Clinical trial NCT02318966: Glycosade v UCCS in the Dietary Management of Hepatic GSD
Official title: A Comparison of Glycosade® and Uncooked Cornstarch (UCCS) for the Dietary Management of Hepatic Glycogen Storage Diseases (GSD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Glycogen Storage Disease
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
Collaborators: name: University College London Hospitals
name: University of Florida
name: Great Ormond Street Hospital for Children NHS Foundation Trust
name: University Medical Center Groningen
name: Hôpital Necker-Enfants Malades
First posted: 2014-12-17
Enrollment: 61
Sex: ALL
Minimum age (years): 2
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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

Detailed description: 'Glyde' is a prospective, randomised, double-blind, crossover trial to compare the effects of two different starches used in the dietary management of GSD.

The study will recruit 64 patients from six centres in the UK, USA, France and The Netherlands. The planned duration per participant is 2 years.

Participants will be randomised into two equally sized groups:

Arm A- Glycosade intake given first, followed by UCCS 1-6 weeks later Arm B- UCCS intake given first, followed by Glycosade 1-6 weeks later.

The objective of this trial is to establish whether Glycosade® improves outcomes for patients with GSD compared to UCCS therapy by comparing the duration of normal blood sugars, lactate and ketone levels of patients with hepatic GSD (Types I, III, VI and IX) post equivalent intakes of UCCS and Glycosade®.

The first part consists of two (blinded) 12 hour starch load tests administered randomly within a maximum 6 week period. Participants will be randomised to receive either UCCS or Glycosade® for the initial intervention period crossing over to the other product for the second intervention period (part 1).

The second part (unblinded) is a 24 month open follow up whereby participant's will continue on the product considered most suitable by the clinician and patient.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: 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
timeFrame: 24 hours
Secondary outcomes:
measure: 1. The insulin area under the curve during dietary management with UCCS compared to dietary management with Glycosade®.
timeFrame: 24 hours
measure: 2. The area under the curve in lactate, BOHB, during dietary management with UCCS compared to dietary management with Glycosade®.
timeFrame: 24 hours
Other outcomes:
Locations:
city: Hartford
country: United States
facility: Connecticut Children's Medical Center
state: Connecticut
city: Clamart
country: France
facility: Hôpital Antoine Béclère
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen
city: London
country: United Kingdom
facility: National Hospital for Neurology and Neurosurgery
state: Greater London
city: London
country: United Kingdom
facility: Evelina Children's Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
Study officials:
affiliation: Guys and St Thomas NHS Foundation Trust
name: Helen Mundy, MRCP MRCPCH
role: PRINCIPAL_INVESTIGATOR
References: