A Trial to Evaluate the Frequency of Genetic Sucrase-Isomaltase Deficiency Genotypes, and the Efficacy and Safety of Sucraid® (Sacrosidase) Oral Solution in Subjects With Chronic Diarrhea and Sucrase Deficiency

NCT02784067 | Withdrawn Phase 4

• 1 other identifier: S09A
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

S09A is a Phase 4, multicenter, randomized, double-blind, placebo-controlled, parallel study examining the efficacy and safety of a Sucraid (sacrosidase) Oral Solution in comparison to a placebo in 150-200 subjects with chronic diarrhea possibly attributable to sucrase deficiency.

Conditions

Genetic Sucrase-Isomaltase Deficiency

Outcome Measures

Primary Outcomes (1)

• Response to Sucraid and placebo in a parallel group study based on improvement in daily stool consistency, as assessed by the BSFS using SHMBT

  Response to Sucraid and placebo based on improvement in daily stool consistency, as assessed by the Bristol Stool Form Scale (BSFS) over a 1-week treatment period in subjects with chronic diarrhea and sucrase deficiency using a sucrose hydrogen methane breath test (SHMBT).

  Up to 2 years

Secondary Outcomes (9)

• Effects of Sucraid and placebo on daily assessments of Bristol Stool Form Scale

  Up to 2 years

• Effects of Sucraid and placebo on daily stool frequency

  Up to 2 years

• Effects of Sucraid and placebo on daily abdominal pain

  Up to 2 years

• Effects of Sucraid and placebo on daily bloating severity

  Up to 2 years

• The relationship between the severity of sucrase deficiency, quantified by a SHMBT

  Up to 2 years

Study Arms (2)

Treatment

EXPERIMENTAL

Subjects randomized to the active treatment arm will take Sucraid, 2 mL (17,000 IU) with every meal or snack, administered orally following dilution with 2 to 4 ounces (60 to 120 mL) of water or milk (either cold or at room temperature). The study treatment period is one week.

Drug: Sucraid

Placebo

PLACEBO COMPARATOR

Subjects randomized to the placebo treatment arm will take Sucraid placebo, 2 mL (17,000 IU) with every meal or snack, administered orally following dilution with 2 to 4 ounces (60 to 120 mL) of water or milk (either cold or at room temperature). The study treatment period is one week.

Drug: Placebo

Interventions

Sucraid DRUG

Study drug

Also known as: Sacrosidase

Treatment

Placebo DRUG

Sucraid placebo

Placebo

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:

• Subject is 16 years of age or older.
• Subject is male or female. Women of childbearing potential must be willing to use one of the following contraception methods (for at least 10 days prior to start of study drug and for 10 to 14 days after last dose of study drug): Oral contraceptive, Injectable progestogen, Implants of levonorgestrel, Estrogenic vaginal ring, Percutaneous contraceptive patches, Intrauterine device, Sterile male partner, Double-barrier method of contraception Women of non-child bearing potential include females regardless of age with functioning ovaries and who have a current tubal ligation (Hatcher, 2004), bilateral oophorectomy, or total hysterectomy, or post-menopausal females. Note: Post-menopausal is defined as 1 year without menses with an appropriate clinical profile (e.g., age appropriate, \>45 years, in the absence of hormone replacement therapy).
• Subject has a minimum of 3 months of self-reported diarrhea (BSFS scores ≥ 5 on at least 3 days per week and ≥1 stool per day)
• Subject has a value in the SHMBT of at least 20 ppm for hydrogen, or 12 ppm for methane or 15 ppm above a previous breath sample for the combination of both gases.
• Subject reports that he/she experienced soft stools or diarrhea within the last 24 hours when contacted by the site 24 hours after completing the SHMBT.
• Subject is able to read, speak, and verbally understand the English language.
• Subject is located in the United States.
• Subject has access to the Internet on a daily basis.
• Subject has access to an acceptable Apple iPhone/iPad/iTouch or Android smartphone/tablet. The sponsor may choose to provide a smartphone in unusual cases (please contact sponsor to request loaner device when applicable)

You may not qualify if:

• Subject has recent history of functional or chronic constipation.
• Subject has known history of ulcerative colitis, Crohn's disease, or Celiac disease.
• Subject has known hypersensitivity to papain, glycerol, or yeast.
• Subject has received bovine serum in the last year.
• Subject has previous history of Sucraid use.
• Subject has taken any prebiotic or probiotic within 5 days prior to Visit 2 and does not agree to refrain from taking them during the study.
• Subject is female and is pregnant, breastfeeding, or planning to become pregnant during the study.
• Subject has known uncontrolled systemic disease.
• Subject has prior diagnosis of Type 1 or Type 2 diabetes.
• Subject has history of bowel resection.
• Subject is undergoing chemotherapy for the treatment of cancer.
• Subject has major physical or psychiatric illness within the last 6 months that in the opinion of the investigator would affect the subject's ability to complete the trial.
• Subject has used an investigational device or investigational drug within 30 days prior to Visit 1.

Sponsors & Collaborators

• QOL Medical, LLC: lead

Related Publications (26)

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  BACKGROUND

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  BACKGROUND

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• Quezada-Calvillo R, Sim L, Ao Z, Hamaker BR, Quaroni A, Brayer GD, Sterchi EE, Robayo-Torres CC, Rose DR, Nichols BL. Luminal starch substrate "brake" on maltase-glucoamylase activity is located within the glucoamylase subunit. J Nutr. 2008 Apr;138(4):685-92. doi: 10.1093/jn/138.4.685.

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• Treem WR. Clinical aspects and treatment of congenital sucrase-isomaltase deficiency. J Pediatr Gastroenterol Nutr. 2012 Nov;55 Suppl 2:S7-13. doi: 10.1097/01.mpg.0000421401.57633.90. No abstract available.

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• Treem WR, McAdams L, Stanford L, Kastoff G, Justinich C, Hyams J. Sacrosidase therapy for congenital sucrase-isomaltase deficiency. J Pediatr Gastroenterol Nutr. 1999 Feb;28(2):137-42. doi: 10.1097/00005176-199902000-00008.

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• Treem WR, Ahsan N, Sullivan B, Rossi T, Holmes R, Fitzgerald J, Proujansky R, Hyams J. Evaluation of liquid yeast-derived sucrase enzyme replacement in patients with sucrase-isomaltase deficiency. Gastroenterology. 1993 Oct;105(4):1061-8. doi: 10.1016/0016-5085(93)90950-h.

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• Wiecek S, Wos H, Radziewicz Winnicki I, Komraus M, Grzybowska Chlebowczyk U. Disaccharidase activity in children with inflammatory bowel disease. Turk J Gastroenterol. 2014 Apr;25(2):185-91. doi: 10.5152/tjg.2014.3994.

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MeSH Terms

Interventions

beta-Fructofuranosidase

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2016
• First Posted: May 26, 2016
• Study Start: May 1, 2016
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: September 25, 2017

Record last verified: 2017-09