N/A
N=14
Vitamin E Supplementation in Hyperinsulinism/Hyperammonemia Syndrome
Hyperinsulinism-Hyperammonemia Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT03797222 ↗Enrolled (actual)
14
Serious AEs
0.0%
Results posted
Nov 2022
Primary outcome: Primary: Tolerability of Vitamin E Based on Responses to a Subject/Parent-reported Symptom Questionnaire After Vitamin E Supplementation Compared to Baseline — 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Vitamin E (Dietary_supplement)
- Age
- Pediatric, Adult · 1+ yrs
- Sex
- All
- Sponsor
- Elizabeth A Rosenfeld
- Primary completion
- Mar 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Tolerability of Vitamin E Based on Responses to a Subject/Parent-reported Symptom Questionnaire After Vitamin E Supplementation Compared to Baseline |
2 | — |
| SECONDARY Plasma Alpha-tocopherol Concentration |
18.6 | — |
| SECONDARY Delta-plasma Glucose Concentration |
1.1 | — |
| SECONDARY Fasting Plasma Glucose Concentration |
-0.3 | — |
| SECONDARY Nadir Plasma Glucose Concentration |
0.01 | — |
| SECONDARY Fasting Plasma Insulin Concentration |
-2.4 | — |
| SECONDARY Peak Plasma Insulin Concentration |
-6.3 | — |
| SECONDARY Delta-plasma Insulin Concentration |
-5.2 | — |
| SECONDARY Fasting Plasma Ammonia Concentration |
1.0 | — |
| SECONDARY Delta-plasma Ammonia Concentration |
-3.4 | — |
| SECONDARY Hypoglycemia Frequency |
0.6 | — |
Summary
Investigators will assess the tolerability of oral Vitamin E supplementation in subjects with congenital hyperinsulinism (HI) and hyperammonemia (HA) syndrome.
Eligibility Criteria
Inclusion Criteria
- 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 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.
Data sourced from ClinicalTrials.gov (NCT03797222). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.