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
- NCT06523582Recruiting
Genetic study of neuroendocrine tumors in Mexican patients
This study aims to find genetic causes of neuroendocrine tumors in Mexican patients. By understanding your DNA, researchers hope to improve diagnosis and treatment for people with these rare conditions.
Mexico City, Mexico CityAges 18 years+ - NCT07608432RecruitingPhase 3
Testing a New DMD Treatment Given Every 4 Weeks
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
Hillsborough, North CarolinaAges 4–18 - NCT07587242RecruitingPhase 3
Testing a New Therapy for DMD with Exon 44 Mutations
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
Westmead, New South WalesAges 7–16 - NCT07525960RecruitingPhase 1
Testing NDI-5001 for X-linked Nephrogenic Diabetes Insipidus
This study tests a new drug called NDI-5001 in adult men with a specific genetic form of nephrogenic diabetes insipidus (a condition where the kidneys cannot concentrate urine properly). The trial checks whether the drug is safe, how your body handles it, and whether it might help reduce excessive urination and thirst.
Jacksonville, FloridaAges 18–55 - NCT07523581RecruitingPhase 2
Study of an experimental drug for Alport syndrome
This study tests an experimental medicine for people with Alport syndrome caused by a specific type of gene change (nonsense mutation). The drug may help reduce protein in the urine and slow kidney decline. It is a blinded study, so you won't know if you get the real drug or a placebo.
Los Angeles, CaliforniaAges 12 years+ - NCT07502664Recruiting
A study on vision and navigation in inherited retinal disease
This study is looking at how people with inherited retinal disease that affects peripheral vision see and move around. It will test a new way to measure vision and navigation to see if it can help doctors understand how the disease affects daily life.
Irvine, CaliforniaAges 18 years+ - NCT07433621RecruitingPhase 1
Quercetin for XIAP deficiency in males ages 2 and up
This trial tests if the supplement quercetin can help patients with XIAP deficiency (an immune condition affecting the body's ability to control cell death and inflammation). It is given as a pill or liquid and might reduce inflammation-related symptoms.
Cincinnati, OhioAges 2 years+ - NCT07414511Recruiting
Hemophilia A research study for pregnant families
This study follows pregnant people who carry a severe hemophilia A gene and their babies after birth. It looks at how hemophilia A is passed down and how it affects families, with the goal of improving understanding and care.
Seattle, WashingtonAges Any age - NCT07328178Recruiting
Understanding IgE proteins in allergies and immune conditions
This study looks at IgE, a protein involved in allergic reactions, to better understand its role in different immune-related diseases. It aims to find out how different forms of IgE relate to health and disease, which may help improve diagnosis or treatment.
Leuven, Vlaams-BrabantAges 18 years+ - NCT07284641RecruitingPhase 2
Stem cell transplant for immune disorders
This trial tests a stem cell transplant to treat common variable immunodeficiency (CVID) and other immune problems. It aims to replace a faulty immune system with a healthy one from a donor.
Pittsburgh, PennsylvaniaAges 5–40 - NCT06278337Recruiting
Moesin-associated immunodeficiency study
This study looks at a rare immune system condition caused by changes in the MOESIN gene. It aims to better understand the condition and may help develop future treatments.
Bethesda, MarylandAges 4–80 - NCT07183579Recruiting
Finding the right dose of burosumab for XLH
This study looks at how young people with X-linked hypophosphataemia (XLH) respond to different doses of burosumab. The goal is to find the best dose to manage the condition.
NottinghamAges 2–18 - NCT07113743Enrolling by invitationPhase 1/Phase 2
Gene therapy for x-linked chronic granulomatous disease
This trial tests a new gene therapy for X-linked chronic granulomatous disease (CGD), a condition where the immune system cannot fight certain infections. The treatment uses your own blood stem cells, which are modified in the lab to help your body produce working immune cells.
Bethesda, MarylandAges 3–60 - NCT06982417Recruiting
RP2 Genetic Condition Registry
This registry study collects information from people who have a genetic change in the RP2 gene (a cause of inherited eye disease) and their families. It helps researchers learn more about the condition and how it affects daily life.
Göttingen, Lower SaxonyAges Any age - NCT06921720Recruiting
Phosphate diabetes muscle study using phosphorus scan
This study uses a special MRI scan to look at phosphorus levels in the muscles of people with phosphate diabetes (a condition where the body loses too much phosphate through the kidneys). It aims to better understand how this condition affects muscles and may help guide future treatments.
BronAges 10 years+ - NCT06581146Recruiting
Liver health study for boys with XLMTM
This study looks at how the liver works in boys with a serious muscle condition called XLMTM. It checks whether their liver is healthy, which may help doctors better understand the condition and guide future treatments.
Santa Monica, CaliforniaAges Up to 17 years - NCT04925349Recruiting
Study blood immune patterns in several rare brain diseases
This study looks at immune cells in blood to better understand rare brain conditions caused by genetic changes. It may help researchers understand why symptoms happen and how the immune system is involved.
Le Kremlin-BicêtreAges 1.3 years–60 years - NCT06147414Recruiting
Blood test for genetic disorders in pregnancy
This study tests a safer blood test to check for certain genetic disorders during pregnancy, instead of an invasive procedure like amniocentesis. It's for pregnant women who are at risk of passing on a known genetic condition.
ParisAges 18 years+ - NCT05902351Recruiting
Study of the natural course of Charcot-Marie-Tooth disease
This study looks at how Charcot-Marie-Tooth disease (CMT) or related inherited nerve conditions change over time. It may help researchers better understand what to expect and guide future treatments.
New York, New YorkAges Any age - NCT05432349Recruiting
Rett syndrome genetic registry for MECP2 changes
This trial is a registry that collects health and genetic information from people with Rett syndrome. It may help researchers better understand how MECP2 loss-of-function changes lead to symptoms and how to support patients.
Birmingham, AlabamaAges birth–99 years - NCT05027945RecruitingPhase 2
Stem cell transplant for people with VEXAS syndrome
This Phase 2 trial tests whether an allogeneic stem cell transplant (from a donor) can treat VEXAS syndrome. It may help calm the body’s overactive inflammation and improve symptoms in eligible adults.
Bethesda, MarylandAges 18–75 - NCT02356653RecruitingEarly Phase 1
Stem cell transplant help for children lacking a matching sibling
This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.
Philadelphia, PennsylvaniaAges Up to 30 years - NCT00697411Recruiting
Study of Aicardi syndrome in X-linked disorders
This trial looks at people with features that suggest Aicardi syndrome, a rare condition that can affect the brain and eyes and may include seizures. Researchers want to better understand the condition and how it shows up, which may help guide care in the future.
Houston, TexasAges Any age - NCT00128973Recruiting
Study immune system problems in people with unusual infections
This study looks at how the immune system works in people who have abnormal immune function, often shown by unusual or repeated infections or long-lasting inflammation. It may involve tests using blood samples and storage of blood for future research.
Bethesda, MarylandAges Any age
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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.