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 (GSID-E)

QOL Medical

Status and phase: Withdrawn; Phase 4

Conditions: Genetic Sucrase-Isomaltase Deficiency

Treatments: Drug: Sucraid; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02784067

S09A

Details and patient eligibility

About

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.

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Subject is 16 years of age or older.
2. 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).
3. 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)
4. 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.
5. 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.
6. Subject is able to read, speak, and verbally understand the English language.
7. Subject is located in the United States.
8. Subject has access to the Internet on a daily basis.
9. 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)

Exclusion criteria

1. Subject has recent history of functional or chronic constipation.
2. Subject has known history of ulcerative colitis, Crohn's disease, or Celiac disease.
3. Subject has known hypersensitivity to papain, glycerol, or yeast.
4. Subject has received bovine serum in the last year.
5. Subject has previous history of Sucraid use.
6. 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.
7. Subject is female and is pregnant, breastfeeding, or planning to become pregnant during the study.
8. Subject has known uncontrolled systemic disease.
9. Subject has prior diagnosis of Type 1 or Type 2 diabetes.
10. Subject has history of bowel resection.
11. Subject is undergoing chemotherapy for the treatment of cancer.
12. 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.
13. Subject has used an investigational device or investigational drug within 30 days prior to Visit 1.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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0 participants in 2 patient groups, including a placebo group

Treatment

Experimental group

Description:

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.

Treatment:

Drug: Sucraid

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo