The Use of Uncooked Sweet Manioc Starch to Treat Hepatic Glycogen Storage Diseases

NCT03871673 | Completed

• 1 other identifier: 160056
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

Hepatic Glycogen Storage Diseases are a group of 10 serious genetic diseases that present in childhood and are characterized more frequently by the occurrence of repetitive hypoglycemia and dyslipidemia. Regarding treatment, the most commonly used strategy is the frequent administration of uncooked cornstarch, in average, every 4 hours. Although this treatment is successful, the use of large amounts of cornstarch can lead to overweight and, especially, to the decrease in the quality of life of patients and caregivers, due to the need to use the starch during the night. The search for a treatment that is widely available and that can lead to the prolongation of the fasting time, can collaborate to improve the care of these patients. The main scientific question to be answered by this research is: does sweet manioc starch, a Brazilian product, safely prolong the fasting time (with normoglycemia) of the patients as already suggested in experimental models? Main objective: To evaluate the efficacy and safety of the use of uncooked Sweet Manioc Starch in the treatment of patients with hepatic Glycogen Storage Diseases, using as model the Glycogen Storage Diseases type Ia.

Conditions

Glycogen Storage Disease Type I

Keywords

Hepatic Glycogen Storage Diseases; Treatment; Clinical trial; Cornstarch; Sweet manioc starch

Outcome Measures

Primary Outcomes (1)

• Duration of normoglycemia

  To verify the normoglycemia will be accessed glucose level. The glucose dosage will be performed by hexokinase enzyme assay. Peripheral blood samples will be collected to determine the baseline value and every hour.

  up to 10 hours

Secondary Outcomes (3)

• Glucose levels

  up to 10 hours

• Lactate levels

  up to 10 hours

• Insulin levels

  up to 10 hours

Study Arms (2)

Cornstarch

ACTIVE COMPARATOR

Ingestion of cornstarch, the standard treatment for hepatic Glycogen Storage Diseases.

Dietary Supplement: Cornstarch

Sweet manioc starch

EXPERIMENTAL

Ingestion of sweet manioc starch, the starch in study.

Dietary Supplement: Sweet manioc starch

Interventions

Sweet manioc starch DIETARY_SUPPLEMENT

100 g of starch diluted in 200 ml of water at room temperature In the subsequent night the patient is crossed over to the other starch.

Sweet manioc starch

Cornstarch DIETARY_SUPPLEMENT

100 g of starch diluted in 200 ml of water at room temperature In the subsequent night the patient is crossed over to the other starch.

Cornstarch

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• patients with a confirmed molecular diagnosis of glycogen storage disease type Ia who are already on uncooked cornstarch therapy

Sponsors & Collaborators

• Hospital de Clinicas de Porto Alegre: lead
• Conselho Nacional de Desenvolvimento Científico e Tecnológico: collaborator
• Fundação Médica do Rio Grande do Sul: collaborator

Study Sites (1)

Ida Vanessa D Schwartz

Porto Alegre, Rio Grande do Sul, 90035007, Brazil

Location

Related Publications (1)

• Monteiro VCL, de Oliveira BM, Dos Santos BB, Sperb-Ludwig F, Refosco LF, Nalin T, Derks TGJ, Moura de Souza CF, Schwartz IVD. A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type Ia. Orphanet J Rare Dis. 2021 Jun 3;16(1):254. doi: 10.1186/s13023-021-01877-3.

  PMID: 34082801 RESULT

Related Links

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

MeSH Terms

Conditions

Glycogen Storage Disease Type I

Interventions

Starch

Condition Hierarchy (Ancestors)

Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glucans; Biopolymers; Polymers; Macromolecular Substances; Dietary Carbohydrates; Carbohydrates; Polysaccharides

Study Officials

• Ida D Schwartz, Dr

  Hospital de Clínicas de Porto Alegre

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 9, 2019
• First Posted: March 12, 2019
• Study Start: April 29, 2017
• Primary Completion: August 30, 2019
• Study Completion: May 31, 2021
• Last Updated: September 26, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

BR (1)