Phase 1 X-Linked Clinical Trials
10 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–10 of 10 trials
Recruiting
Phase 1Phase 2
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
X-linked Severe Combined Immunodeficiency (XSCID)
National Institute of Allergy and Infectious Diseases (NIAID)40 enrolled1 locationNCT01306019
Recruiting
Phase 1Phase 2
Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease
X-Linked Chronic Granulomatous Disease
Ensoma15 enrolled9 locationsNCT06876363
Recruiting
Phase 1Phase 2
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
X-linked Myotubular Myopathy
Astellas Gene Therapies9 enrolled4 locationsNCT07052929
Recruiting
Phase 1Phase 2
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
Chronic Granulomatous Disease (CGD)X-Linked Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID)10 enrolled1 locationNCT06325709
Recruiting
Phase 1Phase 2
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Gene TherapySevere Combined Immunodeficiency, X-Linked
David Williams12 enrolled4 locationsNCT03311503
Recruiting
Phase 1Phase 2
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
X-linked Hypophosphatemia
Kyowa Kirin Co., Ltd.24 enrolled9 locationsNCT06525636
Recruiting
Phase 1Phase 2
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
SCID, X-Linked
Shenzhen Geno-Immune Medical Institute10 enrolled2 locationsNCT03217617
Recruiting
Phase 1
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
VEXAS SyndromeVEXASE1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome
Washington University School of Medicine15 enrolled1 locationNCT06538181
Recruiting
Phase 1Phase 2
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
X-Linked Retinitis Pigmentosa (XLRP)
Frontera Therapeutics32 enrolled1 locationNCT06492850
Recruiting
Phase 1
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Severe Combined Immunodeficiency, X-Linked
Great Ormond Street Hospital for Children NHS Foundation Trust5 enrolled1 locationNCT03601286