Single Patient Expanded Access Protocol: Metabolic Boost

NCT02021266 | Unknown

• 1 other identifier: ICT-14070-120611-Exp Access
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.

Conditions

Metachromatic Leukodystrophy

Interventions

Enriched Hematopoetic Stem Cell Infusion BIOLOGICAL

Enriched Hematopoetic Stem Cell Infusion

Eligibility Criteria

• Sex: female
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject was previously enrolled and qualified for transplant under IND 14070. Subject must be free from infection and have normal liver, kidney, heart and pulmonary function to proceed to a second transplant.
• Patient 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 GFR must be \> 1 cc/kg/min.
• Hepatic transaminases (ALT/AST) 2.5 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 (PFT) demonstrating forced expiratory volume at one second (FEV1) of \>50% of predicted for age. If child is too young or unable to perform PFTs, crying vital capacity result of \>50% of normal value for age or resting pulse oximeter \>92% on room air or clearance by pulmonologist will be required.
• Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
• Patient 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).
• Recipient screening to include glomerular filtration rate (GFR), chest X-ray (CXR), hepatic and renal chemistries, coagulation studies, pulmonary function testing, and ECHO if clinically relevant, chimerism testing, type and screen, and enzyme levels within 30 days of retransplant.

You may not qualify if:

• Uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or need for chronic mechanical ventilation.
• Subject 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.
• Subject with a positive human immunodeficiency virus (HIV) antibody test result
• Subject who are pregnant, as indicated by a positive serum human chorionic gonadotropin (HCG) test
• Subject whose only donor is pregnant at the time of intended transplant
• Subject of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site
• Jehovah's witnesses being unwilling to be transfused
• Patient 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.
• Insufficient funds for bone marrow processing.

Sponsors & Collaborators

• Talaris Therapeutics Inc.: lead
• Duke University: collaborator

Study Sites (1)

Duke University

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Leukodystrophy, Metachromatic

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Sulfatidosis; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Leukoencephalopathies; Demyelinating Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Suzanne T Ildstad, MD

  Talaris Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 19, 2013
• First Posted: December 27, 2013
• Last Updated: October 8, 2020

Record last verified: 2020-10

Locations

US (1)