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Monoclonal Antibodies

Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders (HSCT+RIC Trial)

Phase 2

Recruiting

Led By Paul Szabolcs, MD

Research Sponsored by Paul Szabolcs

Eligibility Criteria Checklist

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

Must have

A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level matched unrelated donor bone marrow or peripheral blood progenitor graft

Be younger than 65 years old

Must not have

Severe acquired aplastic anemia

Allogeneic hematopoietic stem cell transplant within the previous 6 months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post-transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether a reduced-intensity conditioning regimen followed by umbilical cord blood transplant, double cord blood transplant, matched unrelated donor bone marrow transplant, or peripheral blood stem cell transplant is effective in treating patients with non-malignant disorders.

Who is the study for?

This trial is for patients with non-cancerous disorders like juvenile arthritis, bone marrow failure, primary immunodeficiency, anemia, and metabolic diseases. Participants must have proper organ function, no active malignancy or severe aplastic anemia, not be pregnant or nursing mothers, and should not have had a stem cell transplant in the last 6 months.

What is being tested?

The study tests a reduced-intensity conditioning regimen before umbilical cord blood transplant (UCBT), bone marrow transplant (BMT) from unrelated donors or peripheral blood stem cell transplant (PBSCT). It aims to see how well these transplants work for treating various non-malignant disorders.

What are the potential side effects?

Potential side effects may include reactions to medications like Melphalan and Alemtuzumab such as nausea, fatigue, mouth sores; Hydroxyurea can cause skin rashes; Fludarabine might lead to immune system suppression increasing infection risk.

Eligibility Criteria

Inclusion Criteria

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

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I have a suitable donor for my stem cell transplant.

Exclusion Criteria

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

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I have been diagnosed with severe aplastic anemia.

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I had a stem cell transplant from a donor within the last 6 months.

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I have an active cancer or myelodysplastic syndrome.

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I do not have an ongoing serious infection that is getting worse.

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My high blood pressure in the lungs is not well-managed.

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

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

Primary study objectives

GVHD occurrence

Immune Reconstitution

Neurodevelopmental milestones

+2 more

Secondary study objectives

Donor cell engraftment

Grade 3-4 organ toxicity

Late graft failure

+3 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: UCBT:transfusion dependent anemias or increased rejection riskExperimental Treatment5 Interventions

Day -21 to -19: Alemtuzumab + Hydroxyurea; Day -18 to -10: Hydroxyurea; Day -9 to -5: Fludarabine + Hydroxyurea; Day -4 to -3: Melphalan; Day -2: Thiotepa; Day -1: Rest; Day 0: Transplant

Group II: BMT, PBSCT and not transfusion dependent UCBTExperimental Treatment5 Interventions

Start of conditioning to Day -15: Hydroxyurea; Day -14 to -13: Alemtuzumab + Hydroxyurea; Day -12 to -10: Hydroxyurea; Day -9 to -5: Fludarabine + Hydroxyurea; Day -4 to -3: Melphalan; Day -2: Thiotepa; Day -1: Rest; Day 0: Transplant

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Alemtuzumab

2004

Completed Phase 4

~1880

Melphalan

2008

Completed Phase 3

~1500