Short Bowel SyndromePhase 4NCT00742157
Evaluate the Efficacy and Safety Growth Hormone, Glutamine and Diet in Patients With Short Bowel Syndrome (SBS)
A Phase 4 study testing Growth Hormone in people with Short Bowel Syndrome, run by University of Nebraska at 1 site in United States.
Trial summary
Treatment for 3.5-8 weeks with GH (0.05 mg/kg/day) +GLN+Diet, followed by continued compliance to the individualized oral diet and enteral GLN, will result in reduced volume of TPN infusion/week and/or reduced frequency of TPN infusions/week. Read more about this study
- Who is it for?
- Ages 19 to 78 years
- Conditions the study is for
- Short Bowel Syndrome
- What is being tested?
- Growth Hormone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2003to 20 Oct 2008
- Phase
- Phase 4
- 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 19 to 78 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be male or female between 19 and 78 years of age, inclusive
- Have a diagnosis of SBS and 1 or more of the following characteristics:
- Dependent upon TPN and/or IV fluids
- Unable to receive TPN because of a documented history of complications associated with the long-term parenteral infusions (e.g. lack of venous access, multiple septic events, progressive liver dysfunction while on TPN, etc)
- A documented remnant bowel anatomy that is inconsistent with adequately supporting life without parenteral support (e.g. < 70 cm of healthy small intestine and a portion of colon, or less than 150 cm in patients with no colon)
- A documented history of diarrhea and/or malabsorption that has significantly compromised the patient's nutritional and hydration status (e.g. significant weight loss - 5% over 1 month or 10% over 6 months, or a serum albumin < 3.5 g/dl, documented nutrient deficiencies, documented episodes of dehydration, etc).
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- Is able to eat at least some (>500 calories) of solid food on a regular basis or tolerate at least some (>500 calories) of an enteral feeding formula
- Have stable liver and renal function
- For patients with known hypertension and other cardiovascular disorders, be both compensated and stabilized on a regular therapeutic regimen
- For women participating in the study, manifest or give assent to 1 of the following criteria to ensure that the patient does not become pregnant during the study:
- The patient must be surgically sterile or demonstrably postmenopausal.
- Any patient capable of becoming pregnant must have a negative urine pregnancy test and must agree to practice a method of contraception documented to have at least 90% reliability throughout the treatment period.
- Have the ability to understand the requirements of the study, to provide written Informed Consent, and to abide by the study restrictions and agree to complete the required assessments in the Follow-up Period
Who cannot join
- Be pregnant or lactating
- Have a history of mental deficiency or illness that might compromise compliance with the requirements of the study
- Have clinically serious neurological dysfunction
- Have hypoxemic pulmonary diseases (i.e., resting pAO2 < 75 torr)
- Have unstable ischemic heart disease or uncompensated cardiac failure
- Have participated in any study involving an investigations drug within 30 days prior to entry into this trial.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Nebraska Medical Center
Omaha, Nebraska, 68198
About this study
Official title: An Open-label Evaluation of the Long-term Efficacy and Safety of a Standardized Regimen of Growth Hormone, Glutamine and a Modified Diet in the Treatment of Patients With Short Bowel Syndrome
Treatment for 3.5-8 weeks with GH (0.05 mg/kg/day) +GLN+Diet, followed by continued compliance to the individualized oral diet and enteral GLN, will result in reduced volume of TPN infusion/week and/or reduced frequency of TPN infusions/week.
Read the detailed description
Long-term total parenteral nutrition (TPN) is a supportive, rather than curative, therapy for patients with severe short bowel syndrome (SBS). Because of the complications (liver and kidney dysfunction, bone demineralization, nutrient deficiencies, catheter sepsis) and costs (>$100,000/ patient/year) associated with this therapy, researchers have attempted to identify a safe, cost-effective alternative treatment modality. Various surgical procedures (including bowel transplantation) have been explored; however, these options currently offer limited clinical efficacy and significant morbidity and mortality. In contrast, a non-invasive therapy utilizing a growth factor (growth hormone - GH) and a bowel-specific nutrient (glutamine - GLN) in combination with an individualized oral diet (GH+GLN+Diet) has recently been shown to significantly enhance nutrient absorption and eliminate or reduce TPN requirements in patients with severe SBS.
This open-labeled, single-center trial with a total enrollment of 30 patients with severe SBS will examine the safety and effectiveness of a lower dose (0.05 mg/kg/day) of growth hormone. Thirty-two subjects have previously been studied at the Nutritional Restart Center in Massachusetts utilizing a higher dose (0.1 mg/kg/day) of growth hormone and identical treatment parameters as described in this protocol. The University of Nebraska Medical Center and the Nutritional Restart Center have agreed to pool the data from both studies and analyze the data according to the same primary efficacy variable. Subjects recruited to the lower-dose growth hormone group will be matched, using specific clinical criteria, to patients in the higher dose growth hormone group. The primary efficacy variable will be the change in volume of TPN infusion/week and frequency of TPN infusions/week at 6 months following discharge compared to baseline.
After a 3 day, baseline evaluation at The Nebraska Medical Center to determine pre-treatment TPN requirements and to assess the specific indices of nutritional and hydration status and kidney and liver function, and physical functioning capacity, patients will begin to receive lower dose GH (0.05 mg/kg/day) in combination with GLN (30 grams/day, orally) and the individualized oral/enteral diet. Treatment will last for a minimum of 23 days and a maximum of 54 days. Duration of treatment will be tailored to the patients' individual needs (e.g., understanding and acceptance of the modified diet, successful weaning of TPN). Patients may return to their home to continue receiving the GH treatment, provided they are tolerating the GH injections and understand how to properly administer the injections.
- Conditions
- Short Bowel Syndrome
- Being tested
- Growth Hormone
- Keywords
- Growth Hormone, TPN, Total Parenteral Nutrition, Glutamine, Short Bowel Syndrome, Increase in absorption of small bowel.
- Medical subject headings
- Short Bowel Syndrome, Hyperphagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Growth HormoneDrug
Also called Humatrope = Somatropin (rDNA origin) for injection
dosage = 0.05mg/kg/day, in diluent for injection, once a day, for 23 - 54 days.
| Study group | Role | What they receive |
|---|---|---|
| UNMC Group | Other | Compare the low and high dose effects of Growth Hormone from previously pooled patients (high dose) and UNMC patients (low dose). Receives: Drug: Growth Hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Documenting the Reduction of TPN Infusion/Week for Participants at 6 Months Compared to Baseline.
Evaluate the number of participants dosed at UNMC who had a reduction in the frequency of TPN infusions/week at 6 months compared to baseline
Measured: 6 months
Secondary outcomes (4)
Change in Hydration Status
Number of participants who were able to maintain euvolemic status at 6 months post treatment
Measured: 6 months
Change in Kidney Function at 6 Months Post Treatment
Number of participants with a significant change in kidney function at 6 months post treatment as compared to baseline.
Measured: 6 months
Change in Liver Function at 6 Months Post Treatment
Number of participants with an improvement in their liver function ALT at 6 months post treatment as compared to baseline.
Measured: 6 months
Change in Physical Functioning Capacity at 6 Months Post Treatment as Compared to Baseline
Number of participants who had a change in their ability to complete all activities of daily living by self at 6 months compared to baseline.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2008 (estimated)
- Study start1 Sep 2003
- Primary completion20 Oct 2008
- Study completion20 Oct 2008
Why it stopped: Lack of Enrollment
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 Bowel SyndromeStudies of the same condition, open and under way.
- More trials run by University of NebraskaThe 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Nebraska
- Sponsor type
- University, hospital or other
- Collaborators
- Eli Lilly and Company
- Responsible party
- Sponsor
- Investigators
- Fedja Rochling, MB, ChB, Principal investigator, University of Nebraska
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00742157
- Sponsor's protocol ID
- 0061-03-FB
- Status last verified
- Aug 2023
- First submitted
- 26 Aug 2008
- Last updated
- 8 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Evaluation of the Long-term Efficacy and Safety of a Standardized Regimen of Growth Hormone, Glutamine and a Modified Diet in the Treatment of Patients With Short Bowel Syndrome
- ClinicalTrials.gov ID
- NCT00742157
- Acronym / sponsor ID
- 0061-03-FB
- Conditions
- Short Bowel Syndrome
- Keywords
- Growth Hormone, TPN, Total Parenteral Nutrition, Glutamine, Short Bowel Syndrome, Increase in absorption of small bowel.
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of Enrollment
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University of NebraskaUniversity, hospital or other
- Collaborators
- Eli Lilly and Company
- Study start
- 1 Sep 2003 (actual)
- Primary completion
- 20 Oct 2008 (actual)
- Study completion
- 20 Oct 2008 (actual)
- First posted
- 27 Aug 2008 (estimated)
- Results
- Posted 28 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Treatment for 3.5-8 weeks with GH (0.05 mg/kg/day) +GLN+Diet, followed by continued compliance to the individualized oral diet and enteral GLN, will result in reduced volume of TPN infusion/week and/or reduced frequency of TPN infusions/week.
Detailed description
Long-term total parenteral nutrition (TPN) is a supportive, rather than curative, therapy for patients with severe short bowel syndrome (SBS). Because of the complications (liver and kidney dysfunction, bone demineralization, nutrient deficiencies, catheter sepsis) and costs (>$100,000/ patient/year) associated with this therapy, researchers have attempted to identify a safe, cost-effective alternative treatment modality. Various surgical procedures (including bowel transplantation) have been explored; however, these options currently offer limited clinical efficacy and significant morbidity and mortality. In contrast, a non-invasive therapy utilizing a growth factor (growth hormone - GH) and a bowel-specific nutrient (glutamine - GLN) in combination with an individualized oral diet (GH+GLN+Diet) has recently been shown to significantly enhance nutrient absorption and eliminate or reduce TPN requirements in patients with severe SBS.
This open-labeled, single-center trial with a total enrollment of 30 patients with severe SBS will examine the safety and effectiveness of a lower dose (0.05 mg/kg/day) of growth hormone. Thirty-two subjects have previously been studied at the Nutritional Restart Center in Massachusetts utilizing a higher dose (0.1 mg/kg/day) of growth hormone and identical treatment parameters as described in this protocol. The University of Nebraska Medical Center and the Nutritional Restart Center have agreed to pool the data from both studies and analyze the data according to the same primary efficacy variable. Subjects recruited to the lower-dose growth hormone group will be matched, using specific clinical criteria, to patients in the higher dose growth hormone group. The primary efficacy variable will be the change in volume of TPN infusion/week and frequency of TPN infusions/week at 6 months following discharge compared to baseline.
After a 3 day, baseline evaluation at The Nebraska Medical Center to determine pre-treatment TPN requirements and to assess the specific indices of nutritional and hydration status and kidney and liver function, and physical functioning capacity, patients will begin to receive lower dose GH (0.05 mg/kg/day) in combination with GLN (30 grams/day, orally) and the individualized oral/enteral diet. Treatment will last for a minimum of 23 days and a maximum of 54 days. Duration of treatment will be tailored to the patients' individual needs (e.g., understanding and acceptance of the modified diet, successful weaning of TPN). Patients may return to their home to continue receiving the GH treatment, provided they are tolerating the GH injections and understand how to properly administer the injections.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UNMC Group | Other | Drug: Growth HormoneCompare the low and high dose effects of Growth Hormone from previously pooled patients (high dose) and UNMC patients (low dose). |
Who may qualify
- Ages
- 19 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Be male or female between 19 and 78 years of age, inclusive
- Have a diagnosis of SBS and 1 or more of the following characteristics:
- Dependent upon TPN and/or IV fluids
- Unable to receive TPN because of a documented history of complications associated with the long-term parenteral infusions (e.g. lack of venous access, multiple septic events, progressive liver dysfunction while on TPN, etc)
- A documented remnant bowel anatomy that is inconsistent with adequately supporting life without parenteral support (e.g. < 70 cm of healthy small intestine and a portion of colon, or less than 150 cm in patients with no colon)
Key exclusion criteria (6 listed)
- Be pregnant or lactating
- Have a history of mental deficiency or illness that might compromise compliance with the requirements of the study
- Have clinically serious neurological dysfunction
- Have hypoxemic pulmonary diseases (i.e., resting pAO2 < 75 torr)
- Have unstable ischemic heart disease or uncompensated cardiac failure
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 |
|---|---|---|
| Growth HormoneAlso: Humatrope = Somatropin (rDNA origin) for injectiondosage = 0.05mg/kg/day, in diluent for injection, once a day, for 23 - 54 days. | Drug | UNMC Group |
Primary outcome
Documenting the Reduction of TPN Infusion/Week for Participants at 6 Months Compared to Baseline.
Time frame: 6 months
Evaluate the number of participants dosed at UNMC who had a reduction in the frequency of TPN infusions/week at 6 months compared to baseline
4 secondary outcomes
Change in Hydration Status
Time frame: 6 months
Change in Kidney Function at 6 Months Post Treatment
Time frame: 6 months
Change in Liver Function at 6 Months Post Treatment
Time frame: 6 months
Change in Physical Functioning Capacity at 6 Months Post Treatment as Compared to Baseline
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Registry for Participants With Short Bowel Syndrome
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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.
Fedja Rochling, MB, ChB principal investigator
University of Nebraska
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Growth Hormone in people with Short Bowel Syndrome, run by University of Nebraska at 1 site 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 13 inclusion and 6 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 8 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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?
Yes. Results are posted in the registry (first posted 28 Jul 2023). 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?
Documenting the Reduction of TPN Infusion/Week for Participants at 6 Months Compared to Baseline. (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Sep 2023.