Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
Primary Purpose
Metachromatic Leukodystrophy
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Enriched Hematopoetic Stem Cell Infusion
Sponsored by
About this trial
This is an expanded access trial for Metachromatic Leukodystrophy focused on measuring metachromatic leukodystrophy
Eligibility Criteria
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
Sites / Locations
- Duke University
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT02084121
First Posted
March 7, 2014
Last Updated
July 15, 2022
Sponsor
Talaris Therapeutics Inc.
Collaborators
Duke University
1. Study Identification
Unique Protocol Identification Number
NCT02084121
Brief Title
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
Official Title
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
Study Type
Expanded Access
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Talaris Therapeutics Inc.
Collaborators
Duke University
4. Oversight
5. Study Description
Brief Summary
A subject was treated under compassionate use provisions under this study with facilitating cell therapy (FCRx) product manufactured using the CliniMACS (Miltenyi Biotec) device, rather than the Max Sep (Baxter) device.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Metachromatic Leukodystrophy
Keywords
metachromatic leukodystrophy
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Biological
Intervention Name(s)
Enriched Hematopoetic Stem Cell Infusion
Intervention Description
Enriched Hematopoetic Stem Cell Infusion
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
3 Years
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Joanne Kurtzberg, MD
Organizational Affiliation
Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
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