to Evaluate the Safety and Efficacy of A2B395, an Allogeneic Logic-gated CAR T, in Participants With Solid Tumors That Express EGFR and Have Lost HLA-A*02 Expression

Gene Therapy and Biological Therapy in Treating Patients With Ovarian Epithelial Cancer

Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

Combination Chemotherapy Plus Gene Therapy in Treating Patients With CNS Tumors