A Phase 2 interventional study of Stem Cell Transplantation and Cyclophosphamide in Hurler's Syndrome, Maroteaux-Lamy Syndrome and Sly Syndrome, sponsored by Masonic Cancer Center, University of Minnesota. Terminated at 1 site in United States. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2017-12-28.
Sponsored by Masonic Cancer Center, University of Minnesota · Phase 2, Interventional, and Treatment
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).
This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this is a new protocol to take the place of several older protocols. While survival has been very good on the prior protocols over the past decade, incomplete engraftment has remained somewhat problematic. Therefore, we have modified the preparative regimen somewhat to increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that is more immune suppressive. In addition, we have modified the supportive care regimen. Based on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers of inflammation and oxidative stress for the families that consent to these research studies.
74 studies on the registry are indexed under Mucopolysaccharidosis I; 7 are open to participants now.
This study's enrollment of 18 is above the median of 14 across 47 interventional studies indexed under Mucopolysaccharidosis I.
Browse Mucopolysaccharidosis I studies →Masonic Cancer Center, University of Minnesota is the lead sponsor of 286 studies on the registry; 35 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Mucopolysaccharidosis (MPS) Disorders:
Glycoprotein metabolic disorders:
Other Inherited Diseases of Metabolism:
Exclusion Criteria:
Major organ dysfunction. Evidence of major organ impairment, including:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All patients treated with study regimen.
Procedure: Stem Cell Transplantation · Drug: Cyclophosphamide · Drug: Campath-1H · Drug: Busulfan
The purpose of hematopoietic stem cell transplantation is to introduce blood producing cells from a normal donor. These cells can either provide what is missing in the body to the other cells, or can change the body's immune response to the substances that have accumulated in the body. These normal hematopoietic stem cells can come from bone marrow, peripheral blood (i.e., the blood circulating in our body's blood vessels) or umbilical cord blood (i.e., blood taken from the umbilical cord after a baby is born and umbilical cord is cut). The new donor cells repopulate the blood and bone marrow system and enter the organs of the body, including the brain. Wherever these cells go, they will produce the needed enzyme.
Also known as: Bone Marrow Transplant, cord blood transplant
Days before Transplant Drug Frequency * 4 Cyclophosphamide Once, given over 2 hours * 3 Cyclophosphamide Once, given over 2 hours * 2 Cyclophosphamide Once, given over 2 hours * 1 Cyclophosphamide Once, given over 2 hours
Also known as: Cytoxan
Days before Transplant Drug Frequency 12 Campath-1H Once, given over 2 hours 11 Campath-1H Once, given over 2 hours 10 Campath-1H Once, given over 2 hours
Also known as: Alemtuzamab
Days before Transplant Drug Frequency 9 Busulfan Four times per day 8 Busulfan Four times per day 7 Busulfan Four times per day 6 Busulfan Four times per day
Also known as: Busulfex
Number of Patients Achieving Engraftment
Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.
Time frame: Day 100
Overall Survival
Number of patients alive at timepoints.
Time frame: Day 100, 1 Year, 3 Years
| Milestone | Intent-to-Treat |
|---|---|
| Started | 18 |
| Completed | 18 |
| Not completed | 0 |
Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.
| Participants | Intent-to-Treat |
|---|---|
| Number of Patients Achieving Engraftment | 14 |
Number of patients alive at timepoints.
| Participants | Intent-to-Treat |
|---|---|
| Day 100 | 14 |
| 1 Year | 12 |
Collected over Day 1 through Day 100 post-transplant.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Intent-to-Treat | — | 8/18 (44.4%) | 0/18 (0%) |
| Event | Intent-to-Treat |
|---|---|
| DeathGeneral disorders | 6/18 |
| Graft failureBlood and lymphatic system disorders | 2/18 |
| Auto recoveryGeneral disorders | 2/18 |
| Age, Categorical(Participants) | Intent-to-Treat |
|---|---|
| <=18 years | 18 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | Intent-to-Treat |
|---|---|
| Mean | 4.7 ± 4.3 |
| Sex: Female, Male(Participants) | Intent-to-Treat |
|---|---|
| Female | 7 |
| Male | 11 |
| Region of Enrollment(participants) | Intent-to-Treat |
|---|---|
| United States | 18 |
This study is terminated, as verified in Dec 2017. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Cyclophosphamide · Alemtuzumab · Busulfan
Masonic Cancer Center, University of Minnesota →