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

NCT04232085 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2025-11-28

No results posted yet for this study

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.

Conditions

  • Primary Immune Deficiency Disorder
  • Immune Deficiency Disease
  • Bone Marrow Failure
  • Short Telomere Length
  • Fanconi Anemia
  • Non Fanconi DNA-DSB Repair Disorder
  • Hoyeraal-Hreidarsson Syndrome
  • Dyskeratosis Congenita
  • Telomere Biology Disorder
  • Short Telomere Syndrome

Interventions

DRUG

Fludarabine

Preparative regimen

DRUG

Melphalan

Preparative regimen

RADIATION

Low Dose Total Body Irradiation

Preparative regimen

DRUG

Cyclophosphamide

GVHD prophylaxis

DRUG

Tacrolimus

GVHD prophylaxis

DRUG

Mycophenolate Mofetil

GVHD prophylaxis

DRUG

Alemtuzumab

Preparative regimen

Sponsors & Collaborators

  • Maryland Stem Cell Research Fund

    collaborator UNKNOWN

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    lead OTHER

Principal Investigators

  • Heather J Symons, MD, MHS · Johns Hopkins University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
4 Months
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-02-12
Primary Completion
2027-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04232085 on ClinicalTrials.gov