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Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02084121
Expanded Access Status : No longer available
First Posted : March 11, 2014
Last Update Posted : October 8, 2020
Duke University
Information provided by (Responsible Party):
Talaris Therapeutics Inc.

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.

Condition or disease Intervention/treatment
Metachromatic Leukodystrophy Biological: Enriched Hematopoetic Stem Cell Infusion

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)

Intervention Details:
  • Biological: Enriched Hematopoetic Stem Cell Infusion
    Enriched Hematopoetic Stem Cell Infusion

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male

Inclusion Criteria:

  1. 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)

  2. Patients must be ≥ 3 years of age
  3. Patients must have Lansky or Karnofsky performance status ≥40
  4. 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.
  5. Patient must have a related donor [identical or mismatched for 1, 2 or 3 histocompatibility leukocyte antigen (HLA)-A, -B or -DR loci].
  6. Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
  7. Patients must have a minimum life expectancy of at least 6 months.
  8. 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02084121

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United States, North Carolina
Duke University
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Talaris Therapeutics Inc.
Duke University
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Principal Investigator: Joanne Kurtzberg, MD Duke University
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Responsible Party: Talaris Therapeutics Inc. Identifier: NCT02084121    
Other Study ID Numbers: ICT-13080-Compassionate Use
First Posted: March 11, 2014    Key Record Dates
Last Update Posted: October 8, 2020
Last Verified: October 2020
Keywords provided by Talaris Therapeutics Inc.:
metachromatic leukodystrophy
Additional relevant MeSH terms:
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Multiple Sclerosis
Leukodystrophy, Metachromatic
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Lysosomal Storage Diseases, Nervous System
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders