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Condition: Genetic Therapy Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Genetic Therapy

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)
Phase 1/Phase 2 · 2 location(s) · Fundació Institut de Recerca de l'Hospital de la Santa Creu · NCT06648096DFT383 in Pediatric Participants With Nephropathic Cystinosis
Phase 1/Phase 2 · 4 location(s) · Novartis Pharmaceuticals · NCT06910813Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MY
Phase 1/Phase 2 · 10 location(s) · Tenaya Therapeutics · NCT05836259A Study on the Use of Canakinumab Among Familial Mediterranean Fever and Still's Disease Patients
Phase N/A · 1 location(s) · Novartis Pharmaceuticals · NCT07517250Sinonasal Therapies and Histologic Correlations of Patients With Cystic Fibrosis in the Era of Highly Effective Modulato
Phase 3 · 1 location(s) · Indiana University · NCT06996951A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
EARLY_Phase 1 · 1 location(s) · First Affiliated Hospital of Guangxi Medical University · NCT05762510Home-Based Brain Stimulation for Motoric Cognitive Risk Syndrome
NA · 2 location(s) · Hebrew SeniorLife · NCT06821568Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
NA · 33 location(s) · University of Washington, the Collaborative Health Studies C · NCT06654752Real-life Pharmacological Monitoring of Encorafenib-Binimetinib in the Treatment of Metastatic Melanoma
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06774989Oral Ketamine for Treatment of Vaso-Occlusive Pain
Phase 3 · 1 location(s) · Boston Children's Hospital · NCT05378555The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Phase N/A · 6 location(s) · Medical College of Wisconsin · NCT07227155Metformin as Adjunct Therapy in Depression-Obesity Comorbidity: Clinical and Genetic Evaluation
NA · 2 location(s) · Riphah International University · NCT07654179Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor
Phase 2 · 84 location(s) · Merck Sharp & Dohme LLC · NCT04924075A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
Phase N/A · 1 location(s) · LG Chem · NCT01604395Construction and Application of Precise Diagnosis and Treatment System for Thyroid-associated Ophthalmopathy
Phase N/A · 1 location(s) · Second Affiliated Hospital, School of Medicine, Zhejiang Uni · NCT07124572Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Phase 1 · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT05709288ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Phase 1/Phase 2 · 1 location(s) · Children's Hospital of Philadelphia · NCT06364774Investigating Changes in Premonitory Urges During Habit Reversal Training for Tics
NA · 1 location(s) · University of Utah · NCT06825520Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)
NA · 1 location(s) · Nemours Children's Clinic · NCT05383911A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Dise
NA · 1 location(s) · He Huang · NCT06162338Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Phase 1 · 1 location(s) · Daniel Bauer · NCT06647979Exploratory Study on the Treatment of Gout With Potassium Citrate Sustained-release Tablets
Phase 4 · 1 location(s) · Huashan Hospital · NCT06966635A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Ge
Phase 1/Phase 2 · 9 location(s) · Sanofi · NCT06844214Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
Phase 1/Phase 2 · 1 location(s) · Institute of Hematology & Blood Diseases Hospital, China · NCT07471516Propranolol and Von Hippel-Lindau Disease
NA · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT05424016A Prospective Cohort Study of Surgical Treatment for Foot Deformities in HSP
Phase N/A · 1 location(s) · Shanghai 6th People's Hospital · NCT06936163Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
Phase N/A · 1 location(s) · St. Jude Children's Research Hospital · NCT05810181ADNP Syndrome: The Seaver Autism Center for Research and Treatment is Characterizing ADNP-related Neurodevelopmental Dis
Phase N/A · 1 location(s) · Icahn School of Medicine at Mount Sinai · NCT03718936Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambu
Phase 3 · 1 location(s) · Dyne Therapeutics · NCT07608432
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.