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A comparison of Glycosade® and Uncooked Cornstarch (UCCS) for the dietary management of hepatic glycogen storage diseases (GSD)

Completed
Conditions
glycogenosis
metabolic disease
10027424
10021605
Registration Number
NL-OMON45944
Lead Sponsor
Vitaflo International Ltd
Brief Summary

Trial is onging in other countries

Detailed Description

Not available

Recruitment & Eligibility

Status
Completed
Sex
Not specified
Target Recruitment
12
Inclusion Criteria

See the study protocol, §6.1:;Patients with GSD I, III, VI or IX under the care of a metabolic centre meeting the following 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.
* Adults and children aged 2 years or older.
* Established on UCCS for at least 6 months to full dietary requirements as defined by local clinical practice.
* Freely given informed written consent from participant or parent/caregiver.

Exclusion Criteria

See the study protocol, §6.2;* Evidence of any other medical condition which in the opinion of the Investigator makes it undesirable for the subject to participate in the trial or which would jeopardise compliance with the protocol.;* Women pregnant / breastfeeding at the start of the study or planning to become pregnant during its duration. ;Note: Women who become pregnant unexpectedly during this study can continue on the study product if they wish.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<p>The primary outcome for this study will be an increase in the duration of<br /><br>normal blood sugar levels and / or delaying the development of significant<br /><br>ketosis during dietary management with Glycosade® compared to dietary<br /><br>management with UCCS (Part 1) as defined by:<br /><br><br /><br>a. Period of normal blood glucose levels (plasma glucose * 3.6 mmol/l)<br /><br>increased by 60 minutes,<br /><br><br /><br>OR<br /><br><br /><br>b. A delay of 30 minutes in the development of significant ketosis in patients<br /><br>with GSD III, VI, IX as defined by:<br /><br>* BOHB * 1.0 mmol/L (participants < 14 years)<br /><br>* BOHB * 0.4 mmol/L (participants * 14 years)</p><br>
Secondary Outcome Measures
NameTimeMethod
<p>The secondary outcomes for this study will be:<br /><br><br /><br>1. The insulin area under the curve during dietary management with UCCS<br /><br>compared to dietary management with Glycosade®.<br /><br>2. The area under the curve in lactate, BOHB, during dietary management with<br /><br>UCCS compared to dietary management with Glycosade®.<br /><br>3. Tolerability & palatability of both Glycosade® and UCCS. </p><br>
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