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

ConditionsPrimary Immune Deficiency DisorderImmune Deficiency DiseaseBone Marrow FailureShort Telomere LengthFanconi AnemiaNon Fanconi DNA-DSB Repair DisorderHoyeraal-Hreidarsson SyndromeDyskeratosis CongenitaTelomere Biology DisorderShort Telomere Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age4-50
SponsorSidney Kimmel Comprehensive Cancer Center at Johns Hopkins

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

Chronic granulomatous disease (CGD)

Wiskott-Aldrich syndrome (WAS)

Hyper-IgM syndrome

Common variable immunodeficiency (CVID)

Disqualifiers

Patients will not be excluded on the basis of sex, racial or ethnic background.

Positive leukocytotoxic crossmatch.

Prior allogeneic stem cell transplant.

Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment. The investigators recognize that patients with CAEBV may have ongoing EBV viremia at the time of initiating pre-transplant therapy, but other patients should have no uncontrolled bacterial, viral, or fungal infections.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Alemtuzumab

    Drug

    Preparative regimen

  • Fludarabine

    Drug

    Preparative regimen

  • Melphalan

    Drug

    Preparative regimen

  • Low Dose Total Body Irradiation

    Radiation

    Preparative regimen

  • Cyclophosphamide

    Drug

    GVHD prophylaxis

  • Tacrolimus

    Drug

    GVHD prophylaxis

  • Mycophenolate Mofetil

    Drug

    GVHD prophylaxis

Treatment groups

27 Participants
are divided into 3 treatment groups
Group A: PID/IDS/IBMFSExperimental treatment 7 interventions
Group B: Short Telomere SyndromeExperimental treatment 6 interventions
Group C: Fanconi Anemia or DNA-dsb repairExperimental treatment 6 interventions

Trial outcomes

Primary outcomes

1

Donor Engraftment

Rate of donor engraftment ≥95% at day 60 as measured by donor chimerism in whole blood.

Time frame
60 Days

Secondary outcomes

1

Number of patients that have survived at 1 year

Overall Survival (Defined as alive) in patients receiving 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, Fanconi Anemia and non-Fanconi DNA dsb repair.

Time frame
1 year
2

Disease Free Survival at 1 year

Disease-Free Survival (defined as without recurrence of underlying disease, without graft failure, and alive) in patients receiving 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, Fanconi Anemia and non-Fanconi DNA dsb repair.

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Lead sponsor

Maryland Stem Cell Research Fund

Collaborator

Source ClinicalTrials.gov
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