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Hematopoietic Stem Cell Transplantation

Haploidentical HCT for Primary Immunodeficiency

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
Patients must have adequate organ function measured by: Cardiac: asymptomatic or if symptomatic then LVEF at rest must be ≥ 40% or SF ≥ 26%, Pulmonary: asymptomatic or if symptomatic DLCO ≥ 40% of predicted (corrected for hemoglobin) or pulse oximetry ≥ 92% on room air if the patient is unable to perform pulmonary function testing, Renal: Creatinine clearance (CrCl) or glomerular filtration rate (GFR) must be > 50 mL/min/1.73 m2, Hepatic: Serum conjugated (direct) bilirubin < 2.0 x ULN for age; AST and ALT < 5.0 x ULN for age, Karnofsky or Lansky (age-dependent) performance score ≥ 50, Signed written informed consent
Patient has a suitable genotypic identical match of 5/10. The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1 and HLA-DQB1
Must not have
Patient has HIV or uncontrolled fungal, bacterial or viral infections
Patient has 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 a haploidentical transplant, using TCR alpha beta and CD19 depleted stem cells from a donor. The hope is that this will be a safe and effective way to treat the patient's underlying condition.

Who is the study for?
This trial is for children with primary immunodeficiency or inherited metabolic disorders. Eligible participants include those with various immune system issues, autoimmune diseases, and certain metabolic conditions that could benefit from a stem cell transplant. Pregnant individuals, patients with HIV or uncontrolled infections, prior organ transplants, or severe graft-versus-host disease are excluded.
What is being tested?
The study tests a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cells from half-matched family donors to treat the underlying condition. The process involves collecting stem cells through peripheral blood or bone marrow and filtering them using an investigational device called CliniMACS.
What are the potential side effects?
Potential side effects may include reactions related to the immune system's response to foreign cells (graft-versus-host disease), infection risks due to weakened immunity post-transplantation, and complications arising from the procedure itself.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a donor match for a transplant that matches at least half of the required genetic markers.
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I have a disorder that affects my immune system's function.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have HIV or an uncontrolled infection.
Select...
I have severe graft-versus-host disease from a past transplant.

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
Cellular and Immunological reconstitution by laboratory evaluations
Chronic GvHD
+8 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
45 Previous Clinical Trials
5,008,419 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

Haploidentical Hematopoietic Cell Transplantation (Hematopoietic Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT04414046 — Phase 2
Metabolic Diseases Research Study Groups: TCR alpha beta T cell depletion
Metabolic Diseases Clinical Trial 2023: Haploidentical Hematopoietic Cell Transplantation Highlights & Side Effects. Trial Name: NCT04414046 — Phase 2
Haploidentical Hematopoietic Cell Transplantation (Hematopoietic Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04414046 — Phase 2
~2 spots leftby Jun 2025
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