Myeloablative Preparative Regimen for Shwachman-Diamond Syndrome

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Shwachman-Diamond Syndrome+19 More
Myeloablative Preparative Regimen - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial is collecting data on the outcomes of stem cell transplants for patients with non-cancerous blood disorders.

Eligible Conditions
  • Shwachman-Diamond Syndrome
  • Shwachman syndrome
  • Paroxysmal Nocturnal Haemoglobinuria (PNH)
  • Thrombasthenia
  • Transfusion Dependent Alpha- or Beta- Thalassemia
  • Severe Congenital Neutropenia
  • Congenital Hypoplastic Anemia
  • Non-Malignant Hematologic Disorders
  • Anemia, Sickle Cell

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: 6 months, 1 and 2 years

42 days
incidence of graft failure
Year 2
disease free survival
overall survival

Trial Safety

Trial Design

3 Treatment Groups

Myeloablative Preparative Regimen
1 of 3
Reduced Toxicity Ablative Regimen
1 of 3
Reduced Intensity Preparative Regimen
1 of 3

Experimental Treatment

25 Total Participants · 3 Treatment Groups

Primary Treatment: Myeloablative Preparative Regimen · No Placebo Group · N/A

Myeloablative Preparative Regimen
Drug
Experimental Group · 1 Intervention: Myeloablative Preparative Regimen · Intervention Types: Drug
Reduced Toxicity Ablative Regimen
Drug
Experimental Group · 1 Intervention: Reduced Toxicity Ablative Regimen · Intervention Types: Drug
Reduced Intensity Preparative Regimen
Drug
Experimental Group · 1 Intervention: Reduced Intensity Preparative Regimen · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months, 1 and 2 years

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
253 Previous Clinical Trials
15,045 Total Patients Enrolled
2 Trials studying Shwachman-Diamond Syndrome
32 Patients Enrolled for Shwachman-Diamond Syndrome
Ashish Gupta, MBBS, MPHPrincipal InvestigatorMasonic Cancer Center, University of Minnesota
3 Previous Clinical Trials
52 Total Patients Enrolled

Eligibility Criteria

Age < 65 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have an acceptable source of stem cells.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 6th, 2021

Last Reviewed: November 1st, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.