Peripheral Blood Stem Cell Transplantation for Shwachman-Diamond Syndrome
This trial is testing a new way to prepare for a bone marrow transplant that may cause fewer complications.
- Shwachman-Diamond Syndrome
- Bone Marrow Failure Syndrome
- Congenital Pure Red Cell Aplasia
- GATA2-Related Myeloid Neoplasms
- Paroxysmal Nocturnal Hemoglobinuria
- Hereditary Sideroblastic Anemia
- Congenital Amegakaryocytic Thrombocytopenia
1 Primary · 15 Secondary · Reporting Duration: 1 year post-HCT
2 Treatment Groups
treosulfan, fludarabine and Thymoglobulin, tacrolimus and methotrexate
1 of 2
Treatment (conditioning regimen; transplant; GVHD prophylaxis)
1 of 2
40 Total Participants · 2 Treatment Groups
Primary Treatment: Peripheral Blood Stem Cell Transplantation · No Placebo Group · Phase 2
Who is running the clinical trial?
Age 1 - 49 · All Participants · 10 Total Inclusion CriteriaMark “Yes” if the following statements are true for you:
Frequently Asked Questions
How many volunteers are participating in this research?
"That is correct, the online clinicaltrials.gov registry lists this trial as open and recruiting patients. This particular study was originally posted on April 19th, 2020 and was last updated October 12th, 2020. The researchers are looking for a total of 40 participants across 28 different sites." - Anonymous Online Contributor
Could I possibly take part in this clinical trial?
"Children between the ages of 1 year and 49 who have been diagnosed with shwachman-diamond syndrome are ideal candidates for this clinical trial, which is looking to enroll a total of 40 patients." - Anonymous Online Contributor
How many different medical clinics are running this clinical trial today?
"Altogether, there are 28 clinical sites running this study. This includes locations such as Primary Children's/University of Utah in Salt Lake City, MD Anderson Cancer Center in Columbus, and Nationwide Children's Hospital in Nashville." - Anonymous Online Contributor
What are some of the more common reasons why a doctor would choose to prescribe Treosulfan?
"Treosulfan is an effective treatment against non-hodgkin lymphoma, meningeal leukemia, and bladder cancer." - Anonymous Online Contributor
What other medical studies have included Treosulfan in their research?
"703 clinical trials are underway that involve Treosulfan with 110 of those being in the third and final stage. The majority of these studies originate from Philadelphia, Pennsylvania; however, there are 11927 locations running these sorts of investigations." - Anonymous Online Contributor
Has the US Food and Drug Administration accepted Treosulfan for therapeutic use?
"While Phase 2 trials don't have the same robust data sets as later Phase trials, our team estimats that Treosulfan is a safe medication." - Anonymous Online Contributor
Would this experiment still be beneficial for individuals outside the target age range?
"This study's requirements for inclusion are that potential participants must be between 1 Year and 49. Out of the 686 studies done on people under 18, 1686 were conducted with individuals over 65." - Anonymous Online Contributor
Are patients still being recruited for this clinical trial?
"Yes, this trial is still enrolling patients according to the information available on clinicaltrials.gov. The study was first announced on April 19th, 2022 and received its most recent update on October 12th of the same year." - Anonymous Online Contributor