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

StatusRecruiting
PhasePhase 2
Started2020-02-12
View on ClinicalTrials.gov ↗

Amendment history

2025-11-21
Study Status, Conditions, Eligibility v11
Eligibility criteria-46 characters
Inclusion Criteria Cohort A: Confirmed diagnosis of: Primary Immune Deficiencies with indication for HCT: Chroni [...] ligase IV deficiency (LIG4) Nijmegen breakage syndrome (NBS) • Increased DNA breakage after exposure of patient cells to D [...] CT). Lack of recipient anti-donor HLA antibody in recipient o Note: In some instances, low level, non-cytotoxic HLA specific antibodies may be permissible if they are found to be at a level well below that detectable by flow cy [...] s. In the event that family members are unaffected carriers, their eligibility as donors will be decided upon by the PI on a case-by-case [...] [...]
2025-06-23
Study Identification, Study Status, Sponsor/Collaborators, Study Description, Conditions, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v10
Study arms2→3
Eligibility criteria+666 characters
Inclusion Criteria Cohort A: Confirmed diagnosis of: Primary Immune Deficiencies with indication for HCT: Chronic granulomatous disease (CGD) Wiskott-Aldrich syndrome (WAS) Hyper-Immunoglobulin M (IgM) syndrome Common variable immunodeficiency (CVID) Leukocyte [...] eficiency-1 (LAD-1) Severe Combined Immunodeficiency (SCID) CTLA-4 deficiency CARD9 deficiency DOCK8 deficiency Immune Dysregulatory Syndromes: Immunodysregulation polyendo [...] ked (IPEX) syndrome Hemophagocytic lymphohistiocytosis (HLH) or related disorder with indication for transplant CAEBV: Patients with chronic EBV infection (CAEBV) with [...] [...]
Secondary endpointsdetails revised at 2 of 2 entries
Study sitescontacts updated at 1 of 1 site
Collaborators+32 characters
Maryland Stem Cell Research Fund
Study title+4 characters
ReducedRegenerative IntensityMedicine BMTto forRestore Hematopoiesis and Immune DysregulatoryFunction in Immunodeficiencies and Inherited Bone Marrow Failure Syndromes Using Post-Transplant CyclophosphamideFailures
2024-12-05
Study Status v9
Primary completion date2024-12-31→2027-12-31
Completion date2026-12-31→2028-12-31
2024-11-20
Study Status v8
Re-verified, no change to tracked fields
2023-11-01
Study Status, Outcome Measures v7
Secondary endpoints20 to 2 entries
Number of patients that have survived at 2 years Event-Free Survival at 1 year Event-Free Survival at 2 years Disease Free Survival at 2 years Number of patients with Sustained Donor Engraftment Number of patients with Graft failure Median day of neutrophil engraftment Median day of platelet engraft [...]
Show 6 earlier versions
2022-10-20
Study Status v6
Re-verified, no change to tracked fields
2021-09-15
Study Status, Eligibility v5
Eligibility criteria+97 characters
[...] (NAT) Female patients who are diagnosed as pregnant by beta h umanhuman chorionic gonadotropin (bHCG) testing (per institutional practice) or who are breast-feeding. Active malignancy or within the timeframe for significant concern for relapse of prior malignancy Donor Eligibility: Donor must be medically, socially, and ps [...]
2020-07-17
Study Status, Contacts/Locations v4
Re-verified, no change to tracked fields
2020-03-11
Study Status v3
Start dateestimated 2020-03-20→confirmed 2020-02-12
2020-01-30
Study Status v2
Start date2020-01-20→2020-03-20
2020-01-17
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Start date2020-01-06→2020-01-20
Study sites0→1
2020-01-14
Original filing
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.
Trial Details
NCT Number NCT04232085
Lead Sponsor Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Collaborators: Maryland Stem Cell Research Fund
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 +2 more
Enrollment 27 participants
Start Date 2020-02-12
Primary Completion 2027-12-31 (estimated)
Study Completion 2028-12-31 (estimated)
Updated on ClinicalTrials.gov 2025-11-28