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RecruitingPhase 2NCT04232085

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Enrollment

27 participants

Start Date

Feb 12, 2020

Study Type

INTERVENTIONAL

Conditions

Dyskeratosis CongenitaFanconi AnemiaBone Marrow FailureTelomere Biology DisorderPrimary Immune Deficiency DisorderImmune Deficiency DiseaseShort Telomere LengthNon Fanconi DNA-DSB Repair DisorderHoyeraal-Hreidarsson SyndromeShort Telomere Syndrome

Summary

Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.

Eligibility

Min Age: 4 MonthsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Alemtuzumab, a drug called Cyclophosphamide, and others for people with bone marrow failure, dyskeratosis congenita, and other related conditions. The study is currently recruiting participants at 1 location. People eligible for this study include aged 4 Months to 50 Years.

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

DRUGAlemtuzumab

Preparative regimen

DRUGFludarabine

Preparative regimen

DRUGMelphalan

Preparative regimen

RADIATIONLow Dose Total Body Irradiation

Preparative regimen

DRUGCyclophosphamide

GVHD prophylaxis

DRUGTacrolimus

GVHD prophylaxis

DRUGMycophenolate Mofetil

GVHD prophylaxis

Locations(1)

Johns Hopkins University

Baltimore, Maryland, United States

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NCT04232085

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