Clin2
NCT01962415Possibly a fitRecruiting

Reduced-intensity stem cell transplant for non-cancer disorders

Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory ConditionsSystemic Juvenile Idiopathic Arthritis (sJIA)Juvenile Rheumatoid Arthritis (JRA)

Treatments studied

Part of Blood & lymphatic, Bones, joints & muscles, Brain & nervous system, Digestive system, Eyes & vision, Genetic & congenital, Hormones & metabolism, Immune system & allergy, Skin clinical trials.

This trial tests whether a gentler (reduced-intensity) conditioning treatment before a stem cell transplant works for non-cancer diseases. It uses specific donor stem cell matches and checks heart, lung, liver, and kidney function to help lower transplant stress while still aiming for benefit.

Summary written for real people, not researchers, by Clin2.

Phase
Phase 2
Enrollment
100 people
Ages
2 months to 55 years
Study type
Interventional

Who can take part

  • You need a suitable stem cell donor match: matched cord blood or matched donor bone marrow/peripheral blood
  • The donated stem cell amount must meet a minimum level (or you may receive two cord blood units)
  • Your kidneys, liver, and heart need to work well enough for transplant (lab tests and heart scan results)
  • Your lungs need to be strong enough based on breathing test results or oxygen level
  • You must have a non-cancer condition that can be treated with stem cell transplant
  • You must not be recently transplanted (no allogeneic transplant in the last 6 months) and must not have certain infections, pregnancy, or uncontrolled lung blood pressure problems

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

Similar studies

Other trials that look related to this one.

NCT07493538Recruiting· Phase 2
Blood stem cell transplant with lower-intensity chemo for blood cancers

This trial tests a stem cell transplant using milder chemotherapy (treosulfan and fludarabine) followed by a drug called cyclophosphamide to prevent graft-versus-host disease. It aims to make transplant safer for people with blood diseases who cannot handle standard high-dose chemotherapy.

Minneapolis, Minnesota
NCT04339777Recruiting· Phase 2
Bone marrow transplant for inherited immune system disorders

This trial studies how well a donor bone marrow (stem cell) transplant works for people with inherited immune system problems (called immune deficiencies). It’s for patients who haven’t improved with usual treatment or don’t have another good option.

Bethesda, Maryland
NCT04232085Recruiting· Phase 2
Stem cell transplant to rebuild immunity in genetic immune disorders

This Phase 2 trial tests a stem cell (bone marrow) transplant approach to restore bone marrow function and immune function in people with certain inherited immune problems. If you have one of these conditions and a suitable donor, the study may offer a path to rebuild blood and immune systems.

Baltimore, Maryland
NCT07660783Not yet recruiting· Phase 2
Lowering transplant rejection risk in young children

This trial tests a way to lower the risk of graft-versus-host disease (GVHD), a common complication after a bone marrow transplant, by removing certain immune cells from the donor's stem cells before giving them to the patient. It is for young children with non-cancerous blood or immune diseases who need a transplant.

Seattle, Washington
NCT03980769Recruiting· Phase 2
Stem cell transplant trial for non-cancer blood disorders

This Phase 2 study tests a specific chemotherapy combination (treosulfan, fludarabine, and thiotepa) before an unrelated or matched donor stem cell transplant. It may help people with certain non-cancer blood and immune disorders by trying to reset the immune system using donor stem cells.

Seattle, Washington
NCT05794880Recruiting
Immunotherapy lowering immune cells before transplant in children

This trial tests a special immune-cell “preparation” treatment (called T-cell and B-cell depletion) with adjusted anti-thymocyte globulin (ATG) dosing before a stem cell transplant. It aims to reduce immune-related transplant complications in people under 25 with certain blood cancers in a specific remission status.

Milwaukee, Wisconsin

Hear when a new Congenital Bone Marrow Failure Syndromes trial opens

We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.