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Conditions: Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
Interventions: ACDN-01; Drug
Lead sponsor: Ascidian Therapeutics, Inc; Industry
Eligibility: 12 Years and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 10
States / cities: San Francisco, California • Gainesville, Florida • Miami, Florida + 7 more

Conditions: Body Mass Index
Interventions: None, cohort studies; Other
Lead sponsor: Tufts University; Other
Eligibility: 18 Years to 90 Years
Enrollment: 602 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Myotonic Dystrophy
Interventions: SAR446268; Biological
Lead sponsor: Sanofi; Industry
Eligibility: 10 Years to 55 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 4
States / cities: Gainesville, Florida • Tampa, Florida • New York, New York + 1 more

Conditions: Melanoma, Adenocarcinoma, Carcinoma NOS, Neuroendocrine Tumors
Interventions: Not listed
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 1,150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hypertrophic Cardiomyopathy, Left Ventricular Hypertrophy
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 175 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Aging
Interventions: Sciton SkinTyte (800-1800 nm) with Broadband Light Technology; Device
Lead sponsor: Stanford University; Other
Eligibility: 55 Years and older · Female only
Enrollment: 5 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2017
U.S. locations: 1
States / cities: Redwood City, California

Conditions: Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease
Interventions: DCR-PHXC, Sterile Normal Saline (0.9% NaCl); Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 6 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 5
States / cities: San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 2 more

Conditions: Neoplasms
Interventions: BIBW 2992 (Afatinib); Drug
Lead sponsor: Boehringer Ingelheim; Industry
Eligibility: 18 Years to 99 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2008
U.S. locations: 12
States / cities: Los Angeles, California • Denver, Colorado • Indianapolis, Indiana + 9 more

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: Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Interventions: preimplantation diagnosis; Other
Lead sponsor: Natera, Inc.; Industry
Eligibility: Up to 40 Years
Enrollment: 56 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2014
U.S. locations: 1
States / cities: Redwood City, California

Conditions: Hereditary Pulmonary Alveolar Proteinosis
Interventions: Gene-Corrected Macrophages administered by bronchoscopic instillation; Combination Product
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 18 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2038
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Colorectal Cancer
Interventions: GVAX; Biological
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Myeloma, Plasma-Cell, Myeloma-Multiple
Interventions: Cyclophosphamide, Fludarabine, Anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cells; Drug · Biological
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years to 73 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Fabry Disease
Interventions: AMT-191; Drug
Lead sponsor: UniQure Biopharma B.V.; Industry
Eligibility: 18 Years to 50 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2031
U.S. locations: 8
States / cities: Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 5 more

Conditions: Hypertrophic Cardiomyopathy, Genetic Disease, Gene Product Sequence Variation
Interventions: Genomic sequencing; Genetic
Lead sponsor: The Cleveland Clinic; Other
Eligibility: 18 Years to 100 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Breast Neoplasm, Hereditary Neoplastic Syndrome, Melanoma, Ovarian Neoplasm
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2008
U.S. locations: 5
States / cities: Tucson, Arizona • Baltimore, Maryland • Ann Arbor, Michigan + 2 more

Conditions: Sarcoma, Kaposi, HIV Infections
Interventions: Ro 24-7429; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 2
States / cities: Los Angeles, California • Boston, Massachusetts

Conditions: Congenital Heart Disease
Interventions: Blood Sample Collection; Other
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: Not listed
Enrollment: 5,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2030
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Multiple Myeloma, Melanoma, Synovial Sarcoma, Myxoid/Round Cell Liposarcoma
Interventions: NY-ESO-1 redirected autologous T cells with CRISPR edited endogenous TCR and PD-1, Cyclophosphamide, Fludarabine, NY-ESO-1 expression testing; Biological · Drug · Device
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Advanced Nonhaematologic Malignancies
Interventions: BOS172738; Drug
Lead sponsor: Boston Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 117 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Houston, Texas

Conditions: Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions: nab-sirolimus; Drug
Lead sponsor: Aadi Bioscience, Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 159
States / cities: Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more

Conditions: Neovascular Age-Related Macular Degeneration (nAMD), Neovascular (Wet) Age-related Macular Degeneration (AMD)
Interventions: Aflibercept (2.0 mg); Biological
Lead sponsor: Exegenesis Bio; Industry
Eligibility: Not listed
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 2
States / cities: Reno, Nevada • Erie, Pennsylvania

Conditions: Prostate Cancer
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years to 100 Years
Enrollment: 5,118 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2018
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Prostate Cancer
Interventions: Decipher, Prolaris, Oncotype Dx Genomic Prostate Score (GPS), Standard of care (askMUSIC score); Device · Other
Lead sponsor: University of Michigan Rogel Cancer Center; Other
Eligibility: 18 Years and older · Male only
Enrollment: 805 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2031
U.S. locations: 8
States / cities: Ann Arbor, Michigan • Detroit, Michigan • Grand Rapids, Michigan + 4 more

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Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

Epigenomic and Metabolomic Signatures of APOA2 Gene by Saturated Fat Interaction

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

Follow Up Protocol for Subjects Previously Enrolled on NCI Surgery Branch Studies

Factors Contributing to Increased Left Ventricle Size in Patients With Abnormally Enlarged Hearts

Infrared and Broadband Light for Skin Aging

A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2

Phase II Trial of BIBW 2992 (Afatinib) in Genetically Pre-screened Cancers With EGFR and/or HER2 Gene Amplification.

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

Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)

Safety and Efficacy of PMT Therapy of hPAP

Pilot Study of GVAX in Colorectal Cancer Cells

Study of T Cells Targeting B-Cell Maturation Antigen for Previously Treated Multiple Myeloma

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Genomic Sequencing in Patients With HCM Undergoing Septal Myectomy

Microarray Analysis for Human Genetic Disease

A Study of Ro 24-7429 in Patients With HIV-Related Kaposi's Sarcoma

Genetics of Congenital Heart Disease

NY-ESO-1-redirected CRISPR (TCRendo and PD1) Edited T Cells (NYCE T Cells)

Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

A Long-term Follow up Study of EXG102-031 in Patients With wAMD (Everest LTFU)

Identification of Prostate Cancer Genes

Genomics in Michigan to AdJust Outcomes in Prostate canceR (G-MAJOR) for Men With Newly Diagnosed Favorable Risk Prostate Cancer