Status:
COMPLETED
Vitamin E Supplementation in Hyperinsulinism/Hyperammonemia Syndrome
Lead Sponsor:
Elizabeth A Rosenfeld
Collaborating Sponsors:
University of Pennsylvania
Lawson Wilkins Pediatric Endocrine Society
Conditions:
Hyperinsulinism-Hyperammonemia Syndrome
Eligibility:
All Genders
1-40 years
Phase:
NA
Brief Summary
Investigators will assess the tolerability of oral Vitamin E supplementation in subjects with congenital hyperinsulinism (HI) and hyperammonemia (HA) syndrome.
Detailed Description
Congenital hyperinsulinism (HI) is a rare disorder of pancreatic beta cell insulin secretion that causes persistent and severe hypoglycemia starting at birth. Hyperinsulinism/hyperammonemia (HI/HA) sy...
Eligibility Criteria
Inclusion
- Individuals age ≥12 months and ≤40 years
- Diagnosis of HI/HA syndrome
- On diazoxide therapy for treatment of hypoglycemia
- Females ≥11 years of age or menstruating must have a negative urine/serum pregnancy test and must use an acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study.
- Informed consent for participants ≥18 years. Parental/guardian permission (informed consent) and, if appropriate, child assent for participants \<18 years.
Exclusion
- Individuals age \<12 months or \>40 years
- Individuals who have experienced an allergic reaction to Vitamin E
- Individuals with a known allergy to dairy, whey, or soy
- On concurrent therapy with a medication known to be metabolized by the CYP3A pathway
- Individuals with a known increased risk of bleeding (bleeding disorder or on antiplatelet or anticoagulation therapy)
- Vitamin E supplementation within 30 days prior to enrollment, including multivitamins containing Vitamin E
- Severe hypoglycemia (plasma glucose \<50 mg/dL on repeat checks using home glucose meter) more than once weekly within 30 days prior to enrollment.
- Evidence of a medical condition that might alter results or compromise the interpretation of results, including active infection, kidney failure, severe liver dysfunction, severe respiratory or cardiac failure.
- Evidence of severe hematologic abnormality including severe anemia and/or thrombocytopenia.
- Any investigational drug use within 30 days prior to enrollment.
- Pregnant or lactating females.
- Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
- Unable to provide informed consent (e.g. impaired cognition or judgment).
- Parents/guardians or subjects with limited English proficiency.
Key Trial Info
Start Date :
April 15 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 23 2020
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT03797222
Start Date
April 15 2019
End Date
March 23 2020
Last Update
November 25 2022
Active Locations (1)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104