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Alkylating agent

Haplocompatible Transplant for Severe Combined Immunodeficiency

Phase 1 & 2

Waitlist Available

Led By Ewelina Mamcarz, MD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age ≥2 months old at the time of chemotherapy administration

Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/min/1.73m2 or serum Creatinine ≤1.2mg/dL

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post transplant

Awards & highlights

Study Summary

This trial is for infants with SCID who lack a sibling donor. The goal is to provide a conditioning regimen that is well tolerated and provision of immune cells that altogether should establish rapid immune recovery providing protection from life threatening infections without increasing the risk of dangerous Graft-Versus-Host-Disease.

Who is the study for?

This trial is for infants over 2 months old with severe combined immunodeficiency (SCID) who have a genetic mutation confirmed by DNA sequencing. They need to have a suitable sibling or unrelated donor, or a family member donor that's at least half-matched. Their heart and kidneys must function well, they should be able to breathe without assistance, and their liver enzymes should not be too high.Check my eligibility

What is being tested?

The study tests a transplant method using TCRα/β depletion followed by CD45RA-depleted donor lymphocyte infusions in SCID patients lacking sibling donors. The goal is to establish rapid immune recovery with tolerable conditioning and reduce the risk of Graft-Versus-Host-Disease while monitoring survival rates after one year.See study design

What are the potential side effects?

Potential side effects may include reactions to anti-thymocyte globulin like fever and chills; chemotherapy-related nausea, vomiting, mouth sores; busulfan can cause seizures; thiotepa might lead to hair loss; risks from the infusion include infection and Graft-Versus-Host Disease.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am at least 2 months old.

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My kidney function is normal or only slightly reduced.

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My liver enzymes are within 5 times the normal limit.

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My DNA test shows a specific genetic mutation.

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I can do most activities but need help with some.

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My bilirubin levels are within three times the normal limit for my age.

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I have a donor match for my condition and my heart is functioning well.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year post transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year post transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year 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

Number of overall grade 3-4 acute Graft-Versus-Host-Disease (GVHD)

Number of treatment related deaths

Overall Survival(OS)

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Donor Lymphocyte InfusionsExperimental Treatment2 Interventions

Phase I: On the Phase I portion of the study, up to 4 different dose levels will be evaluated: Dose level -1, Dose ≥0.1 to ≤0.3; Dose level 1, Dose >0.3 to ≤0.56; Dose level 2, Dose >0.56 to ≤1.8; Dose level 3, Dose >1.80 to ≤3.0 Dosing is determined based on the number of CD3+CD45RA-cells/kg and the patient weight in kilograms. Phase II: Participants will receive the Phase I determined maximum tolerated dose (MTD) of DLI. Cells for infusion are prepared using the CliniMACS System.

Group II: TCRα/β/CD19-depleted SCTActive Control5 Interventions

A preparative regimen based on the type of SCID will be given followed by infusion of donor cells. Cells for infusion are prepared using the CliniMACS System Regimen 1 - IL2RG, JAK 3 (Haplocompatible) and all MSD ATG (rabbit) IV Days -9 -8 and -7, Rest Days -6 and -5, Busulfan IV Days -4, -3, and -2, Rest Day -1, TCRα/β/CD19-depleted SCT, Day 0 Regimen 2 - RAG1, RAG2 (Haplocompatible) ATG (rabbit) IV Days -9 -8 and -7, Fludarabine IV Days -7, -6, -5 and -4, Busulfan IV Days -5, -4 and -3, Thiotepa IV twice daily, Day -2, Rest Day -1, TCRα/β/CD19-depleted SCT, Day 0 Regimen 3 - ADA, IL7R, CD45 deficiency, CD3 subunits (Haplocompatible) ATG (rabbit) IV Days -9 -8 and -7, Fludarabine IV Days -7, -6, -5 and -4, Busulfan: IV Days -4, -3 and -2, Rest Day -1, TCRα/β/CD19-depleted SCT, Day 0

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CliniMACS

2005

Completed Phase 3

~770

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

St. Jude Children's Research HospitalLead Sponsor

428 Previous Clinical Trials

5,306,609 Total Patients Enrolled

3 Trials studying Severe Combined Immunodeficiency

1,032 Patients Enrolled for Severe Combined Immunodeficiency

Ewelina Mamcarz, MDPrincipal InvestigatorSt. Jude Children's Research Hospital

Media Library

Busulfan (Alkylating agent) Clinical Trial Eligibility Overview. Trial Name: NCT03597594 — Phase 1 & 2

Severe Combined Immunodeficiency Research Study Groups: TCRα/β/CD19-depleted SCT, Donor Lymphocyte Infusions

Severe Combined Immunodeficiency Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT03597594 — Phase 1 & 2

Busulfan (Alkylating agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03597594 — Phase 1 & 2

Frequently Asked Questions

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

For what ailment is Donor Lymphocyte Infusion typically administered?

"Donor Lymphocyte Infusion is typically used to aid in the management of polycythemia vera (pv), but can also be utilized for other afflictions such as immunosuppressive treatment, cancer and kidney disorders."

Answered by AI

Are there any historical precedents demonstrating the efficacy of Donor Lymphocyte Infusion?

"As of now, 456 clinical trials for Donor Lymphocyte Infusion are in progress. Of those currently running studies, 40 have entered Phase 3 status. Philadelphia, Pennsylvania houses the majority of these experiments; however there are 5289 test centres worldwide offering this procedure."

Answered by AI

Is it still possible for individuals to join this clinical exploration?

"As published on clinicaltrials.gov this investigation is actively recruiting patients, having been initially posted on September 8th 2021 and most recently revised October 6th 2022."

Answered by AI

What is the current tally of participants in this clinical investigation?

"Affirmative. Clinicaltrials.gov validates that this clinical trial, which was inaugurated on September 8th 2021, is actively recruiting subjects. There are 42 participants sought to be enrolled from a single location."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)

2021. "Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03597594.