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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years post transplant

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

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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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years post transplant

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of successful donor engraftment

Secondary outcome measures

Acute grade II-IV GvHD and Chronic GvHD

Cellular and Immunological reconstitution by laboratory evaluations

Blood Platelets

+4 more

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

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Johns Hopkins All Children's HospitalLead Sponsor

43 Previous Clinical Trials

5,007,522 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this investigation accepting new participants at the present time?

"According to the official listing on clinicaltrials.gov, enrollment is still open for this trial. It was first posted in July of 2020 and its data has been updated most recently as of September 13th 2022."

Answered by AI

What is the cohort size for this experiment?

"Affirmative. Clinicaltrials.gov hosts the information that this medical trial, which commenced on July 22nd 2020, is currently recruiting for 17 participants from a singular site."

Answered by AI

Who is eligible to join this medical investigation?

"For this clinical trial, 17 participants aged 0 to 21 with anemia and aplastic anemia are being admitted. In addition to these criteria, potential candidates must have had a cerebrovascular accident lasting longer than 24 hours; recurrent (≥ 3 in lifetime) acute chest syndrome events necessitating erythrocyte transfusion therapy; impaired neuropsychological function with abnormal brain MRI/MRA; frequent (≥ 3 per year for preceding 2 years) painful vaso-occlusive episodes or any combination of ≥ 3 acute chest syndrome episodes and vaso-occlusive pain episodes yearly for 3 years plus failure treatment by"

Answered by AI

Is this experiment open to participants aged 50 or older?

"The conditions for inclusion in this medical trial mandate that participants are aged between 0 and 21 years old. There are 85 trials specifically targeting those under 18, while 88 have been set up to accommodate individuals over 65."

Answered by AI

Has Haploidentical Hematopoietic Cell Transplantation been granted authorization by the FDA?

"The safety of Haploidentical Hematopoietic Cell Transplantation receives a score of 2, as the Phase 2 trial only provides evidence to suggest its security and not yet any efficacy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

Deepak Chellapandian, MD MBBS 2020. "TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04356469.

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