Treatment
Base-edited hematopoietic stem and progenitor cells clinical trials
We’re tracking 4 recruiting studies that include Base-edited hematopoietic stem and progenitor cells, each written for real people, not researchers, below.
Recruiting studies
- NCT07775313Enrolling by invitationPhase 1/Phase 2
Gene therapy for WHIM syndrome
This trial tests a one-time gene therapy treatment for WHIM syndrome, a rare immune disorder. It aims to fix the genetic cause to improve your body's ability to fight infections. Your stem cells will be collected, edited in a lab, and given back to you after some chemotherapy to prepare your body.
Bethesda, MarylandAges 3–75 - NCT06959771RecruitingPhase 1/Phase 2
Gene therapy for CD40L deficiency (Hyper-IgM syndrome) - single patient study
This trial tests a new gene therapy that uses a person's own blood stem cells and T cells, which are corrected with a gene-editing tool called base editing. It is designed for people with a specific CD40L gene mutation who also have liver problems and portal hypertension. The goal is to see if this treatment can improve their immune system and liver health.
Bethesda, MarylandAges 37–120 - 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 - 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
What Base-edited hematopoietic stem and progenitor cells is being studied for
Conditions with recruiting Base-edited hematopoietic stem and progenitor cells studies.
Common questions
- Are there clinical trials studying Base-edited hematopoietic stem and progenitor cells?
- Yes. Clin2 currently lists 4 recruiting studies that include Base-edited hematopoietic stem and progenitor cells, drawn from the U.S. registry and rewritten for real people so you can see what each is testing and who it's for.
- How do I know if I qualify for a Base-edited hematopoietic stem and progenitor cells trial?
- Each study lists its own eligibility criteria — rules about condition, age, and prior treatments. On every Clin2 trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Is Base-edited hematopoietic stem and progenitor cells an approved treatment?
- A drug being studied in a trial may be investigational (still being tested) or already approved and being studied for a new use. Each study page explains what phase it's in. Clin2 does not provide medical advice — talk with your doctor about whether Base-edited hematopoietic stem and progenitor cells or a specific trial is right for you.
Related treatments
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. A drug appearing here is being studied in a trial and may be investigational. Talk with your doctor about whether a specific treatment or trial is right for you.