← Back to Search

Immunosuppressant

Stem Cell Transplant for Aplastic Anemia

Phase 1

Recruiting

Led By Ryotaro Nakamura

Research Sponsored by City of Hope Medical Center

Eligibility Criteria Checklist

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

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from enrollment until non-disease related death or last follow up and from date of cd4+ t-cell-depleted product infusion, assessed up to 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a reduced-intensity drug regimen prior to a partial-match stem cell transplant to treat severe aplastic anemia. Drugs used may damage cell DNA, block protein needed for cell growth, and suppress immune cells.

Who is the study for?

This trial is for patients aged 40-75 with severe aplastic anemia that's resistant or has returned after treatment. They must have tried immunosuppressive therapy, have a suitable relative as a donor, and meet specific health criteria like proper kidney function and no HIV or hepatitis infections. Women of childbearing age need a negative pregnancy test.

What is being tested?

The study tests if giving cyclophosphamide, pentostatin, and anti-thymocyte globulin before transplanting stem cells from a half-matched donor can be safe and work well for treating severe aplastic anemia. It aims to prepare the body for new cells and reduce disease complications.

What are the potential side effects?

Possible side effects include immune system suppression leading to increased infection risk, potential damage to DNA by cyclophosphamide which could affect cell growth, allergic reactions to anti-thymocyte globulin, organ inflammation, fatigue, nausea, liver enzyme changes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from enrollment until non-disease related death or last follow up and from date of cd4+ t-cell-depleted product infusion, assessed up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from enrollment until non-disease related death or last follow up and from date of cd4+ t-cell-depleted product infusion, assessed up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and from enrollment until non-disease related death or last follow up and from date of cd4+ t-cell-depleted product infusion, assessed up to 3 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Ability to meet CD4+ T-cell depleted product release requirements (feasibility)

Ability to meet minimum required cell dose (feasibility)

Incidence of adverse events

Secondary study objectives

Chimerism

Cumulative incidence of acute GvHD (II-IV and III-IV)

Cumulative incidence of chronic GvHD (any and moderate-severe)

+8 more

Awards & Highlights

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: Treatment (conditioning, haploHCT)Experimental Treatment8 Interventions

Patients receive cyclophosphamide PO and IV, pentostatin IV, anti-thymocyte globulin IV and undergo CD4+ T-cell depleted haploHCT on study. Patients also undergo bone marrow aspirate, bone marrow biopsy, and collection of blood samples at screening and follow-up.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bone Marrow Aspirate

2015

Completed Phase 3

~40

Anti-Thymocyte Globulin

2009

Completed Phase 4

~1040