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NCT01998633 · ClinicalTrials.gov registry record · Phase 2
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
A Phase 2 study, sponsored by Medical College of Wisconsin.
- Completed
- Registry status
- Phase 2
- Development phase
- 47
- Enrollment target
NCT01998633: Completed Phase 2 study, sponsored by Medical College of Wisconsin.
NCT01998633 is a Phase 2 study that has completed, run by Medical College of Wisconsin. The registered enrollment target is 47 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01998633, a Phase 2 study, has completed, sponsored by Medical College of Wisconsin.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 47 participants
- Enrollment target
Study Summary
HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal without Hematopoietic Cell Transplant (HCT). However, transplant is often complicated by inflammation, infection and other co-morbidities. In addition, these disorders have been shown to be cured with partial chimerism, making them an ideal target for the use of reduced intensity approaches, where a portion of patients may not achieve full donor chimerism, but instead achieve stable mixed chimerism. Reduced-intensity conditioning strategies have demonstrated improved survival with decreased Treatment Related Mortality (TRM) in institutional series for patients with HLH (Cooper et al., 2006; Marsh et al., 2010; Marsh et al., 2011). However, graft loss and unstable chimerism remain challenges. An institutional case series from Cincinnati Children's Hospital demonstrated full or high-level chimerism and improved durable engraftment using intermediate (Day -14) timing alemtuzumab (Marsh et al., 2013b). This study aims to test the efficacy of the Intermediate RIC strategy in a prospective multi-center study including HLH as well as other primary immunodeficiencies where allogeneic transplant with RIC has been shown to be feasible and stable chimerism is curative.
Primary Outcome
Overall survival is defined as survival of death from any cause.
Interventions
- BIOLOGICAL Hematopoietic Stem Cell Transplant
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 47 participants |
| Start Date | 2013-12 |
| Est. Completion | 2016-12 |
| Phase | Phase 2 |
What the finished NCT01998633 record still lists
NCT01998633 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 47 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower).
The record links to 0 conditions, and to 1 intervention - of which Hematopoietic Stem Cell Transplant is the first listed.
NCT01998633 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01998633 about?
NCT01998633 is a clinical study titled "Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)". HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal without Hematopoietic Cell Transplant (HCT). However, transplant is ofte...
What is the current status of trial NCT01998633?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 47 participants. The study started on 2013-12. Estimated completion is 2016-12.
What interventions are being tested in trial NCT01998633?
The interventions under investigation include: Hematopoietic Stem Cell Transplant (BIOLOGICAL).
Who is sponsoring clinical trial NCT01998633?
This trial is sponsored by Medical College of Wisconsin, which has 447 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01998633's enrollment target sits among peer trials
47 1283rd of 2000 higher than 715 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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