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Conditions: Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
Interventions: LX2020; Genetic
Lead sponsor: Lexeo Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 5
States / cities: Stanford, California • Baltimore, Maryland • Ann Arbor, Michigan + 2 more

Conditions: X-Linked Retinitis Pigmentosa
Interventions: Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose; Biological
Lead sponsor: Janssen Research & Development, LLC; Industry
Eligibility: 3 Years and older
Enrollment: 97 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2029
U.S. locations: 10
States / cities: La Jolla, California • Los Angeles, California • Palo Alto, California + 7 more

Conditions: Recurrent Acute Leukemia of Ambiguous Lineage, Recurrent Acute Lymphoblastic Leukemia, Recurrent Acute Myeloid Leukemia, Refractory Acute Lymphoblastic Leukemia, Refractory Acute Myeloid Leukemia, Refractory Acute Leukemia of Ambiguous Lineage
Interventions: Chemosensitivity Assay, Cytology Specimen Collection Procedure, Gene Expression Analysis, Genetic Variation Analysis, In Vitro Sensitivity-Directed Chemotherapy; Other · Genetic · Drug
Lead sponsor: University of Washington; Other
Eligibility: 3 Years and older
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions: AT-GTX-501; Genetic
Lead sponsor: Emily de los Reyes; Other
Eligibility: 1 Year and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2024
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Inflammatory Bowel Diseases
Interventions: Baseline blood sample, Baseline intestinal biopsies, Crohn's disease activity index (CDAI), Post treatment blood sample, Post treatment intestinal biopsies, Baseline stool sample, Post treatment stool sample; Diagnostic Test · Other
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 96 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 2
States / cities: Nashville, Tennessee

Conditions: Ataxia Telangiectasia
Interventions: Antisense oligonucleotide targeting the ATM gene; Drug
Lead sponsor: Timothy Yu; Other
Eligibility: 0 Years to 17 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2036
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Myotubular Myopathy
Interventions: Genetic Testing; Other
Lead sponsor: Cure CMD; Other
Eligibility: 30 Days and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Torrance, California

Conditions: Autism Spectrum Disorder, Movement Disorders, Seizures, Dystonia, NEDAMSS
Interventions: No interventions are planned; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 2 Months to 80 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Dilated Cardiomyopathy (DCM)
Interventions: RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3); Genetic
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 3
States / cities: San Diego, California • Rochester, Minnesota • Charleston, South Carolina

Conditions: Pre-diabetes
Interventions: Oral glucose tolerance test; Other
Lead sponsor: Mayo Clinic; Other
Eligibility: 35 Years to 80 Years
Enrollment: 190 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2012
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Lynch Syndrome
Interventions: Not listed
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 18 Years and older
Enrollment: 5,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2054
U.S. locations: 2
States / cities: Aurora, Colorado • Chicago, Illinois

Conditions: Liver Cancer, Liver Metastases
Interventions: Hepatic trans-arterial embolization (TAE), MRI/CT scan, biopsy, blood draw; Procedure · Device · Other
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2016
U.S. locations: 1
States / cities: New York, New York

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: Neurodevelopmental Disorders, Intellectual Disability, Developmental Delay, Autism Spectrum Disorder, Seizures, Hypertonia, Muscle, Hypotonia
Interventions: Not listed
Lead sponsor: Columbia University; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Carcinoma, Squamous Cell, Carcinoma, Squamous, Squamous Cell Carcinoma, Lung Neoplasms, Cancer of Lung, Cancer of the Lung, Lung Cancer, Neoplasms, Lung, Neoplasms, Pulmonary, Pulmonary Cancer, Pulmonary Neoplasms
Interventions: DNA Extraction; Genetic
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Interventions: RP-A601; Genetic
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 3
States / cities: La Jolla, California • Durham, North Carolina • Philadelphia, Pennsylvania

Conditions: Induced Pluripotent Stem Cells
Interventions: Not listed
Lead sponsor: National Institute on Drug Abuse (NIDA); NIH
Eligibility: 21 Years to 65 Years
Enrollment: 49 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2014
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Abnormal Elastogenesis, Vascular Diseases, Connective Tissue Diseases
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 100 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Kidney Stones, Urolithiasis, Calcium Oxalate Urolithiasis
Interventions: Not listed
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 2,900 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2027
U.S. locations: 1
States / cities: Jacksonville, Florida

Conditions: Huntington Disease
Interventions: Mutation in the gene; Genetic
Lead sponsor: National Institute of Nursing Research (NINR); NIH
Eligibility: Not listed
Enrollment: 422 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2008
U.S. locations: 1
States / cities: Iowa City, Iowa

Conditions: Metastatic Pancreatic Ductal Adenocarcinoma, Homologous Recombination Deficiency (HRD)
Interventions: Pembrolizumab, Olaparib; Drug
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 7
States / cities: Middletown, New Jersey • Montvale, New Jersey • Commack, New York + 3 more

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: X-Linked Retinitis Pigmentosa
Interventions: rAAV2tYF-GRK1-RPGR; Biological
Lead sponsor: Beacon Therapeutics; Industry
Eligibility: 8 Years to 50 Years · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 6
States / cities: Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more

Conditions: 16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder), ACTL6B, ADNP, AHDC1, ANK2, ANKRD11, ARID1B, ASH1L, BCL11A, CHAMP1, CHD2, CHD8, CSNK2A1, CTBP1, CTNNB1 Gene Mutation, CUL3, DDX3X, DNMT3A, DSCAM, DYRK1A, FOXP1, GRIN2A, GRIN2B, HIVEP2-Related Intellectual Disability, HNRNPH2, KATNAL2, KDM5B, KDM6B, KMT2C Gene Mutation, KMT2E, KMT5B, MBD5, MED13L, PACS1, PPP2R5D-Related Intellectual Disability, PTCHD1, REST, SCN2A Encephalopathy, SETBP1 Gene Mutation, SETD5, SMARCA4 Gene Mutation, SMARCC2, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability, TBR1, ARHGEF9, HNRNPU, PPP3CA, PPP2R1A, SLC6A1, 2p16.3 Deletions, 5q35 Deletions, 5q35 Duplications, 7q11.23 Duplications, 15Q13.3 Deletion Syndrome, 16p11.2 Triplications, 16P12.2 Microdeletion, 16P13.11 Microdeletion Syndrome (Disorder), 17Q12 Microdeletion Syndrome (Disorder), 17Q12 Duplication Syndrome, 17Q21.31 Deletion Syndrome, 17q21.3 Duplications, ACTB, ADSL, AFF2, ALDH5A1, ANK3, ARX, ATRX Gene Mutation, AUTS2 Syndrome, BCKDK, BRSK2, CACNA1C, CAPRIN1, CASK, CASZ1, CHD3, CIC, CNOT3, CREBBP Gene Mutation, CSDE1, CTCF, DEAF1, DHCR7, DLG4, EBF3, EHMT1, EP300 Gene Mutation, GIGYF1, GRIN1, GRIN2D, IQSEC2-Related Syndromic Intellectual Disability, IRF2BPL, KANSL1, KCNB1, KDM3B, NEXMIF, KMT2A, MBOAT7, MEIS2, MYT1L, NAA15, NBEA, NCKAP1, NIPBL, NLGN2, NLGN3, NLGN4X, NR4A2, NRXN1, NRXN2, NSD1 Gene Mutation, PHF21A, PHF3, PHIP, PSMD12, RELN, RERE, RFX3, RIMS1, RORB, SCN1A, SETD2 Gene Mutation, SHANK2, SIN3A, SLC9A6, SON, SOX5, SPAST, SRCAP, TAOK1, TANC2, TCF20, TLK2, TRIO, TRIP12, UPF3B, USP9X, VPS13B, WAC, WDFY3, ZBTB20, ZNF292, ZNF462, 2Q37 Deletion Syndrome, 9q34 Duplications, 15q15 Deletions, 15Q24 Deletion, NR3C2, SYNCRIP, 2q37.3 Deletion, 6q16 Deletion, 15q11.2 BP1-BP2 Deletion, 16p13.3 Deletion, 17Q11.2 Microduplication Syndrome (Disorder), 17p13.3, Xq28 Duplication, CLCN4, CSNK2B, DYNC1H1, EIF3F, GNB1, MED13, MEF2C, RALGAPB, SCN1B, YY1, Xp11.22 Duplication, PACS2, MAOA, MAOB, HNRNPC, HNRNPD, HNRNPK, HNRNPR, HNRNPUL2, 5P Deletion Syndrome, TCF7L2 Gene Mutation, HECW2, PPM1D, RNU4-2, SNAP25, FOXP2, ITSN1
Interventions: Not listed
Lead sponsor: Simons Searchlight; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2050
U.S. locations: 2
States / cities: Boston, Massachusetts • Lewisburg, Pennsylvania

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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

High Throughput Drug Sensitivity Assay and Genomics- Guided Treatment of Patients With Relapsed or Refractory Acute Leukemia

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

Cytokines and Genes in Therapeutic Response in Crohn's Disease

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

Myotubular Myopathy Genetic Testing Study

Database Of Clinical Data For Individuals With Variants In The IRF2BPL Gene

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Common Genetic Variation and Type 2 Diabetes

Lynch Syndrome Integrative Epidemiology and Genetics

A Study to See if we Can Predict How Your Liver Tumor or Liver Metastases Will Respond to Trans-Arterial Embolization (TAE)

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

Longitudinal Study of Neurogenetic Disorders

Identification of Prognostic Biomarkers for Progression of Invasive Squamous Cell Carcinoma

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Stem Cell Study of Genetics and Drug Addiction

Gene First Approach to Connective Tissue Disease

Pilot Study to Evaluate the Contribution of Gene Variants to Idiopathic Urolithiasis

Family Health After Predictive Huntington Disease (HD) Testing

A Study of Pembrolizumab and Olaparib for People With Metastatic Pancreatic Ductal Adenocarcinoma and Homologous Recombination Deficiency or Exceptional Treatment Response to Platinum-Based Therapy

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)

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight