X-Linked Clinical Trials

1 recruiting

Frequently Asked Questions

Common questions about X-Linked clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 36 trials

Recruiting

Evaluation of Patients With Immune Function Abnormalities

Chronic Granulomatous Disease (CGD)X-Linked Severe Combined Immune Deficiency (XSCID)Leukocyte Adhesion Deficiency 1 (LAD)+1 more
National Institute of Allergy and Infectious Diseases (NIAID)3,500 enrolled1 locationNCT00128973
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

Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes

GranulomaGranulomatous Disease, ChronicLeukocyte Disease+2 more
National Institute of Allergy and Infectious Diseases (NIAID)850 enrolled1 locationNCT00001405
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

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 3

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

Erythropoietic Protoporphyria (EPP)X-Linked Protoporphyria (XLP)
Disc Medicine, Inc150 enrolled28 locationsNCT06910358
Recruiting
Not Applicable

Physical Activity and Community EmPOWERment Project

Williams SyndromeWAGR SyndromePrader-Willi Syndrome+10 more
University of North Carolina, Chapel Hill376 enrolled2 locationsNCT06740162
Recruiting
Early Phase 1

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

LeukemiaInborn Errors of MetabolismBone Marrow Failure Syndromes+2 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT02356653
Recruiting
Phase 2

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics10 enrolled6 locationsNCT07174726
Recruiting

Hemophilia A Research Program

PregnancyHemophilia APregnancy Complications+10 more
University of Washington500 enrolled1 locationNCT07414511
Recruiting
Not Applicable

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

Tuberous SclerosisFragile X SyndromeRett Syndrome+5 more
Rush University Medical Center92 enrolled1 locationNCT06139172
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting

Analysis of the Role of IgE Proteoforms in Health and Disease

AnaphylaxisHealthy ControlMastocytosis+6 more
KU Leuven200 enrolled1 locationNCT07328178
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

The Myelin Disorders Biorepository Project

AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369
Recruiting

Effective Dosing of Burosumab in XLH

X-linked Hypophosphatemia (XLH)
University of Nottingham120 enrolled1 locationNCT07183579
Recruiting
Phase 2

Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD

Chronic Granulomatous Disease X-linked (X-CGD)
IRCCS San Raffaele3 enrolled2 locationsNCT03055247
Recruiting
Not Applicable

IT and IV Lentiviral Gene Therapy for X-ALD

X-linked Adrenoleukodystrophy
Shenzhen Geno-Immune Medical Institute30 enrolled1 locationNCT03727555
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

VEXASE1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome
Washington University School of Medicine15 enrolled1 locationNCT06538181