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Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
NCT04232085 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Fludarabine
- DRUG Melphalan
- DRUG Alemtuzumab
- RADIATION Low Dose Total Body Irradiation
Study Locations (1)
Maryland
- Johns Hopkins University — Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 27 participants |
| Start Date | 2020-02-12 |
| Est. Completion | 2028-12-31 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04232085
The ClinicalTrials.gov registry entry for NCT04232085 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 27 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, which has 311 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 10 conditions, with Fanconi Anemia appearing as the primary indexed condition, and to 5 interventions — of which Cyclophosphamide is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04232085 reports 1 study location spanning 1 distinct geographic area — top geographies include Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04232085 about?
NCT04232085 is a clinical study titled "Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures". 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 bo...
What is the current status of trial NCT04232085?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 27 participants. The study started on 2020-02-12. Estimated completion is 2028-12-31.
What conditions does trial NCT04232085 study?
This clinical trial studies the following conditions: Fanconi Anemia, Dyskeratosis Congenita, Bone Marrow Failure, Primary Immune Deficiency Disorder, Telomere Biology Disorder. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04232085?
The interventions under investigation include: Cyclophosphamide (DRUG), Fludarabine (DRUG), Melphalan (DRUG), Alemtuzumab (DRUG), Low Dose Total Body Irradiation (RADIATION). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04232085?
This trial is sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, which has 311 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04232085 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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