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Clinical Trial
. 2021 Jun 3;16(1):254.
doi: 10.1186/s13023-021-01877-3.

A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type Ia

Vaneisse C L Monteiro  1 , Bibiana M de Oliveira  1 , Bruna B Dos Santos  1 , Fernanda Sperb-Ludwig  1   2 , Lilia F Refosco  3 , Tatiele Nalin  4 , Terry G J Derks  5 , Carolina F Moura de Souza  3 , Ida V D Schwartz  6   7   8   9   10
Affiliations
Clinical Trial

A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type Ia

Vaneisse C L Monteiro et al. Orphanet J Rare Dis. .

Abstract

Background: Glycogen storage disease type 1a (GSD Ia) is characterized by severe fasting hypoglycemia. The clinical management includes the administration of uncooked cornstarch (UCCS). Although such a diet approach is effective in achieving euglycemia, its impact on the quality of life of patients should be considered. In vitro analyses suggest a longer release of glucose when using sweet manioc starch (SMS).

Methods: We compared the efficacy and safety of the administration of SMS and UCCS during a short-fasting challenge in patients with GSD Ia in a randomized, triple-blind, phase I/II, cross-over study. GSD Ia patients aged ≥ 16 years and treated with UCCS were enrolled. Participants were hospitalized for two consecutive nights, receiving UCCS or SMS in each night. After the administration of the starches, glucose, lactate and insulin levels were measured in 1-h interval throughout the hospitalization period. The procedures were interrupted after 10 h of fasting or in a hypoglycemic episode (< 3.88 mmol/L).

Results: Eleven individuals (mean age: 21.6 ± 4.3 years; all presenting body mass index > 25 kg/m2) participated in the study. The average fasting period was 8.2 ± 2.0 h for SMS and 7.7 ± 2.3 h for UCCS (p = 0.04). SMS maintained euglycemia for a greater period over UCCS. Increased lactate concentrations were detected even in absence of hypoglycemia, not being influenced by the different starches investigated (p = 0.17). No significant difference was found in total cholesterol, HDL, triglycerides and uric acid levels in both arms. None of the patients showed severe adverse events.

Conclusions: SMS appears to be non-inferior to UCCS in the maintenance of euglycemia, thus emerging as a promising alternative to the treatment of GSD Ia.

Keywords: Cornstarch; Dietary treatment; Hepatic glycogen storage disease; Inborn errors of metabolism; Sweet manioc starch; Treatment strategies.

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Conflict of interest statement

TN is currently an employee of Ultragenyx Farmacêutica Brasil LTDA and hold stock in Ultragenyx Pharmaceutical Inc. All other authors declare no conflicts of interest.

Figures

Fig. 1
Fig. 1
Kaplan Meier curve indicating test durations for each starch load performed (n = 11). UCCS: uncooked cornstarch and SMS: sweet manioc starch
Fig. 2
Fig. 2
Blood concentrations of glucose and lactic acid after UCCS or SMS throughout the study period. A Glucose level for each starch load performed (n = 11), normal range: > 3.88 mmol/L; B Lactic acid levels for each starch load performed (n = 11), normal range: 0.5 to 2.2 mmol/L. UCCS (uncooked cornstarch load) or SMS (sweet manioc starch)
Fig. 3
Fig. 3
Study design. *refers to blood collection for the evaluation of glucose, lactic acid, insulin, total cholesterol, triglyceride and uric acid levels. **refers to blood collection for the glucose, lactic acid and insulin levels. GSD Ia: glycogen storage disorder type Ia; UCCS: uncooked cornstarch and SMS: sweet manioc starch
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