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Hematopoietic Stem Cell Transplantation

Haploidentical Transplant for Severe Aplastic Anemia

Phase 2

Recruiting

Led By Amr Qudeimat, 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

Adequate organ function defined as: Left ventricular ejection fraction > 40% or shortening fraction ≥ 25%. Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/ min/1.73m2. Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry ≥ 92% on room air if patient is unable to perform pulmonary function testing. Karnofsky or Lansky (age-dependent) performance score ≥ 50. Bilirubin ≤ 3 times the upper limit of normal for age. Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence until after the last dose of chemotherapy has been administered

Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.

Must not have

Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as progression or no clinical improvement on appropriate medical treatment.

Known clinical or genetic diagnosis of dyskeratosis congenita

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

All Individual Drugs Already Approved

Approved for 20 Other Conditions

No Placebo-Only Group

Summary

This trial is for patients with Severe Aplastic Anemia who will receive a haploidentical transplantation (from a family member that is not fully matched). The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year.

Who is the study for?

This trial is for patients under 21 with Severe Aplastic Anemia who have failed at least one immunosuppressive therapy, lack a fully matched donor, and have good organ function. Donors must be family members partially matching the patient (≥3 of 6 HLA types), willing to donate stem cells, and HIV negative. Pregnant or breastfeeding women can't participate.

What is being tested?

The study tests haploidentical hematopoietic cell transplantation (HCT) in SAA patients using chemotherapy, radiation, transplanted cells from half-matched family donors, and additional white blood cell infusions. It aims to assess engraftment success at 30 days post-transplantation and survival rates after one year.

What are the potential side effects?

Possible side effects include reactions to medication like Anti-Thymocyte Globulin or Cyclophosphamide such as fever or chills; organ inflammation due to G-CSF; low blood counts from Fludarabine; immune complications like graft-versus-host disease; infection risks due to weakened immunity.

Eligibility Criteria

Inclusion Criteria

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

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I don't have a matching family or unrelated donor for a cell transplant.

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My potential donor is ready and can donate stem cells.

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I am 18 years old or older.

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I am 21 years old or younger.

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I have been diagnosed with severe aplastic anemia or a single blood cell deficiency, confirmed by specific blood and bone marrow tests.

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I have a family member who is at least a half match for a transplant.

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I have a family member who is a partial match for a donation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have an ongoing infection that isn't getting better with treatment.

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I have been diagnosed with dyskeratosis congenita.

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My bone marrow test shows changes linked to early or actual MDS.

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I have been diagnosed with Fanconi anemia, confirmed by specific testing.

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I have had a stem cell transplant from a donor.

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I have antibodies against a donor organ.

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I have not received Alemtuzumab or ATG in the last 2 weeks.

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I have had a solid organ transplant.

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I am not pregnant or breastfeeding.

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I have been diagnosed with myelodysplastic syndrome.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Engraftment

Overall and event free survival

Secondary study objectives

Graft rejection

Graft vs host disease

Immune reconstitution

+1 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.

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

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: Haploidentical HCTExperimental Treatment9 Interventions

To assess the safety and efficacy of haploidentical donor transplantation for patients with severe aplastic anemia who lack an available HLA-matched donor. The goal of this study is to develop a novel, reduced-toxicity, post-transplant pharmacologic immunosuppression (GVHD prophylaxis)- free, highly tolerogenic haploidentical transplant regimen that is associated with few post- transplant complications or late toxicities and is available promptly to all patients, irrespective of matched donor availability. Cells for infusion are prepared using the CliniMACS System.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Coenzyme M

FDA approved

Filgrastim

FDA approved

Total Lymphoid Irradiation (TLI)

2008

Completed Phase 2

~450