Single Patient Expanded Access Protocol: Metabolic Boost

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02021266
• Date of registration: 19/12/2013
• Prospective Registration: No
• Primary sponsor: Talaris Therapeutics Inc.
• Public title: Single Patient Expanded Access Protocol: Metabolic Boost Boost
• Scientific title: Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
• Date of first enrolment: April 2013
• Recruitment status: No longer available
• URL: https://clinicaltrials.gov/show/NCT02021266
• Study type: Expanded Access

Countries of recruitment

United States

Contacts

• Name: Suzanne T Ildstad, MD
• Affiliation: Talaris Therapeutics Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

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.

1. 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.

2. Patient, and parent, or legal guardian must have given written informed consent
according to FDA guidelines.

3. Patient must have a minimum life expectancy of at least 6 months.

4. 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).

5. 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.

Exclusion Criteria:

1. Uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or
need for chronic mechanical ventilation.

2. Subject must not have had previous radiation therapy that would preclude total body
irradiation (TBI) (as determined by radiation therapist)

3. Uncontrolled infection or severe concomitant diseases, which in the judgment of the
Principal Investigator, could not tolerate reduced intensity transplantation.

4. Subject with a positive human immunodeficiency virus (HIV) antibody test result

5. Subject who are pregnant, as indicated by a positive serum human chorionic
gonadotropin (HCG) test

6. Subject whose only donor is pregnant at the time of intended transplant

7. Subject of childbearing potential who are not practicing adequate contraception as
defined by the investigator at the site

8. Jehovah's witnesses being unwilling to be transfused

9. 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.

10. Insufficient funds for bone marrow processing.



Age minimum: N/A
Age maximum: N/A
Gender: Female

Health Condition(s) or Problem(s) studied

Metachromatic Leukodystrophy

Intervention(s)

Biological: Enriched Hematopoetic Stem Cell Infusion

Secondary ID(s)

ICT-14070-120611-Exp Access

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Duke University