Glycogen Storage Disease Type INCT03871673
The Use of Uncooked Sweet Manioc Starch to Treat Hepatic Glycogen Storage Diseases
A study testing Sweet manioc starch and Cornstarch in people with Glycogen Storage Disease Type I, run by Hospital de Clinicas de Porto Alegre at 1 site in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Glycogen Storage Disease Type I
- What is being tested?
- Sweet manioc starch and Cornstarch
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteBrazil
- Study dates
- 29 Apr 2017to 31 May 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- patients with a confirmed molecular diagnosis of glycogen storage disease type Ia who are already on uncooked cornstarch therapy
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Novel Approach to Treatment of Hepatic Glycogen Storage Diseases: a Study Based on the Use of Uncooked Sweet Manioc Starch
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.
Read the detailed description
A randomize and crossover study will be conducted on two consecutive days, comparing the use of classic cornstarch to the use of sweet manioc starch in patients with Glycogen Storage Disease type Ia. Ten patients diagnosed with Glycogen Storage Disease type Ia, confirmed by genetic analysis, will be included in the study. Sweet manioc starch and cornstarch, both of Brazilian origin, were analyzed in a previous study of the team in the artificial gastrointestinal model (TIM-1). They will be supplied uncooked, in the same way that cornstarch is currently used in the treatment of hepatic Glycogen Storage Diseases. Doses administered to patients will be 100 g of starch diluted in 200 ml of water at room temperature. To carry out the study, the patients will remain two consecutive nights in the hospital. They will remain in their normal diet until the administration of the starches at 22 hours. To perform the test, at 10 pm blood collection will be performed and after that the patient will ingest the starch (cornstarch or sweet manioc starch, determined by previous randomization). In sequence, blood collection will be performed every hour until the patient has a serum glucose value below 70mg/dL or after the patient stays 10h in a fast. Afterwards, the patient will return to his usual diet until the next test at 22 hours. The same procedures will be performed on the second night, the only change will be the starch used, and if the patient ingested uncooked cornstarch the first night, the same night will ingest the sweet manioc starch, or the opposite.If the patient has hypoglycaemia during the study, it will be discontinued.
- Conditions
- Glycogen Storage Disease Type I
- Being tested
- Sweet manioc starch, Cornstarch
- Keywords
- Hepatic Glycogen Storage Diseases, Treatment, Clinical trial, Cornstarch, Sweet manioc starch
- Medical subject headings
- Glycogen Storage Disease Type I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sweet manioc starchDietary 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.
CornstarchDietary 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.
| Study group | Role | What they receive |
|---|---|---|
| Cornstarch | Active comparator | Ingestion of cornstarch, the standard treatment for hepatic Glycogen Storage Diseases. Receives: Dietary Supplement: Cornstarch |
| Sweet manioc starch | Experimental | Ingestion of sweet manioc starch, the starch in study. Receives: Dietary Supplement: Sweet manioc starch |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: up to 10 hours
Secondary outcomes (3)
Glucose levels
The glucose dosage will be performed by hexokinase enzyme assay. Peripheral blood samples will be collected to determine the baseline value and every hour.
Measured: up to 10 hours
Lactate levels
The lactase dosage will be performed by colorimetric (Lactate → Pyruvate). Peripheral blood samples will be collected to determine the baseline value and every hour.
Measured: up to 10 hours
Insulin levels
The insulin dosage will be performed by microparticle chemiluminescent immunoassay. Peripheral blood samples will be collected to determine the baseline value and every hour.
Measured: up to 10 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2019
- Study start29 Apr 2017
- Primary completion30 Aug 2019
- Study completion31 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Glycogen Storage Disease Type IStudies of the same condition, open and under way.
- More trials run by Hospital de Clinicas de Porto AlegreThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital de Clinicas de Porto Alegre
- Sponsor type
- University, hospital or other
- Collaborators
- Conselho Nacional de Desenvolvimento Científico e Tecnológico, Fundação Médica do Rio Grande do Sul
- Responsible party
- Sponsor
- Investigators
- Ida D Schwartz, Dr, Principal investigator, Hospital de Clinicas de Porto Alegre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03871673
- Sponsor's protocol ID
- 160056
- Status last verified
- Sep 2023
- First submitted
- 9 Mar 2019
- Last updated
- 26 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34082801
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Novel Approach to Treatment of Hepatic Glycogen Storage Diseases: a Study Based on the Use of Uncooked Sweet Manioc Starch
- ClinicalTrials.gov ID
- NCT03871673
- Acronym / sponsor ID
- 160056
- Conditions
- Glycogen Storage Disease Type I
- Keywords
- Hepatic Glycogen Storage Diseases, Treatment, Clinical trial, Cornstarch, Sweet manioc starch
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Hospital de Clinicas de Porto AlegreUniversity, hospital or other
- Collaborators
- Conselho Nacional de Desenvolvimento Científico e Tecnológico, Fundação Médica do Rio Grande do Sul
- Study start
- 29 Apr 2017 (actual)
- Primary completion
- 30 Aug 2019 (actual)
- Study completion
- 31 May 2021 (actual)
- First posted
- 12 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
A randomize and crossover study will be conducted on two consecutive days, comparing the use of classic cornstarch to the use of sweet manioc starch in patients with Glycogen Storage Disease type Ia. Ten patients diagnosed with Glycogen Storage Disease type Ia, confirmed by genetic analysis, will be included in the study. Sweet manioc starch and cornstarch, both of Brazilian origin, were analyzed in a previous study of the team in the artificial gastrointestinal model (TIM-1). They will be supplied uncooked, in the same way that cornstarch is currently used in the treatment of hepatic Glycogen Storage Diseases. Doses administered to patients will be 100 g of starch diluted in 200 ml of water at room temperature. To carry out the study, the patients will remain two consecutive nights in the hospital. They will remain in their normal diet until the administration of the starches at 22 hours. To perform the test, at 10 pm blood collection will be performed and after that the patient will ingest the starch (cornstarch or sweet manioc starch, determined by previous randomization). In sequence, blood collection will be performed every hour until the patient has a serum glucose value below 70mg/dL or after the patient stays 10h in a fast. Afterwards, the patient will return to his usual diet until the next test at 22 hours. The same procedures will be performed on the second night, the only change will be the starch used, and if the patient ingested uncooked cornstarch the first night, the same night will ingest the sweet manioc starch, or the opposite.If the patient has hypoglycaemia during the study, it will be discontinued.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cornstarch | Active comparator | Dietary Supplement: CornstarchIngestion of cornstarch, the standard treatment for hepatic Glycogen Storage Diseases. |
| Sweet manioc starch | Experimental | Dietary Supplement: Sweet manioc starchIngestion of sweet manioc starch, the starch in study. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- patients with a confirmed molecular diagnosis of glycogen storage disease type Ia who are already on uncooked cornstarch therapy
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sweet manioc starch100 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. | Dietary supplement | Sweet manioc starch |
| Cornstarch100 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. | Dietary supplement | Cornstarch |
Primary outcome
Duration of normoglycemia
Time frame: up to 10 hours
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.
3 secondary outcomes
Glucose levels
Time frame: up to 10 hours
Lactate levels
Time frame: up to 10 hours
Insulin levels
Time frame: up to 10 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2023.
- Results publication: PubMed 34082801Monteiro 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.
- A triple-blinded crossover study to evaluate the short-term safety of sweet manioc starch for the treatment of glycogen storage disease type IaLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ida D Schwartz, Dr principal investigator
Hospital de Clinicas de Porto Alegre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Sweet manioc starch and Cornstarch in people with Glycogen Storage Disease Type I, run by Hospital de Clinicas de Porto Alegre at 1 site in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Duration of normoglycemia (time frame: up to 10 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03871673, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Sep 2023.