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Chemotherapy

Stem Cell Transplantation for Osteopetrosis

Phase 2
Recruiting
Led By Paul Orchard, M.D.
Research Sponsored by Masonic Cancer Center, University of Minnesota
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Severe Osteopetrosis (OP)
Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study Summary

This trial tests a new way to do bone marrow transplants that may be safer for people with certain disorders.

Who is the study for?
This trial is for patients aged 0-55 with various inherited metabolic disorders like Hurler syndrome, Hunter syndrome without severe neurologic disease, and severe osteopetrosis. Participants must have a suitable stem cell donor, good organ function, and no recent myeloablative chemotherapy or uncontrolled infections.Check my eligibility
What is being tested?
The study tests the effectiveness of busulfan- and fludarabine-based conditioning regimens with busulfan drug monitoring in achieving successful stem cell engraftment while minimizing transplant-related mortality in patients with inherited metabolic disorders and osteopetrosis.See study design
What are the potential side effects?
Potential side effects include reactions to medication such as nausea, vomiting, diarrhea; risk of infection; mouth sores; fatigue; liver problems indicated by changes in skin/eye coloration; low blood counts leading to bleeding or bruising.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with severe Osteopetrosis.
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I have been diagnosed with MNGIE.
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I am between 0 and 55 years old.
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I have been diagnosed with Infantile Refsum disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percent of subjects who achieve high-level donor hematopoietic engraftment
Secondary outcome measures
Graft-versus-host disease
Post-HSCT changes in disease
Regimen-related toxicity
+1 more

Trial Design

7Treatment groups
Experimental Treatment
Group I: cALD SR-B (Standard-Risk, Regimen B)Experimental Treatment3 Interventions
See intervention descriptions.
Group II: cALD SR-A (Standard-Risk, Regimen A)Experimental Treatment3 Interventions
See intervention descriptions.
Group III: cALD HR-D (High-Risk, Regimen D)Experimental Treatment3 Interventions
See intervention descriptions.
Group IV: cALD HR-C (High-Risk, Regimen C)Experimental Treatment3 Interventions
See intervention descriptions.
Group V: OP and IMD -Haplo-Identical OnlyExperimental Treatment2 Interventions
Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD) -Haplo-Identical Only See intervention descriptions.
Group VI: OP - Except Haplo-IdenticalExperimental Treatment2 Interventions
Severe Osteoperosis (OP) - Except Haplo-Identical See intervention descriptions.
Group VII: IMD - Except Haplo-identicalExperimental Treatment2 Interventions
Inherited Metabolic Disease (IMD) - Except Haplo-Identical See intervention descriptions.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Stem Cell Transplantation
2003
Completed Phase 3
~610

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
272 Previous Clinical Trials
14,553 Total Patients Enrolled
Paul Orchard, M.D.Principal InvestigatorMasonic Cancer Center, University of Minnesota
1 Previous Clinical Trials
20 Total Patients Enrolled

Media Library

cALD HR-D (High-Risk, Regimen C) (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT02171104 — Phase 2
Refsum Disease Research Study Groups: IMD - Except Haplo-identical, OP - Except Haplo-Identical, OP and IMD -Haplo-Identical Only, cALD SR-A (Standard-Risk, Regimen A), cALD HR-C (High-Risk, Regimen C), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen D)
Refsum Disease Clinical Trial 2023: cALD HR-D (High-Risk, Regimen C) Highlights & Side Effects. Trial Name: NCT02171104 — Phase 2
cALD HR-D (High-Risk, Regimen C) (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02171104 — Phase 2
Refsum Disease Patient Testimony for trial: Trial Name: NCT02171104 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many research participants has this clinical trial enrolled so far?

"Affirmative. Information hosted on clinicaltrials.gov shows that this research project, initially posted in July of 2014, is currently looking for participants. A total of 100 patients are required from a single medical facility."

Answered by AI

Are there any slots available in this research program for participants?

"According to clinicaltrials.gov, this experiment is still welcoming participants. It was first created on July 10th 2014 and edited most recently in November of 2022."

Answered by AI

Has Stem Cell Transplantation been given the go-ahead by federal regulatory bodies?

"The safety of Stem Cell Transplantation was assessed as a 2 on the 1 to 3 scale due to its Phase 2 status, which implies limited data supporting safety but no evidence for efficacy."

Answered by AI

Who else is applying?

What state do they live in?
Alabama
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

Please help my son.
PatientReceived 2+ prior treatments
Recent research and studies
clinicaltrials.govStudy
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
2014. "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02171104.
All > White Matter Disease
~11 spots leftby Jul 2025
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