RecruitingPhase 2NCT01962415

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation


Sponsor

Paul Szabolcs

Enrollment

100 participants

Start Date

Feb 4, 2014

Study Type

INTERVENTIONAL

Conditions

Summary

The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.


Eligibility

Min Age: 2 MonthsMax Age: 55 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a reduced-intensity preparation regimen before a bone marrow transplant (using a donor's stem cells) for children and adults with non-cancerous blood disorders, to see if a gentler approach can still be effective while reducing serious side effects. **You may be eligible if...** - You have been diagnosed with a non-cancerous blood or immune system disorder (such as sickle cell disease, thalassemia, bone marrow failure, or an immune deficiency) - A bone marrow or stem cell transplant from a matched donor has been recommended as your treatment - You are not healthy enough to tolerate the standard high-intensity transplant preparation - You have a suitable donor (related or unrelated, with a good tissue match) **You may NOT be eligible if...** - You have an active, uncontrolled infection - You have significant damage to your heart, liver, or kidneys - You have previously had a stem cell transplant that failed - You are pregnant - Your donor is not adequately matched Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGHydroxyurea

Oral administration

DRUGAlemtuzumab

Intravenous (IV) administration.

DRUGFludarabine

IV administration

DRUGMelphalan

IV administration

DRUGThiotepa

IV administration


Locations(1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

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NCT01962415


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