NCT02318966

Brief Summary

To compare efficacy of Glycosade® with uncooked corn starch (UCCS for the dietary management of hepatic glycogen storage diseases (GSD).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
61

participants targeted

Target at P25-P50 for not_applicable

Timeline
Completed

Started Feb 2016

Longer than P75 for not_applicable

Geographic Reach
4 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 12, 2014

Completed
5 days until next milestone

First Posted

Study publicly available on registry

December 17, 2014

Completed
1.2 years until next milestone

Study Start

First participant enrolled

February 22, 2016

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 14, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 14, 2020

Completed
Last Updated

February 16, 2024

Status Verified

February 1, 2024

Enrollment Period

4.5 years

First QC Date

December 12, 2014

Last Update Submit

February 15, 2024

Conditions

Glycogen Storage Disease

Outcome Measures

Primary Outcomes (1)

  • To assess if Glycosade® will result in improved blood glucose, lactate and ketone levels in a larger group of patients with GSD I, III, VI & IX as measured by duration of normal blood glucose levels or prevention of ketosis

    24 hours

Secondary Outcomes (2)

  • 1. The insulin area under the curve during dietary management with UCCS compared to dietary management with Glycosade®.

    24 hours

  • 2. The area under the curve in lactate, BOHB, during dietary management with UCCS compared to dietary management with Glycosade®.

    24 hours

Study Arms (2)

Glycosade

ACTIVE COMPARATOR

Participants will be randomised to receive the medical food Glycosade as a starch load with a maximum dose of 100g. Glycosade to be taken as one dose.

Dietary Supplement: Medical Food - Glycosade

Uncooked corn starch

PLACEBO COMPARATOR

Participants will be randomised to receive uncooked corn starch as a starch load with a maximum dose of 100g. Uncooked corn starch to be taken as one dose.

Dietary Supplement: Medical Food - Glycosade

Interventions

Medical Food - GlycosadeDIETARY_SUPPLEMENT

double blind randomised crossover of 2 starches - glycosade and UCCS

GlycosadeUncooked corn starch

Eligibility Criteria

Age2 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosed by either genetic mutation analysis or enzymology study (this includes patients with only a single mutation but who also have supportive enzymology consistent with the condition). For those whom mutation analysis is not completed this will be assessed during the study.
  • Aged 2 years or older (5 years or older in the USA)
  • Established on full intake of uncooked corn starch therapy for at least 6 months

You may not qualify if:

  • Women who are pregnant or breastfeeding at the start of the study or planning to become pregnant during the study will be excluded.
  • children less than 2 years of age

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

Hôpital Antoine Béclère

Clamart, 92140 Cedex, France

Location

Universitair Medisch Centrum Groningen

Groningen, 9700RB, Netherlands

Location

National Hospital for Neurology and Neurosurgery

London, Greater London, WC1N 3BG, United Kingdom

Location

Evelina Children's Hospital

London, United Kingdom

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

MeSH Terms

Conditions

Glycogen Storage Disease

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Helen Mundy, MRCP MRCPCH

    Guys and St Thomas NHS Foundation Trust

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 12, 2014

First Posted

December 17, 2014

Study Start

February 22, 2016

Primary Completion

August 14, 2020

Study Completion

August 14, 2020

Last Updated

February 16, 2024

Record last verified: 2024-02

Data Sharing

IPD Sharing
Will not share

Locations

GB(3)US(1)NL(1)FR(1)