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RECRUITING Phase 2

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

NCT04356469 · View on ClinicalTrials.gov ↗

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.

Interventions

  • BIOLOGICAL Haploidentical Hematopoietic Cell Transplantation

Study Locations (1)

Florida

  • Johns Hopkins All Children's Hospital — St. Petersburg

Trial Details

FieldValue
Enrollment Target 17 participants
Start Date 2020-07-22
Est. Completion 2026-06-30
Phase Phase 2

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04356469

The ClinicalTrials.gov registry entry for NCT04356469 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 17 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Johns Hopkins All Children's Hospital, which has 32 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT04356469 reports 1 study location spanning 1 distinct geographic area — top geographies include Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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). These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT04356469?

The interventions under investigation include: Haploidentical Hematopoietic Cell Transplantation (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT04356469?

This trial is sponsored by Johns Hopkins All Children's Hospital, which has 32 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial