Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Recruiting Phase 2 study of Metabolic Disease and Primary Immune Deficiency Disorders, sponsored by Johns Hopkins All Children's Hospital.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
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 NCT04414046 shows while recruiting
NCT04414046 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 4,189-participant average among 33 other Metabolic Disease trials with a reported enrollment target (100% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for Metabolic Disease
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 NCT04414046's enrollment target sits among peer trials
17 33rd of 33 higher than 1 of 33 other Metabolic Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Metabolic Disease 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
-
Improving Coronary Vascular Health in Women
RECRUITING · Phase 2
-
NAUTICAL: Effect of Natriuretic Peptide Augmentation on Cardiometabolic Health in Black Individuals
RECRUITING · Phase 2
-
Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency
COMPLETED · Phase 2
-
Metabolic Adaptations to Weight Loss With and Without Exercise
RECRUITING · NA
-
Insomnia Treatment and Cardiometabolic Health in Older Adults With Posttraumatic Stress Disorder
RECRUITING · NA
-
Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
RECRUITING
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02090140 · 17 participants · NA
Microfracture Versus Adipose Derived Stem Cells for the Treatment of Articular Cartilage Defects
- NCT03317860 · 17 participants · NA
Improving Measurement and Treatment of Post-stroke Neglect
- NCT04356469 · 17 participants · Phase 2
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
- NCT04473781 · 17 participants · Phase 1
Interstitial Brachytherapy for the Treatment of Unresectable/Unablatable Kidney Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI