What is the mechanism of action of this treatment?

Sucraid® (sacrosidase) is an enzyme replacement therapy used to treat Sucrose-Isomaltase Deficiency (CSID). It works by providing the sucrase enzyme that patients with CSID lack, allowing them to properly digest sucrose into glucose and fructose, which can then be absorbed by the body. This treatment is crucial for CSID patients as it helps alleviate gastrointestinal symptoms such as bloating, gas, and diarrhea that result from undigested sucrose fermenting in the gut. By improving the digestion of sucrose, Sucraid® enhances nutrient absorption and overall quality of life for individuals with CSID.

What side effects are associated with this type of intervention?

The most common side effects of Sucraid® (sacrosidase) for treating Sucrose-Isomaltase Deficiency include abdominal pain, nausea, vomiting, and diarrhea. Some patients may also experience hypersensitivity reactions such as wheezing, difficulty breathing, or skin rashes. It is important to monitor for allergic reactions, especially in patients with a history of asthma or allergies.

What prior FDA approvals exist?

Sucraid® (sacrosidase) is an FDA-approved enzyme replacement therapy specifically designed to treat congenital sucrase-isomaltase deficiency (CSID). It provides the sucrase enzyme that individuals with CSID lack, helping them digest sucrose. There are no other enzyme replacement therapies currently approved by the FDA for this condition, making Sucraid® the primary treatment option available for patients with sucrase deficiency.

What other types of research exist?

As of 2024, promising novel alternatives to Sucraid® for treating Sucrose-Isomaltase Deficiency include: 1) Gene therapy approaches targeting the underlying genetic mutations responsible for CSID, which are currently under investigation and show potential for long-term correction of enzyme deficiencies. 2) New enzyme replacement therapies that are being developed to improve the stability and efficacy of sucrase-isomaltase enzymes. 3) Probiotic treatments designed to enhance gut microbiota and aid in the digestion of disaccharides. Patients should discuss these options with their healthcare provider to determine the most appropriate treatment based on their specific condition and the latest available research.

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Enzyme Replacement Therapy

Sucraid for Congenital Sucrase-Isomaltase Deficiency

Phase 4

Recruiting

Led By Weng Tao, M.D., Ph. D

Research Sponsored by QOL Medical, LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject is male or female, ages 6 months to 17 years old

Be younger than 18 years old

Must not have

Subjects with a history of diabetes mellitus

Subjects that do not have the mental capacity to understand the study requirements and are unable to comply

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Drug Has Already Been Approved

Summary

This trial tests Sucraid®, a medication that helps break down sugars, in children with digestive issues due to low sucrase levels. It aims to see if Sucraid® can reduce symptoms like diarrhea and stomach pain by providing the enzyme needed to digest sugar.

Who is the study for?

This trial is for U.S. children aged 6 months to 17 years with low, moderate, or normal sucrase levels as shown by a test within the last year. They must not have used Sucraid before and should be free from major illnesses or conditions like diabetes that could affect their participation.

What is being tested?

The study tests how effective a 7-day treatment of Sucraid (sacrosidase) Oral Solution is in kids with different levels of sucrase enzyme activity. It also looks at the link between genetic mutations related to CSID and sucrase activity levels.

What are the potential side effects?

Potential side effects are not detailed here but may include reactions due to allergies to sucrose, yeast, papain, or glycerol which are ingredients in the medication.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 6 months and 17 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of diabetes.

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I understand and can follow the study's requirements.

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I have a disease that is not currently under control.

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I have not had a fever or flu-like symptoms in the last 5 days.

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My abdominal issues are not due to conditions like IBD, celiac, or pancreatitis.

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I have previously taken Sucraid®.

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD).

Secondary study objectives

Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Single Arm Open Labeled Commercial SucraidExperimental Treatment1 Intervention

All subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid.

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Sucraid® (sacrosidase) is an enzyme replacement therapy used to treat Sucrose-Isomaltase Deficiency (CSID). It works by providing the sucrase enzyme that patients with CSID lack, allowing them to properly digest sucrose into glucose and fructose, which can then be absorbed by the body. This treatment is crucial for CSID patients as it helps alleviate gastrointestinal symptoms such as bloating, gas, and diarrhea that result from undigested sucrose fermenting in the gut. By improving the digestion of sucrose, Sucraid® enhances nutrient absorption and overall quality of life for individuals with CSID.

Galactosemia: Towards Pharmacological Chaperones.Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.