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Hematopoietic Stem Cell Transplantation

Haploidentical HCT for Primary Immunodeficiency

Q: Are there any available opportunities for individuals to join this experiment?

Affirmative. Information found on clinicaltrials.gov confirms that this scientific research, which was first presented on July 22nd 2020, is actively enrolling participants. 17 volunteers need to be enrolled from 1 medical centre.

Q: Does the FDA sanction Haploidentical Hematopoietic Cell Transplantation?

Haploidentical Hematopoietic Cell Transplantation's safety was assigned a rating of 2 due to the Phase 2 nature of its clinical trial, which has provided some evidence towards its security but no proof that it is effective.

Q: What is the current capacity of this clinical trial?

Affirmative. According to clinicaltrials.gov, this medical experiment which was initially posted on July 22nd 2020 is actively recruiting participants. Seventeen patients need to be recruited from one location for the study's completion.

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

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 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.Check my eligibility

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.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 (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

Select...

I have a donor match for a transplant that matches at least half of the required genetic markers.

Select...

I have a disorder that affects my immune system's function.

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

Cellular and Immunological reconstitution by laboratory evaluations

Chronic GvHD

+8 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 / 3Eligibility 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

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

Frequently Asked Questions

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

Are there any available opportunities for individuals to join this experiment?

"Affirmative. Information found on clinicaltrials.gov confirms that this scientific research, which was first presented on July 22nd 2020, is actively enrolling participants. 17 volunteers need to be enrolled from 1 medical centre."

Answered by AI

Does the FDA sanction Haploidentical Hematopoietic Cell Transplantation?

"Haploidentical Hematopoietic Cell Transplantation's safety was assigned a rating of 2 due to the Phase 2 nature of its clinical trial, which has provided some evidence towards its security but no proof that it is effective."

Answered by AI

What is the current capacity of this clinical trial?

"Affirmative. According to clinicaltrials.gov, this medical experiment which was initially posted on July 22nd 2020 is actively recruiting participants. Seventeen patients need to be recruited from one location for the study's completion."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

Deepak Chellapandian, MD MBBS 2020. "TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04414046.

All > Primary Immunodeficiency