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NCT04414046 · ClinicalTrials.gov registry record · Phase 2
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children
A Phase 2 study of Metabolic Disease and Primary Immune Deficiency Disorders, sponsored by Johns Hopkins All Children's Hospital.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 17
- Enrollment target
- 1
- Study location
NCT04414046 is a Phase 2 study of Metabolic Disease and Primary Immune Deficiency Disorders that is actively recruiting participants, run by Johns Hopkins All Children's Hospital. The registered enrollment target is 17 participants, below the 4,189-participant average among 33 other Metabolic Disease trials with a reported enrollment target (100% lower). The trial reports 1 study location across 1 state.
The verdict
NCT04414046, a Phase 2 study of Metabolic Disease and Primary Immune Deficiency Disorders, 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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04414046
The ClinicalTrials.gov registry entry for NCT04414046 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 17 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 4,189-participant average among 33 other Metabolic Disease trials with a reported enrollment target (100% lower). The listed sponsor is Johns Hopkins All Children's Hospital, which has 24 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Metabolic Disease appearing as the primary indexed condition, and to 1 intervention - of which Haploidentical Hematopoietic Cell Transplantation is the first listed.
NCT04414046 reports 1 study location spanning 1 distinct geographic area - top geographies include Florida.
Frequently Asked Questions
What is clinical trial NCT04414046 about?
NCT04414046 is a clinical study titled "TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic 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 NCT04414046?
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 NCT04414046 study?
This clinical trial studies the following conditions: Metabolic Disease, Primary Immune Deficiency Disorders.
What interventions are being tested in trial NCT04414046?
The interventions under investigation include: Haploidentical Hematopoietic Cell Transplantation (BIOLOGICAL).
Who is sponsoring clinical trial NCT04414046?
This trial is sponsored by Johns Hopkins All Children's Hospital, which has 24 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04414046 being conducted?
This trial has 1 study location across Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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