X-Linked Clinical Trials

45 recruitingLast updated: May 13, 2026

There are 45 actively recruiting x-linked clinical trials across 26 countries. Studies span Phase 2, Phase 1, Not Applicable, Early Phase 1, Phase 3. Top locations include Boston, Massachusetts, United States, Los Angeles, California, United States, Philadelphia, Pennsylvania, United States. Updated daily from ClinicalTrials.gov.


X-Linked Trials at a Glance

45 actively recruiting trials for x-linked are listed on ClinicalTrialsFinder across 6 cities in 26 countries. The largest study group is Phase 2 with 12 trials, with the heaviest enrollment activity in Boston, Los Angeles, and Philadelphia. Lead sponsors running x-linked studies include National Institute of Allergy and Infectious Diseases (NIAID), Children's Hospital of Philadelphia, and Astellas Gene Therapies.

Browse x-linked trials by phase

Treatments under study

About X-Linked Clinical Trials

Looking for clinical trials for X-Linked? There are currently 1 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new X-Linked trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

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 45 trials

Recruiting

Effective Dosing of Burosumab in XLH

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

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

Immune DysregulationPrimary Immune Regulatory DisorderChronic Granulomatous Disease+12 more
Paul Szabolcs25 enrolled1 locationNCT07284641
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

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

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

Erythropoietic Protoporphyria (EPP)X-Linked Porphyria (XLP)
Portal Therapeutics, Inc.50 enrolled19 locationsNCT07567131
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 3

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

X-linked Retinoschisis
Atsena Therapeutics Inc.97 enrolled4 locationsNCT05878860
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
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

A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

X-linked Myotubular Myopathy
Astellas Gene Therapies50 enrolled6 locationsNCT06581146
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

Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)

X-linked RetinoschisisRetinitis PigmentosaStargardt Disease+3 more
Ray Therapeutics, Inc.25 enrolled1 locationNCT07502664
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

Analysis of the Role of IgE Proteoforms in Health and Disease

AnaphylaxisHealthy ControlMastocytosis+6 more
KU Leuven200 enrolled1 locationNCT07328178