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Anti-metabolites
Treosulfan-Based Conditioning for Bone Marrow Failure
Phase 2
Recruiting
Research Sponsored by Fred Hutchinson Cancer Research Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
SAMD9 or SAMD9L disorders with a pathogenic mutation(s)
Underlying BMFD treatable by allogenic HCT
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post-hct
Awards & highlights
Study Summary
This trial is testing a new way to prepare for a bone marrow transplant that may cause fewer complications.
Who is the study for?
This trial is for people aged 1 to under 50 with bone marrow failure diseases treatable by transplant, who have specific genetic mutations or meet diagnostic criteria. Excluded are those with certain other conditions, previous transplants, severe lung function impairment, liver issues, uncontrolled infections, HIV positive status or unwillingness to use contraception.Check my eligibility
What is being tested?
The study tests a pre-transplant conditioning regimen using treosulfan combined with fludarabine and rabbit antithymocyte globulin (rATG) in patients with bone marrow failure diseases. The goal is to see if this combination reduces complications post-transplant.See study design
What are the potential side effects?
Potential side effects include immune system reactions due to rATG, organ inflammation from chemotherapy drugs like treosulfan and fludarabine phosphate, increased risk of infection and possible negative impact on fertility.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have a SAMD9 or SAMD9L disorder with a confirmed mutation.
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My bone marrow failure disorder can be treated with a stem cell transplant from a donor.
Select...
I have Shwachman-Diamond syndrome with a confirmed genetic mutation.
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My condition involves a GATA2 mutation causing bone marrow failure.
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I have been diagnosed with paroxysmal nocturnal hemoglobinuria.
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I am between 1 and 49 years old.
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I have Diamond Blackfan Anemia with a confirmed genetic mutation.
Select...
I have a genetic form of anemia known as Sideroblastic anemia.
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I have a genetic condition that affects my platelets.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year post-hct
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post-hct
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Graft-Versus Host-Disease (GVHD)-Free Event-Free Survival (EFS)
Secondary outcome measures
Chronic GVHD
Donor Chimerism (CD3 and Myeloid)
Event-Free Survival
+12 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: Treatment (conditioning regimen; transplant; GVHD prophylaxis)Experimental Treatment13 Interventions
CONDITIONING REGIMEN: Patients receive treosulfan IV over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and rATG IV over 4-6 hours on days -4 to -2.
TRANSPLANTATION: Patients undergo bone marrow or peripheral blood stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV continuously beginning on day -2 and a taper beginning on day 180. Patients may also receive tacrolimus PO. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
Patients undergo ECHO or MUGA during screening and undergo bone marrow biopsy and aspiration at baseline and follow up. Patients may optionally undergo blood sample collection throughout the trial.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Allogeneic Bone Marrow Transplantation
2009
Completed Phase 2
~470
Echocardiography
2013
Completed Phase 4
~11670
Methotrexate
2013
Completed Phase 4
~3800
Peripheral Blood Stem Cell Transplantation
1997
Completed Phase 3
~1330
Bone Marrow Biopsy
2021
Completed Phase 2
~10
Bone Marrow Aspiration
2011
Completed Phase 2
~1740
Biospecimen Collection
2004
Completed Phase 2
~1700
Treosulfan
2009
Completed Phase 3
~2320
Fludarabine Phosphate
1997
Completed Phase 3
~2390
Tacrolimus
2011
Completed Phase 4
~4740
Find a Location
Who is running the clinical trial?
Fred Hutchinson Cancer Research CenterLead Sponsor
443 Previous Clinical Trials
148,332 Total Patients Enrolled
Fred Hutchinson Cancer CenterLead Sponsor
556 Previous Clinical Trials
1,343,355 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,665 Previous Clinical Trials
40,925,814 Total Patients Enrolled
1 Trials studying Shwachman-Diamond Syndrome
4,000 Patients Enrolled for Shwachman-Diamond Syndrome
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am HIV positive.I have had a solid organ transplant.My bilirubin levels are not more than twice the normal limit, unless due to Gilbert's syndrome.You have had a severe, life-threatening reaction to Thymoglobulin in the past.My oxygen level is below 92% without extra oxygen, and I can't do lung tests due to my age or developmental reasons.I have been diagnosed with MDS or leukemia according to WHO standards.My kidney function is reduced, with a creatinine clearance rate under 60 mL/min.I have a SAMD9 or SAMD9L disorder with a confirmed mutation.My bone marrow failure disorder can be treated with a stem cell transplant from a donor.I have Shwachman-Diamond syndrome with a confirmed genetic mutation.My condition involves a GATA2 mutation causing bone marrow failure.I have a diagnosed blood disorder such as aplastic anemia, Fanconi anemia, dyskeratosis congenita, or congenital neutropenia.I have had a stem cell transplant from a donor.My weight is 10 kg or less at the time of joining the study.Your lung test results show that your lung function is less than half of what is expected.I have too much iron in my body due to my condition.I have had cancer before, but it was either skin cancer that was removed or treated cervical cancer.I have been diagnosed with paroxysmal nocturnal hemoglobinuria.I am not pregnant or breastfeeding.I am between 1 and 49 years old.I agree to use birth control or abstain from sex for 12 months post-transplant.I do not have any ongoing serious infections.You are allergic to treosulfan or fludarabine.I am mostly unable to care for myself due to my health condition.I am using supplemental oxygen.I have Diamond Blackfan Anemia with a confirmed genetic mutation.I rely on dialysis for kidney function.You have antibodies that could react against donated cells.My liver enzymes are more than four times the normal limit, or I have severe liver disease.I have a genetic form of anemia known as Sideroblastic anemia.Your heart's pumping ability is less than 50% according to certain heart tests.My bone marrow failure disorder is either not specified or has a genetic mutation not listed, pending approval.I have a genetic condition that affects my platelets.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment (conditioning regimen; transplant; GVHD prophylaxis)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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