Genetic Sucrase-Isomaltase DeficiencyPhase 4GSID-ENCT02784067
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
A Phase 4 study testing Sucraid in people with Genetic Sucrase-Isomaltase Deficiency, run by QOL Medical, LLC.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Genetic Sucrase-Isomaltase Deficiency
- What is being tested?
- Sucraid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- May 2016to Dec 2017
- Phase
- Phase 4
- 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
- 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.
Show all 9Show fewer
- 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)
Who cannot join
- 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.
Show all 13Show fewer
- 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.
About this study
Official title: A Multicenter, Double-Blind, Placebo-Controlled 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
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
- Being tested
- Sucraid, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SucraidDrug
Also called Sacrosidase
Study drug
PlaceboDrug
Sucraid placebo
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Up to 2 years
Secondary outcomes (9)
Effects of Sucraid and placebo on daily assessments of Bristol Stool Form Scale
Measured: Up to 2 years
Effects of Sucraid and placebo on daily stool frequency
Measured: Up to 2 years
Effects of Sucraid and placebo on daily abdominal pain
Measured: Up to 2 years
Effects of Sucraid and placebo on daily bloating severity
Measured: Up to 2 years
The relationship between the severity of sucrase deficiency, quantified by a SHMBT
Measured: Up to 2 years
The mean improvement in the BSFS for each treatment group.
Measured: Up to 2 years
Overall frequency of the 4 most common sucrase-isomaltase deficiency genetic variants
Overall frequency of the 4 most common sucrase-isomaltase deficiency genetic variants in comparison to the frequency in public proxy databases of broad populations.
Measured: Up to 2 years
The number of less common sucrase-isomaltase polymorphisms in this study population.
Measured: Up to 2 years
The allele frequency of the most common sucrase-isomaltase genetic variants in subjects with chronic diarrhea attributable to sucrase deficiency compared to the allele frequency in other databases
Assess the allele frequency of the 38 most common sucrase-isomaltase genetic variants in subjects with chronic diarrhea attributable to sucrase deficiency compared to the allele frequency of sucrase-isomaltase genetic variants in the Exome Variant Server, the ExAC server, and other public proxy and private genetic databases.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2016 (estimated)
- Study startMay 2016
- Primary completionDec 2017 (estimated)
- Study completionDec 2017 (estimated)
Why it stopped: Clinical Trial Material
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (26)Papers the sponsor has linked to this study.
- Other trials for Genetic Sucrase-Isomaltase DeficiencyStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- QOL Medical, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02784067
- Sponsor's protocol ID
- S09A
- Acronym
- GSID-E
- Status last verified
- Sep 2017
- First submitted
- 18 May 2016
- Last updated
- 25 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Arthur AB. Intestinal disaccharidase deficiency in children with coeliac disease. Arch Dis Child. 1966 Oct;41(219):519-24. doi: 10.1136/adc.41.219.519. No abstract available.
PubMed 5957725Bayless TM, Christopher NL. Disaccharidase deficiency. Am J Clin Nutr. 1969 Feb;22(2):181-90. doi: 10.1093/ajcn/22.2.181.
PubMed 4885929Chumpitazi BP, Robayo-Torres CC, Tsai CM, Opekun AR, Baker SS, Nichols BL, Gilger MA, Su1110 Yield of prospective disaccharidase testing in children with recurrent abdominal pain. Gastroenterology. May 2013;144(5):S-401-S402.
Cross HS, Quaroni A. Inhibition of sucrose-isomaltase expression by EGF in the human colon adenocarcinoma cells Caco-2. Am J Physiol. 1991 Dec;261(6 Pt 1):C1173-83. doi: 10.1152/ajpcell.1991.261.6.C1173.
PubMed 1767818Czernichow B, Simon-Assmann P, Kedinger M, Arnold C, Parache M, Marescaux J, Zweibaum A, Haffen K. Sucrase-isomaltase expression and enterocytic ultrastructure of human colorectal tumors. Int J Cancer. 1989 Aug 15;44(2):238-44. doi: 10.1002/ijc.2910440209.
PubMed 2759730DeMets DL, Lan KK. Interim analysis: the alpha spending function approach. Stat Med. 1994 Jul 15-30;13(13-14):1341-52; discussion 1353-6. doi: 10.1002/sim.4780131308.
PubMed 7973215El-Chammas K, Williams S, Miranda A. Su1254 Lactase and sucrase deficiencies in pediatric subjects with chronic abdominal pain. Gastroenterology. May 2014;146(5):S-415-S416.
Gupta SK, Chong SK, Fitzgerald JF. Disaccharidase activities in children: normal values and comparison based on symptoms and histologic changes. J Pediatr Gastroenterol Nutr. 1999 Mar;28(3):246-51. doi: 10.1097/00005176-199903000-00007.
PubMed 10067723Hatcher RA, Trussell J, Stewart F, Nelson AL, Cates W, Guest F, Kowal DD, editors. Contraceptive Technology. New York: Ardent Media, 2004:226.
Heitlinger LA, Rossi TM, Lee PC, Lebenthal E. Human intestinal disaccharidase activities: correlations with age, biopsy technique, and degree of villus atrophy. J Pediatr Gastroenterol Nutr. 1991 Feb;12(2):204-8.
PubMed 1904933Hyams JS, Batrus CL, Grand RJ, Sallan SE. Cancer chemotherapy-induced lactose malabsorption in children. Cancer. 1982 Feb 15;49(4):646-50. doi: 10.1002/1097-0142(19820215)49:43.0.co;2-m.
PubMed 7055778Kerry KR, & Townley, R. R. Genetic aspects of intestinal sucrase-isomaltase deficiency. Australian Paediatric Journal. 1965;1:223.
Lehmacher W, Wassmer G. Adaptive sample size calculations in group sequential trials. Biometrics. 1999 Dec;55(4):1286-90. doi: 10.1111/j.0006-341x.1999.01286.x.
PubMed 11315085Mones RL, Yankah A, Duelfer D, Bustami R, Mercer G. Disaccharidase deficiency in pediatric patients with celiac disease and intact villi. Scand J Gastroenterol. 2011 Dec;46(12):1429-34. doi: 10.3109/00365521.2011.619276. Epub 2011 Sep 22.
PubMed 21936724Murray IA, Smith JA, Coupland K, Ansell ID, Long RG. Intestinal disaccharidase deficiency without villous atrophy may represent early celiac disease. Scand J Gastroenterol. 2001 Feb;36(2):163-8. doi: 10.1080/003655201750065915.
PubMed 11252408
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Double-Blind, Placebo-Controlled 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
- ClinicalTrials.gov ID
- NCT02784067
- Acronym / sponsor ID
- GSID-E / S09A
- Conditions
- Genetic Sucrase-Isomaltase Deficiency
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Clinical Trial Material
- Enrolment
- 0 participants (actual)
- Lead sponsor
- QOL Medical, LLCIndustry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- Dec 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 26 May 2016 (estimated)
- 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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: SucraidSubjects 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. |
| Placebo | Placebo comparator | Drug: PlaceboSubjects 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. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
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 |
|---|---|---|
| SucraidAlso: SacrosidaseStudy drug | Drug | Treatment |
| PlaceboSucraid placebo | Drug | Placebo |
Primary outcome
Response to Sucraid and placebo in a parallel group study based on improvement in daily stool consistency, as assessed by the BSFS using SHMBT
Time frame: Up to 2 years
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).
9 secondary outcomes
Effects of Sucraid and placebo on daily assessments of Bristol Stool Form Scale
Time frame: Up to 2 years
Effects of Sucraid and placebo on daily stool frequency
Time frame: Up to 2 years
Effects of Sucraid and placebo on daily abdominal pain
Time frame: Up to 2 years
Effects of Sucraid and placebo on daily bloating severity
Time frame: Up to 2 years
The relationship between the severity of sucrase deficiency, quantified by a SHMBT
Time frame: Up to 2 years
The mean improvement in the BSFS for each treatment group.
Time frame: Up to 2 years
Overall frequency of the 4 most common sucrase-isomaltase deficiency genetic variants
Time frame: Up to 2 years
The number of less common sucrase-isomaltase polymorphisms in this study population.
Time frame: Up to 2 years
The allele frequency of the most common sucrase-isomaltase genetic variants in subjects with chronic diarrhea attributable to sucrase deficiency compared to the allele frequency in other databases
Time frame: Up to 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2017.
- Background: PubMed 5957725Arthur AB. Intestinal disaccharidase deficiency in children with coeliac disease. Arch Dis Child. 1966 Oct;41(219):519-24. doi: 10.1136/adc.41.219.519. No abstract available.
- Background: PubMed 4885929Bayless TM, Christopher NL. Disaccharidase deficiency. Am J Clin Nutr. 1969 Feb;22(2):181-90. doi: 10.1093/ajcn/22.2.181.
- BackgroundChumpitazi BP, Robayo-Torres CC, Tsai CM, Opekun AR, Baker SS, Nichols BL, Gilger MA, Su1110 Yield of prospective disaccharidase testing in children with recurrent abdominal pain. Gastroenterology. May 2013;144(5):S-401-S402.
- Background: PubMed 1767818Cross HS, Quaroni A. Inhibition of sucrose-isomaltase expression by EGF in the human colon adenocarcinoma cells Caco-2. Am J Physiol. 1991 Dec;261(6 Pt 1):C1173-83. doi: 10.1152/ajpcell.1991.261.6.C1173.
- Background: PubMed 2759730Czernichow B, Simon-Assmann P, Kedinger M, Arnold C, Parache M, Marescaux J, Zweibaum A, Haffen K. Sucrase-isomaltase expression and enterocytic ultrastructure of human colorectal tumors. Int J Cancer. 1989 Aug 15;44(2):238-44. doi: 10.1002/ijc.2910440209.
- Background: PubMed 7973215DeMets DL, Lan KK. Interim analysis: the alpha spending function approach. Stat Med. 1994 Jul 15-30;13(13-14):1341-52; discussion 1353-6. doi: 10.1002/sim.4780131308.
- BackgroundEl-Chammas K, Williams S, Miranda A. Su1254 Lactase and sucrase deficiencies in pediatric subjects with chronic abdominal pain. Gastroenterology. May 2014;146(5):S-415-S416.
- Background: PubMed 10067723Gupta SK, Chong SK, Fitzgerald JF. Disaccharidase activities in children: normal values and comparison based on symptoms and histologic changes. J Pediatr Gastroenterol Nutr. 1999 Mar;28(3):246-51. doi: 10.1097/00005176-199903000-00007.
- BackgroundHatcher RA, Trussell J, Stewart F, Nelson AL, Cates W, Guest F, Kowal DD, editors. Contraceptive Technology. New York: Ardent Media, 2004:226.
- Background: PubMed 1904933Heitlinger LA, Rossi TM, Lee PC, Lebenthal E. Human intestinal disaccharidase activities: correlations with age, biopsy technique, and degree of villus atrophy. J Pediatr Gastroenterol Nutr. 1991 Feb;12(2):204-8.
- Background: PubMed 7055778Hyams JS, Batrus CL, Grand RJ, Sallan SE. Cancer chemotherapy-induced lactose malabsorption in children. Cancer. 1982 Feb 15;49(4):646-50. doi: 10.1002/1097-0142(19820215)49:43.0.co;2-m.
- BackgroundKerry KR, & Townley, R. R. Genetic aspects of intestinal sucrase-isomaltase deficiency. Australian Paediatric Journal. 1965;1:223.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Sucraid in people with Genetic Sucrase-Isomaltase Deficiency, run by QOL Medical, LLC.
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 9 inclusion and 13 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: withdrawn, as of the sponsor's last update on 25 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Response to Sucraid and placebo in a parallel group study based on improvement in daily stool consistency, as assessed by the BSFS using SHMBT (time frame: Up to 2 years). 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 NCT02784067, which also lists 26 linked publications.
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 25 Sep 2017.