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Immunosuppressant

Stem Cell Transplant for Bone Marrow Failure Syndromes

Phase 2 & 3
Waitlist Available
Led By Paul Orchard, MD
Research Sponsored by Masonic Cancer Center, University of Minnesota
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
No Placebo-Only Group

Summary

The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

Eligible Conditions
  • Shwachman-Diamond Syndrome
  • Diamond-Blackfan Anemia
  • Kostmann Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 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
Number of Patients Alive (Survival) at 2 Years
Secondary study objectives
Number of Patients Alive at Three Years (Survival)
Number of Patients With Chronic Graft Versus Host Disease
Number of Patients With Disease Recurrence
+2 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 5 Other Conditions
This treatment demonstrated efficacy for 5 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: Bone Marrow Failure DisordersExperimental Treatment5 Interventions
Patients with Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
anti-thymocyte globulin
2005
Completed Phase 3
~1000
Fludarabine
FDA approved
Total lymphoid irradiation
2009
Completed Phase 3
~60
Busulfan
FDA approved
Stem cell transplant
2011
Completed Phase 3
~700

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
281 Previous Clinical Trials
15,597 Total Patients Enrolled
2 Trials studying Shwachman-Diamond Syndrome
47 Patients Enrolled for Shwachman-Diamond Syndrome
Paul Orchard, MDPrincipal InvestigatorUniversity of Minnesota Medical Center
14 Previous Clinical Trials
382 Total Patients Enrolled
~0 spots leftby Dec 2025
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