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NCT04339777 · ClinicalTrials.gov registry record · Phase 2
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
A Phase 2 study of Immune System Diseases and Lymphoproliferative Disorders, sponsored by National Cancer Institute (NCI).
- Recruiting
- Registry status
- Phase 2
- Development phase
- 66
- Enrollment target
- 1
- Study location
NCT04339777: Recruiting Phase 2 study of Immune System Diseases and Lymphoproliferative Disorders, sponsored by National Cancer Institute (NCI).
NCT04339777 is a Phase 2 study of Immune System Diseases and Lymphoproliferative Disorders that is actively recruiting participants, run by National Cancer Institute (NCI). The registered enrollment target is 66 participants, below the 149-participant average among 18 other Immune System Diseases trials with a reported enrollment target (56% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04339777, a Phase 2 study of Immune System Diseases and Lymphoproliferative Disorders, is actively recruiting participants, sponsored by National Cancer Institute (NCI).
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 66 participants
- Enrollment target
- 1
- Study location
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
Primary Outcome
neutrophil recovery with ANC \>/= 500/mm\^3 for 3 consecutive days with \>50% or \>75% T-cell and myeloid donor chimerism
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 |
What NCT04339777 shows while recruiting
NCT04339777 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 66 participants, a relatively small participant target, below the 149-participant average among 18 other Immune System Diseases trials with a reported enrollment target (56% lower).
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.
NCT04339777 reports a single indexed study location in Maryland.
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.
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).
Who is sponsoring clinical trial NCT04339777?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04339777's enrollment target sits among peer trials
66 8th of 18 higher than 11 of 18 other Immune System Diseases trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Immune System Diseases trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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