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CAR T-cell Therapy

Haploidentical Stem Cell Transplant for Blood Diseases

Phase 2
Recruiting
Led By Deepak Chellapandian, MD
Research Sponsored by Johns Hopkins All Children's Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Bone marrow failure syndromes and autoimmune cytopenias meeting specific criteria
Patient with a suitable genotypic identical match of 5/10
Must not have
Participants with an HLA-matched sibling who is able and willing to donate bone marrow
Patients with active GVHD (> grade II) or chronic extensive GVHD due to a previous allograft at the time of inclusion
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years post transplant
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group

Summary

This trial is testing a new way to do haploidentical transplants (using cells from a donor who is a close relative) that may be safer and more effective than the current method.

Who is the study for?
This trial is for children with severe blood diseases like sickle cell, thalassemia, and bone marrow failure who haven't responded to other treatments. They must have specific symptoms or complications of their condition. Kids can't join if they've had a solid organ transplant, active GVHD from previous transplants, an available HLA-matched sibling donor, are pregnant/breastfeeding, have HIV or uncontrolled infections.
What is being tested?
The study tests a new type of stem cell transplant from half-matched family donors using TCR alpha beta and CD19 depleted grafts. It aims to see if this method is safe and effective in treating non-malignant hematological disorders in kids.
What are the potential side effects?
Potential side effects may include reactions related to the immune system's response to foreign cells (GVHD), infection risks due to weakened immunity post-transplantation, and possible infusion-related reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a bone marrow condition or an autoimmune blood issue.
Select...
I have a half-matched donor for a transplant.
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I have a severe form of sickle cell disease.
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I have been diagnosed with Thalassemia major.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a sibling who matches my HLA type and can donate bone marrow.
Select...
I have severe graft-versus-host disease from a past transplant.
Select...
I have HIV or an uncontrolled infection.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years post transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years post transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of successful donor engraftment
Secondary study objectives
Acute grade II-IV GvHD and Chronic GvHD
Cellular and Immunological reconstitution by laboratory evaluations
Blood Platelets
+4 more

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: TCR alpha beta T cell depletionExperimental Treatment1 Intervention
The leukapheresis product will undergo TCR alpha beta negative selection following a standardized protocol

Find a Location

Who is running the clinical trial?

Johns Hopkins All Children's HospitalLead Sponsor
46 Previous Clinical Trials
5,008,467 Total Patients Enrolled
Deepak Chellapandian, MDPrincipal Investigator - Johns Hopkins All Children's Hospital
Johns Hopkins All Children's Hospital
1 Previous Clinical Trials
17 Total Patients Enrolled

Media Library

TCR Alpha Beta T-cell Depleted Haploidentical HCT (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04356469 — Phase 2
Aplastic Anemia Research Study Groups: TCR alpha beta T cell depletion
Aplastic Anemia Clinical Trial 2023: TCR Alpha Beta T-cell Depleted Haploidentical HCT Highlights & Side Effects. Trial Name: NCT04356469 — Phase 2
TCR Alpha Beta T-cell Depleted Haploidentical HCT (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04356469 — Phase 2
~2 spots leftby Jun 2025
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