- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02084121
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Expanded Access Type
- Individual Patients
Contacts and Locations
Study Locations
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North Carolina
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Durham, North Carolina, United States, 27705
- Duke University
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
Patients must have a confirmed diagnosis of inherited metabolic disorder / inborn error of metabolism. Diagnosis should be confirmed by appropriate test(s) (enzyme and/or mutation analysis) before study entry. Patients must not be eligible for myeloablative chemotherapy as a preparative regimen for transplant due to age, co-morbidities or organ dysfunction.
Inborn errors of metabolism / Inherited Metabolic Disorders (IMD) eligible for this Compassionate Use Provision include Metachromatic Leukodystrophy (MLD)
- Patients must be ≥ 3 years of age
- Patients must have Lansky or Karnofsky performance status ≥40
Patients must have adequate function of other organ systems as measured by:
- Creatinine < 2.0 mg/dl and creatinine clearance ≥60 cc/min/1.73m2. Newborns must have a creatinine clearance > 25 cc/min. For babies < 3 months of age, the raw value on glomerular filtration rate (GFR) must be > 1 cc/kg/min.
- Hepatic transaminases (ALT/AST) ≤4 x normal, bilirubin <2.0mg/dl
- Normal cardiac function by echocardiogram or radionuclide scan (ejection fraction or shortening fraction >80% of normal value for age)
- Pulmonary function tests demonstrating forced expiratory volume at one second (FEV1) of >50% of predicted for age. If child is too young for pulmonary function tests (PFTs), crying vital capacity result of >50% of normal value for age or resting pulse oximeter >85% on room air or clearance by pulmonologist will be required.
- Patient must have a related donor [identical or mismatched for 1, 2 or 3 histocompatibility leukocyte antigen (HLA)-A, -B or -DR loci].
- Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
- Patients must have a minimum life expectancy of at least 6 months.
- Female patients of childbearing potential cannot be pregnant or lactating/breast-feeding and must be either surgically sterile, postmenopausal (no menses for the previous 12 months), or must be practicing an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy).
Exclusion Criteria:
- Patients with uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or need for chronic mechanical ventilation.
- Patients with allogeneic stem cell transplant with cytoreductive therapy in the past 6 months.
- Subjects must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by radiation therapist)
- Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, could not tolerate reduced intensity transplantation.
- Severe impairment of functional performance as evidenced by a Karnofsky (patients >16 years old) or Lansky (children <16 years old) score <40%
- Subjects with a positive human immunodeficiency virus (HIV) antibody test result
- Subjects who are pregnant, as indicated by a positive serum human chorionic gonadotropin (HCG) test
- Subjects whose only donor is pregnant at the time of intended transplant
- Subjects of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site
- Jehovah's witness
- Patients that have any comorbid condition which, in the view of the Principal Investigators, renders the patient at too high a risk from treatment complications and regimen related morbidity/mortality.
- Lack of related donors
Study Plan
How is the study designed?
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Joanne Kurtzberg, MD, Duke University
Study record dates
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Immune System Diseases
- Demyelinating Autoimmune Diseases, CNS
- Autoimmune Diseases of the Nervous System
- Demyelinating Diseases
- Autoimmune Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Leukoencephalopathies
- Hereditary Central Nervous System Demyelinating Diseases
- Sulfatidosis
- Multiple Sclerosis
- Leukodystrophy, Metachromatic
Other Study ID Numbers
- ICT-13080-Compassionate Use
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
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