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Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
NCT04339777 · View on ClinicalTrials.gov ↗
Study Summary
Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications. Objective: To see if stem cell transplant can be successfully performed in people with primary immunodeficiency disease and cure them. Eligibility: People ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory Disorder (PIRD), has caused significant health problems and either standard management has not worked or there are no standard management options, along with their donors Design: Donors will be screened under protocol 01-C-0129. They will donate blood or bone marrow. Participants will be screened with: Medical history Physical exam Blood, urine, and heart tests CT or PET scans Before transplant, participants will have dental and eye exams. They will have a bone marrow biopsy. For this, a needle will be inserted through the skin into the pelvis to remove marrow. Participants will be hospitalized before their transplant. They will have a central catheter put into a vein in their chest or neck. They will get medications through the catheter to prevent complications. Participants will get stem cells through the catheter. They will stay in the hospital for at least 4 weeks. They will give blood, urine, bone marrow, and stool samples. They may need blood transfusions. They may need more scans. They will take more medications. Participants will have visits on days 30, 60, 100, 180, and 360, and 24 months after the transplant. Then they will have visits once a year for about 5 years
Conditions Studied
Interventions
- DRUG Fludarabine
- DRUG Busulfan test dose
- DRUG Busulfan
- DRUG Alemtuzumab
- RADIATION Total body Irradiation
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center — Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 66 participants |
| Start Date | 2020-09-22 |
| Est. Completion | 2027-11-30 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04339777
The ClinicalTrials.gov registry entry for NCT04339777 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 66 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Cancer Institute (NCI), which has 2,390 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 5 conditions, with Immune System Diseases appearing as the primary indexed condition, and to 5 interventions — of which Fludarabine 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: NCT04339777 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 NCT04339777 about?
NCT04339777 is a clinical study titled "Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity". Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications. Objective: To see if stem cel...
What is the current status of trial NCT04339777?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 66 participants. The study started on 2020-09-22. Estimated completion is 2027-11-30.
What conditions does trial NCT04339777 study?
This clinical trial studies the following conditions: Immune System Diseases, Lymphoproliferative Disorders, Common Variable Immunodeficiency, Autoimmune Lymphoproliferative, Primary T-cell Immunodeficiency Disorders. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04339777?
The interventions under investigation include: Fludarabine (DRUG), Busulfan test dose (DRUG), Busulfan (DRUG), Alemtuzumab (DRUG), Total body Irradiation (RADIATION). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04339777?
This trial is sponsored by National Cancer Institute (NCI), which has 2,390 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04339777 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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