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NCT04356469 · ClinicalTrials.gov registry record · Phase 2
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
A Phase 2 study of Severe Aplastic Anemia and Bone Marrow Failure Syndrome, sponsored by Johns Hopkins All Children's Hospital.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 17
- Enrollment target
- 1
- Study location
NCT04356469: Recruiting Phase 2 study of Severe Aplastic Anemia and Bone Marrow Failure Syndrome, sponsored by Johns Hopkins All Children's Hospital.
NCT04356469 is a Phase 2 study of Severe Aplastic Anemia and Bone Marrow Failure Syndrome that is actively recruiting participants, run by Johns Hopkins All Children's Hospital. The registered enrollment target is 17 participants, below the 126-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (87% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04356469, a Phase 2 study of Severe Aplastic Anemia and Bone Marrow Failure Syndrome, is actively recruiting participants, sponsored by Johns Hopkins All Children's Hospital.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 17 participants
- Enrollment target
- 1
- Study location
Study Summary
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
Primary Outcome
The incidence of engraftment at day 100 will be described based on donor chimerism in the whole blood and or fractions sorted for T-cell and myeloid subsets. The donor chimerism will be scored as autologous reconstitution (\< 5% donor), mixed chimerism (5-49%=low mixed, 50-95%=high mixed), \> 95%=full donor chimerism.
Conditions Studied
Interventions
- BIOLOGICAL Haploidentical Hematopoietic Cell Transplantation
Study Locations (1)
Florida
- Johns Hopkins All Children's Hospital - St. Petersburg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2020-07-22 |
| Est. Completion | 2026-06-30 |
| Phase | Phase 2 |
What NCT04356469 shows while recruiting
NCT04356469 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 17 participants, a relatively small participant target, below the 126-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (87% lower).
The record links to 3 conditions, with Severe Aplastic Anemia appearing as the primary indexed condition, and to 1 intervention - of which Haploidentical Hematopoietic Cell Transplantation is the first listed.
NCT04356469 reports a single indexed study location in Florida.
Frequently Asked Questions
What is clinical trial NCT04356469 about?
NCT04356469 is a clinical study titled "TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children". This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow fr...
What is the current status of trial NCT04356469?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 17 participants. The study started on 2020-07-22. Estimated completion is 2026-06-30.
What conditions does trial NCT04356469 study?
This clinical trial studies the following conditions: Severe Aplastic Anemia, Bone Marrow Failure Syndrome, Hemoglobinopathy (Disorder).
What interventions are being tested in trial NCT04356469?
The interventions under investigation include: Haploidentical Hematopoietic Cell Transplantation (BIOLOGICAL).
Who is sponsoring clinical trial NCT04356469?
This trial is sponsored by Johns Hopkins All Children's Hospital, which has 24 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04356469 being conducted?
This trial has 1 study location across Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Severe Aplastic Anemia
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 NCT04356469's enrollment target sits among peer trials
17 16th of 16 higher than 1 of 16 other Severe Aplastic Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Severe Aplastic Anemia 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
-
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
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-
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