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Peripheral Blood Stem Cell Transplantation for Shwachman-Diamond Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Boston Children's Hospital, Boston, MAShwachman-Diamond Syndrome+8 MorePeripheral Blood Stem Cell Transplantation - Procedure
Eligibility
1 - 49
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new way to prepare for a bone marrow transplant that may cause fewer complications.

Eligible Conditions
  • 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

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

1
Cell Therapy
1
Melphalan
1
Allogeneic Hematopoietic Stem Cell Transplantation

Study Objectives

1 Primary · 15 Secondary · Reporting Duration: 1 year post-HCT

1 year post-HCT
Chronic GVHD
Donor Chimerism (CD3 and Myeloid)
Event-Free Survival
Graft-Versus Host-Disease (GVHD)-Free Event-Free Survival (EFS)
Overall Survival
6 months post-HCT
Incidence of grade 2-3 systemic infections
Year 1
Hematologic Recovery: Neutrophil recovery
Secondary graft failure/rejection post-HCT
Day 100 post-HCT
Grade II-IV and grade III-IV GVHD at day 100
Hematologic Recovery: Platelet recovery
Incidence of grade 3-5 toxicities
Day 180 post-HCT
Grade II-IV and grade III-IV GVHD at day 180
Incidence of EBV-associated lymphoproliferative disorder
Incidence of Epstein Barr virus (EBV) reactivation requiring therapy
Day 42 post-HCT
Primary graft failure/rejection
Day 180
Incidence of cytomegalovirus (CMV) reactivation requiring therapy by day 180 post-HCT

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

1
Cell Therapy
1
Melphalan
1
Allogeneic Hematopoietic Stem Cell Transplantation

Trial Design

2 Treatment Groups

treosulfan, fludarabine and Thymoglobulin, tacrolimus and methotrexate
1 of 2
Treatment (conditioning regimen; transplant; GVHD prophylaxis)
1 of 2

Experimental Treatment

40 Total Participants · 2 Treatment Groups

Primary Treatment: Peripheral Blood Stem Cell Transplantation · No Placebo Group · Phase 2

treosulfan, fludarabine and Thymoglobulin, tacrolimus and methotrexateExperimental Group · 5 Interventions: Thymoglobulin (Rabbit Anti-thymocyte Globulin; rATG), Treosulfan, Fludarabine, Tacrolimus, Methotrexate · Intervention Types: Biological, Drug, Drug, Drug, Drug
Treatment (conditioning regimen; transplant; GVHD prophylaxis)Experimental Group · 8 Interventions: Quality-of-Life Assessment, Peripheral Blood Stem Cell Transplantation, Fludarabine Phosphate, Treosulfan, Allogeneic Bone Marrow Transplantation, Lapine T-Lymphocyte Immune Globulin, Tacrolimus, Methotrexate · Intervention Types: Other, Procedure, Drug, Drug, Procedure, Biological, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Peripheral Blood Stem Cell Transplantation
1997
Completed Phase 3
~1360
Fludarabine Phosphate
1997
Completed Phase 3
~2560
Treosulfan
FDA approved
Allogeneic Bone Marrow Transplantation
2009
Completed Phase 2
~440
Fludarabine
FDA approved
Tacrolimus
FDA approved
Methotrexate
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year post-hct

Who is running the clinical trial?

Blood and Marrow Transplant Clinical Trials NetworkNETWORK
49 Previous Clinical Trials
14,001 Total Patients Enrolled
National Marrow Donor ProgramOTHER
59 Previous Clinical Trials
182,409 Total Patients Enrolled
1 Trials studying Shwachman-Diamond Syndrome
22 Patients Enrolled for Shwachman-Diamond Syndrome
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,617 Previous Clinical Trials
47,118,314 Total Patients Enrolled
Fred Hutchinson Cancer CenterLead Sponsor
532 Previous Clinical Trials
1,315,221 Total Patients Enrolled
Fred Hutchinson Cancer Research CenterLead Sponsor
443 Previous Clinical Trials
149,141 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,153 Previous Clinical Trials
41,162,268 Total Patients Enrolled
1 Trials studying Shwachman-Diamond Syndrome
4,000 Patients Enrolled for Shwachman-Diamond Syndrome
Lauri Burroughs, M.D.Study ChairFred Hutch/University of Washington Cancer Consortium
Margaret MacMillan, M.D.Study ChairUniversity of Minnesota
Lauri Burroughs, MDStudy ChairFred Hutch/University of Washington Cancer Consortium
Margaret MacMillan, MDStudy ChairUniversity of Minnesota
5 Previous Clinical Trials
72 Total Patients Enrolled

Eligibility Criteria

Age 1 - 49 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a medical condition called Shwachman-Diamond syndrome.
You have a specific genetic mutation that causes Shwachman-Diamond syndrome.
Your pancreatic isoamylase levels (for ages 3 and up) or fecal elastase levels are too low.
You have Diamond Blackfan Anemia.
You have low red blood cell count, large red blood cells, low platelet count without any clear cause or low cell count in your bone marrow.
You have a specific genetic mutation that causes Diamond Blackfan anemia.
You have a medical history where there were not enough red blood cells in your bone marrow, even though the bone marrow had normal overall cell count.
You have a bone marrow disorder that can be treated with a bone marrow transplant from a donor.
You must be at least 1 year old but younger than 50 years old to participate in the study.
You have severe anemia (low red blood cell count) or severe thrombocytopenia (low platelet count) and may require frequent blood transfusions. Your eligibility will be reviewed by a committee if you have additional medical conditions or infections.
References

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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