Clinical trials
X-Linked clinical trials
Below are recruiting x-linked clinical trials, each written for real people, not researchers. We’re tracking 49 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05814952Recruiting
LX103 eye treatment for X-linked retinoschisis
This trial studies whether LX103 can improve vision and is safe for people with X-linked retinoschisis (XLRS) caused by RS1 gene changes. It focuses on a specific level of vision in the study eye and checks for medical conditions that could affect eye testing or safety.
ShanghaiAges 6 years+ - NCT05874310RecruitingEarly Phase 1
Gene therapy trial for RPGR-related vision loss
This early study tests a gene therapy designed for people whose vision problems are caused by an RPGR genetic change. It may help slow or improve the eye disease by treating the underlying cause.
Beijing, Beijing MunicipalityAges 8–45 - NCT05939232Recruiting
Registry for X-linked adrenal and brain fat disease
This study collects health information and test results from people with X-linked adrenoleukodystrophy (X-ALD) and some genetic carriers. It helps researchers better understand the condition and how it varies, which can guide future care and studies.
BeijingAges 6–70 - NCT06178120Recruiting
Study of disease progression in women with X-linked adrenoleukodystrophy
This study follows women with a genetic condition called X-linked adrenoleukodystrophy (ALD) over time to see how the disease progresses. It includes annual check-ups and MRI scans of the brain and spinal cord to monitor changes.
ParisAges 18 years+ - NCT07820085RecruitingPhase 2/Phase 3
Oral treatment for light sensitivity in EPP and XLP
This study tests an oral medication, PORT-77, to reduce pain and sensitivity to sunlight in people with EPP or XLP. It may help those who experience symptoms shortly after sun exposure.
Huntington Beach, CaliforniaAges 12 years+ - NCT07567131Recruiting
Understanding sun sensitivity in rare porphyria disorders
This study tracks how your body reacts to sunlight if you have erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP)—rare genetic conditions that cause painful skin reactions to sun exposure. Researchers will use special devices and daily logs to better understand your symptoms and how light affects your skin.
Boston, MassachusettsAges 12 years+ - NCT06345898RecruitingEarly Phase 1
Gene therapy for X-linked retinoschisis in young males
This trial tests a single gene therapy injection under the retina for boys with X-linked retinoschisis, a condition that causes vision loss. It aims to see if the treatment is safe and can improve vision.
Chengdu, SichuanAges 5–18 - NCT04064307Recruiting
Register people with myotubular or centronuclear myopathy
This study sets up a patient registry for people diagnosed with myotubular myopathy or centronuclear myopathy. Your information can help researchers better understand these conditions and plan future studies or treatments.
Newcastle upon Tyne, Tyne and WearAges Any age - NCT04980638RecruitingPhase 2
Check ER004 given in the womb for a genetic tooth-skin disorder
This Phase 2 study tests ER004 put into the amniotic fluid during pregnancy to treat male babies with X-linked hypohidrotic ectodermal dysplasia (XLHED). It also looks at effects in affected male relatives with the same gene change.
Los Angeles, CaliforniaAges 18 years+ - NCT07052929RecruitingPhase 1/Phase 2
Gene therapy study for young boys with XLMTM on ventilators
This study tests a new gene therapy called ASP2957 in young boys with X-linked myotubular myopathy who are dependent on a ventilator. The goal is to see if it can help improve their breathing and muscle strength.
Chicago, IllinoisAges Up to 3 years - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT06868979Recruiting
Eye imaging for X-linked disorders
This study uses special eye imaging to look at changes in the brain and eyes of people with creatine transporter deficiency (a condition that affects brain energy) or fragile X syndrome. It aims to find a way to see these brain changes without needing a biopsy or spinal tap.
BronAges 5–60 - NCT06851767Enrolling by invitationPhase 1/Phase 2
Gene therapy for X-linked severe combined immunodeficiency
This study tests a gene therapy to treat X-linked severe combined immunodeficiency (X-SCID) without a stem cell transplant from a matched sibling. It aims to improve immune function and reduce infections using your own stem cells modified to carry the correct gene.
Bethesda, MarylandAges 3–99 - NCT06538181RecruitingPhase 1
Testing pacritinib for VEXAS syndrome
This trial tests a new drug called pacritinib for people with VEXAS syndrome, a rare autoinflammatory condition. The goal is to see if pacritinib can help control symptoms like rashes, joint pain, and low blood counts.
St Louis, MissouriAges 18 years+ - NCT06525636RecruitingPhase 1/Phase 2
Study of a new drug for X-linked hypophosphatemia
This study tests an investigational drug called KK8123 for adults with X-linked hypophosphatemia (XLH), a rare condition that causes low phosphate levels and can lead to bone problems. The goal is to see if it helps improve phosphate levels in the body.
San Francisco, CaliforniaAges 18–65 - NCT03055247RecruitingPhase 2
Try ibuprofen plus growth factor and stem-cell mobilizer for X-CGD
This trial tests a combination of medicines to help your body release (“mobilize”) stem cells for future treatment in people with X-linked chronic granulomatous disease (X-CGD). If it works well and is safe for you, it may make stem-cell collection easier and more successful.
Rome, LazioAges 18–45 - NCT06325709RecruitingPhase 1/Phase 2
Gene editing trial for X-linked chronic granulomatous disease
This trial tests a new gene-editing treatment for men with a specific genetic mutation that causes X-linked chronic granulomatous disease (CGD). The goal is to fix the faulty gene in your own blood stem cells to help your immune system fight infections better.
Bethesda, MarylandAges 18–75 - NCT06275620Enrolling by invitationPhase 2
Testing a gene therapy for X-linked retinitis pigmentosa in males
This trial tests two doses of a gene therapy (AGTC-501) in males who already had one eye treated with a similar gene therapy for X-linked retinitis pigmentosa caused by RPGR mutations. The goal is to see if the treatment is safe and can help preserve or improve vision.
Jacksonville, FloridaAges 12 years+ - NCT03047369Recruiting
Myelin brain disorder research biobank for samples and data
This study collects brain-related medical information and biological samples to help researchers better understand leukodystrophies and other white-matter brain disorders. It may help future diagnosis and treatments by building a valuable “biobank” of patient data and samples.
Los Angeles, CaliforniaAges Any age - NCT05878860RecruitingPhase 3
Gene therapy for X-linked retinoschisis
This trial tests a gene therapy called ATSN-201 for people with X-linked retinoschisis (XLRS), a condition that affects the retina and can cause vision loss. The therapy aims to fix the faulty gene that causes the condition, potentially halting vision loss and improving vision. Participants receive a one-time injection into the eye, and the study monitors safety and effectiveness.
Phoenix, ArizonaAges 5 years+ - NCT04675749Recruiting
Quality of life study in adult women with X-ALD
This study looks at quality of life in adult women who have X-linked adrenoleukodystrophy (X-ALD). It may help researchers understand how this condition affects daily life and well-being.
Leipzig, SaxonyAges 18 years+ - NCT03193476Recruiting
XLH patient registry for people with X-linked hypophosphatemia
This registry collects information about people diagnosed with X-linked hypophosphatemia (XLH). It helps researchers better understand the condition and may support future studies by tracking patients over time.
BrusselsAges Any age - NCT01306019RecruitingPhase 1/Phase 2
Gene therapy for children with severe immune deficiency (XSCID)
This trial tests a one-time gene therapy approach to help children with X-linked SCID (a serious inherited immune disorder) make better immune cells. It’s for people who don’t have a fully matched sibling donor and need long-term follow-up after treatment.
Bethesda, MarylandAges 2–50 - NCT06740162Recruiting
Physical activity program for adults with intellectual disabilities
This study tests a program to help adults with intellectual disabilities become more physically active. You and your caregiver will work with a coach to increase your daily movement, with the goal of improving health and community connection.
Fayetteville, ArkansasAges 18 years+
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Common questions
- Are there clinical trials for x-linked?
- Yes. Clin2 currently lists 49 recruiting x-linked studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a x-linked trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a x-linked trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.