Short Gut SyndromePhase 2NCT04604275

Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

A Phase 2 study testing Sucrase in people with Short Gut Syndrome, run by University of Miami at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

Who is it for?: Any age
Conditions the study is for: Short Gut Syndrome
What is being tested?: Sucrase
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 3 participants
Where: 2 sitesUnited States
Study dates: 31 Jan 2022to 1 Sep 2024
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Short bowel syndrome, of all ages, with dependence on parental support to provide at least 50% of fluid or caloric needs.
Must be on diet containing sucrose.
Must be willing and able to sign informed consent
Adult and Pediatric patients (all ages)

Who cannot join

Current IV antibiotic administration for confirmed bout of bacteremia.
No enteral nutrition
Any condition, disease, illness, or circumstance that in the investigator's opinion puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Jackson Memorial Hospital
Miami, Florida, 33136
University of Miami
Miami, Florida, 33136

Other trials in United States

About this study

Short gut syndrome with intestinal failure patients may have decreased production of disaccharidases, like sucrase, an enzyme responsible for digesting sugar in foods. This can happen due to loss of bowel length from surgery or from loss of cellular function in the intestines due to use of parenteral nutrition intravenously. Therefore, patients with these conditions may not be able to digest sucrose (sugar) fully. Patients might experience abdominal distension/pain, vomiting and diarrhea when sugar is taken in orally or through the g-tube, which can limit patients' ability to increase oral or g-tube feeds in short gut syndrome patients with intestinal failure.

In patients with short gut syndrome and intestinal failure, the administration of exogenous sucrase (enzyme) may improve sucrose (sugar) digestion and thus the ability to tolerate more oral or g-tube feeds.

Conditions: Short Gut Syndrome
Being tested: Sucrase, Placebo
Keywords: Intestinal failure
Medical subject headings: Short Bowel Syndrome, Intestinal Failure

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SucraseDrug
1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
PlaceboOther
1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.

Study groups in NCT04604275
Study group	Role	What they receive
Sucrase intervention followed by placebo	Experimental	Participants in this arm will receive sucrase for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of placebo. Receives: Drug: Sucrase; Other: Placebo
Placebo followed by sucrase intervention	Experimental	Participants in this arm will receive placebo for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of sucrase. Receives: Drug: Sucrase; Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in Carbohydrate Malabsorption
Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.
Measured: baseline, 9 weeks
Change in Carbohydrate Malabsorption as Measured by Patient Symptom Survey
Degree of carbohydrate malabsorption will be assessed by change in patient symptomatology by change in score on patient symptom survey.
The survey has range from 0-52 with higher score being worse symptoms and lower being better.
Measured: baseline, 9 weeks
Change in Carbohydrate Malabsorption as Measured by Growth Velocity
Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week
Measured: baseline, 9 weeks
Change in Carbohydrate Malabsorption as Measured by Enteral Nutrition Tolerance
Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day
Measured: baseline, 9 weeks

Secondary outcomes (3)

Change in Digestion
Change in digestion will be measured by change in abdominal distension/girth measured in cm
Measured: baseline, 9 weeks
Change in Digestion as Measured by Amount of Emesis
Change in digestion will be assessed by number of emesis per day
Measured: baseline, 9 weeks
Change in Digestion as Measured by Stool Consistency
Change in digestion will be assessed as a change in stool consistency from liquid (7) to solid(1) using Bristol stool chart
Measured: baseline, 9 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

31 Jan 20221 Sep 2024

First posted27 Oct 2020
Study start31 Jan 2022
Primary completion1 Sep 2024
Study completion1 Sep 2024

Why it stopped: Inability to recruit further patients for study

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Short Gut SyndromeStudies of the same condition, open and under way.
More trials run by University of MiamiThe 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04604275
Sponsor's protocol ID: 20191253
Status last verified: Sep 2025
First submitted: 22 Oct 2020
Last updated: 1 Oct 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
Short Bowel Syndrome
Not yet recruitingPhase 2
Efficacy and Safety of TRA16 Administered Orally in Patients With Enteric Hyperoxaluria (EH).
Enteric Hyperoxaluria
RecruitingPhase 3
Efficacy And Safety Evaluation of Glepaglutide in Treatment of SBS
Short Bowel Syndrome
Active, not recruitingObservational study
Registry for Participants With Short Bowel Syndrome
Short Bowel Syndrome

All Short Gut Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure
ClinicalTrials.gov ID: NCT04604275
Acronym / sponsor ID: 20191253
Conditions: Short Gut Syndrome
Keywords: Intestinal failure
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Inability to recruit further patients for study
Enrolment: 3 participants (actual)
Lead sponsor: University of MiamiUniversity, hospital or other
Collaborators: QOL Medical, LLC
Study start: 31 Jan 2022 (actual)
Primary completion: 1 Sep 2024 (actual)
Study completion: 1 Sep 2024 (actual)
First posted: 27 Oct 2020 (actual)
Results: Posted 1 Oct 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Sucrase intervention followed by placebo	Experimental	Drug: Sucrase, Other: PlaceboParticipants in this arm will receive sucrase for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of placebo.
Placebo followed by sucrase intervention	Experimental	Drug: Sucrase, Other: PlaceboParticipants in this arm will receive placebo for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of sucrase.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

Short bowel syndrome, of all ages, with dependence on parental support to provide at least 50% of fluid or caloric needs.
Must be on diet containing sucrose.
Must be willing and able to sign informed consent
Adult and Pediatric patients (all ages)

Key exclusion criteria (3 listed)

Current IV antibiotic administration for confirmed bout of bacteremia.
No enteral nutrition
Any condition, disease, illness, or circumstance that in the investigator's opinion puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results

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
Sucrase1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.	Drug	Placebo followed by sucrase intervention, Sucrase intervention followed by placebo
Placebo1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.	Other	Placebo followed by sucrase intervention, Sucrase intervention followed by placebo

Primary outcome

Change in Carbohydrate Malabsorption
Time frame: baseline, 9 weeks
Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.
Change in Carbohydrate Malabsorption as Measured by Patient Symptom Survey
Time frame: baseline, 9 weeks
Degree of carbohydrate malabsorption will be assessed by change in patient symptomatology by change in score on patient symptom survey. The survey has range from 0-52 with higher score being worse symptoms and lower being better.
Change in Carbohydrate Malabsorption as Measured by Growth Velocity
Time frame: baseline, 9 weeks
Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week
Change in Carbohydrate Malabsorption as Measured by Enteral Nutrition Tolerance
Time frame: baseline, 9 weeks
Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day

3 secondary outcomes

Change in Digestion
Time frame: baseline, 9 weeks
Change in Digestion as Measured by Amount of Emesis
Time frame: baseline, 9 weeks
Change in Digestion as Measured by Stool Consistency
Time frame: baseline, 9 weeks

Results and safety

Results posted to the registry on 1 Oct 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Sucrase Intervention Followed by Placebo	2	2
Placebo Followed by Sucrase Intervention	1	1

Primary outcome: Change in Carbohydrate Malabsorption

Time frame: baseline, 9 weeks

Population analysed: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.

Primary outcome: Change in Carbohydrate Malabsorption as Measured by Patient Symptom Survey

Time frame: baseline, 9 weeks

Primary outcome: Change in Carbohydrate Malabsorption as Measured by Growth Velocity

Time frame: baseline, 9 weeks

Primary outcome: Change in Carbohydrate Malabsorption as Measured by Enteral Nutrition Tolerance

Time frame: baseline, 9 weeks

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: 9 weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Sucrase Intervention Followed by Placebo	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Placebo Followed by Sucrase Intervention	0 of 1 (0%)	0 of 1 (0%)	0 of 1

Limitations and caveats (from the sponsor): The study was terminated due to low accrual. The target population consisted of individuals with a rare condition that is not commonly found in the general population, which significantly limited recruitment feasibility. Despite outreach efforts and engagement with specialized clinical sites, the rarity of the condition and strict eligibility criteria resulted in insufficient enrollment.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Short Gut Syndrome

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
Short Bowel Syndrome
Not yet recruitingPhase 2
Efficacy and Safety of TRA16 Administered Orally in Patients With Enteric Hyperoxaluria (EH).
Enteric Hyperoxaluria
RecruitingPhase 3
Efficacy And Safety Evaluation of Glepaglutide in Treatment of SBS
Short Bowel Syndrome
Active, not recruitingObservational study
Registry for Participants With Short Bowel Syndrome
Short Bowel Syndrome

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.

Amanda Fifi, MD principal investigator

University of Miami

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sucrase in people with Short Gut Syndrome, run by University of Miami at 2 sites in United States.

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 4 inclusion and 3 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: stopped early, as of the sponsor's last update on 1 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. 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.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change in Carbohydrate Malabsorption (time frame: baseline, 9 weeks). The study lists 4 primary outcomes in all. 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 NCT04604275.

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 1 Oct 2025.