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Vitamin E for Hyperinsulinism-Hyperammonemia Syndrome

Phase 2
Waitlist Available
Led By Amanda M Ackermann, MD, PhD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 8-11 weeks
Awards & highlights

Study Summary

This trial is testing whether vitamin E can help treat congenital hyperinsulinism (HI), a disorder that causes persistent and severe hypoglycemia starting at birth. HI/HA syndrome is the second most common type of congenital HI and is caused by activating mutations in glutamate dehydrogenase (GDH).

Eligible Conditions
  • Hyperinsulinism-Hyperammonemia Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~8-11 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 8-11 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
protein-induced hyperinsulinemia
Secondary outcome measures
ammonia
hypoglycemic episodes
mean glucose concentration
+5 more
Other outcome measures
brain glutamate
number of participants with EEG abnormalities

Side effects data

From 2021 Phase 4 trial • 31 Patients • NCT03176966
4%
hepatic encephalopathy
4%
bruising
100%
80%
60%
40%
20%
0%
Study treatment Arm
Vitamin E
Ropinirole

Trial Design

1Treatment groups
Experimental Treatment
Group I: Vitamin E Dose-EscalationExperimental Treatment1 Intervention
Subjects will take an oral Vitamin E (dl-alpha-tocopherol) supplement twice daily with a fat-containing meal for 6-9 weeks. The dose will be increased every 2-3 weeks over the course of the study (initial dose 600 IU twice daily, next dose 1,200 IU twice daily, final dose 2,400 IU twice daily). Formulations include softgel capsules in 200, 400, and 1,000 IU doses.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vitamin E
2003
Completed Phase 4
~94880

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor
712 Previous Clinical Trials
8,588,996 Total Patients Enrolled
University of PennsylvaniaOTHER
2,025 Previous Clinical Trials
42,883,022 Total Patients Enrolled
1 Trials studying Hyperinsulinism-Hyperammonemia Syndrome
14 Patients Enrolled for Hyperinsulinism-Hyperammonemia Syndrome
Lawson Wilkins Pediatric Endocrine SocietyOTHER
2 Previous Clinical Trials
124 Total Patients Enrolled
1 Trials studying Hyperinsulinism-Hyperammonemia Syndrome
14 Patients Enrolled for Hyperinsulinism-Hyperammonemia Syndrome
~0 spots leftby Jun 2025