Short Bowel SyndromePhase 1/2NCT04379856
Proof of Concept Study in Patients With Short Bowel Syndrome
A Phase 1/2 study testing NM-002 in people with Short Bowel Syndrome, run by 9 Meters Biopharma, Inc. at 1 site in United States.
Trial summary
This is a Phase I/II. proof of concept, open label, two-dose, dose escalation study of NM-002 in adult patients with SBS who previously responded to exenatide. NM-002 is planned to be administered twice, at up to 3 different dose levels, in up to 3 cohorts, each consisting of 3-4 patients. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Short Bowel Syndrome
- What is being tested?
- NM-002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Jun 2020to 31 Dec 2020
- Phase
- Phase 1/2
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females with SBS secondary to surgical resection of small intestine, with or without an intact colon
- 18-75 years of age at the time of screening
- Female subjects must be postmenopausal (at least 2 years prior to dosing) or surgically sterile or agree to use an acceptable form of birth control from screening until 30 days after last dose. If oral contraceptives are used, Subject must have been on a stable dose for ≥6 months. (See below, "Participation of Women", for additional detail.)
- Male subjects must agree to use an acceptable form of birth control during the study and for 90 days after the last dose. Male subjects may not donate sperm for 90 days after last dose.
- At least 6 months since last surgical bowel resection
- Patients may be on Parenteral support (nutrition and/or fluid and electrolytes [PS]) for at least some of their nutritional needs.
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- If on PS, stable administration of PS volume for 1 month (±20% vol) prior to enrollment.
- Able to ingest solid foods and drink
- Willing to adhere to a defined oral intake of fluids on certain days as required by protocol and based on the individual's routine daily consumption.
Who cannot join
- Positive results on the HIV, Hepatitis, or drug screens.
- Pregnancy or lactation
- Body mass index <18 or >30 kg/m2
- Clinically significant intestinal adhesions and/or chronic abdominal pain
- Active Crohn's disease or IBD (as evaluated by standard procedures employed by the investigator/institution). If in remission, must be ≥12 weeks of remission prior to enrollment.
- If on chronic systemic narcotics, the patient must have been on a stable dose for >12 weeks
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- Inflammatory bowel disease patients who have NOT been on a stable drug treatment regimen for at least the past 3 months.
- Visible blood in the stool within the last 3 months
- Catheter sepsis experienced within the last 3 months
- Known heart failure or active coronary disease
- Known celiac disease
- Radiation enteritis, scleroderma, coeliac disease, refractory or tropical sprue, diabetes
- Alcohol or drug abuse within the last 12 months
- Inadequate hepatic function as defined by: ALT and ASAST both >2.0X ULN; TBL >2X ULN; or ALP >2.5X ULN
- Inadequate renal function as defined by serum creatinine <0.7 or >1.3 mg/dL (in men) and <0.6 or >1.1 mg/dL in women.
- Personal or family history of medullary thyroid cancer.
- History of pancreatitis.
- Any patient who receives insulin in their PS.
- Any hospitalization within 1 month before screening visit
- Systemic corticosteroids, methotrexate, cyclosporine, tacrolimus, sirolimus, infliximab, or mycophenolate mofetil (CellCept®) within 30 days of screening
- Any use of growth hormone, or growth factors such as native GLP-2 or GLP-2 analog (teduglutide) within the last 3 months
- Use of antibiotics within the last 30 days
- Subject not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements.
Trial locations and contacts
1 site in 1 country.
United States1 site
Research Facility One
Los Angeles, California, 90048
About this study
Official title: A Phase I/II Proof of Concept, Open-Label, Repeat Dose, Dose Escalation Study of NM-002 in Adult Patients With Short Bowel Syndrome (SBS)
This is a Phase I/II. proof of concept, open label, two-dose, dose escalation study of NM-002 in adult patients with SBS who previously responded to exenatide. NM-002 is planned to be administered twice, at up to 3 different dose levels, in up to 3 cohorts, each consisting of 3-4 patients. Doses will be administered on Days 1 and 15 by subcutaneous injection. Patients will be monitored for their usage of parenteral supplementation, and will fill out a daily diary for their symptoms of SBS. Urine output will be measured on a daily basis. Patients will be followed for 6 weeks after the second dose.
Read the detailed description
During a baseline period, all subjects will remain on their current prescription for administration of PS. Between Days -7 to -5 patients who have met initial screening criteria will return to the clinic and be enrolled into the study. Patients will be trained on diary usage and patients receiving the SBS meds will be instructed to discontinue use of their medications; Imodium, lomotil, and opium tincture.
On Day -4, subjects will begin diary entries. For 48 hours, on Days -1 and 0, patients will record in their diaries total urine output, PS usage, stool output and oral liquid intake.
On Study Day -1, patients will visit the clinic for baseline safety assessment, and review of symptoms of malabsorption and PS usage. Baseline SF36 and Bowel Symptom questionnaires will be administered.
On Day 1 of treatment, patients will return to the clinic with a sample of urine collected during the baseline run-in for urinalysis. They will have blood samples drawn for clinical laboratory assessment and for pre-dose PK levels, anti-drug antibody, and AE assessments. Following administration of study drug on Day 1, subjects will undergo additional serial PK blood draws.
Serial blood samples for the pharmacokinetic evaluation of NM-002 will be drawn on Days 1 and 15, with single samples obtained on 2, 3, 4, 5, 8, 16, 17, 18, 19, 22 and 29. Additional blood samples will be taken for clinical laboratory evaluations.
Blood glucose meters will be provided to patients to record their daily blood glucose levels, and to check their blood glucose should any symptoms related to hypoglycemia occur. Patients will receive written instructions regarding the symptoms of hypoglycemia and how to manage them at home. They will be provided with glucose gel tubes and instructed in their use.
At any time on Days 6 through 24, the investigator may call for a reduction or increase in PS, based on patient diary data and his/her clinical judgment. If a change in PS occurs, serum electrolytes will be assessed within 48 hours of implementing the change in PS.
If patients have experienced a reduction in PS administration during the study, on Day 25 they will be returned to their pre-study, standard PS regimen. On Day 27, patients will be asked to return to their individual oral liquid intake established during the baseline run-in period, and they will record their urine output volume for 48 hours (on Days 27 and 28).
Any patients who experience an increase in PS administration over the original baseline level will remain on the new level of PS on Days 27 and 28.
Patients will return to the clinic on Day 29. Blood samples will be drawn for clinical laboratory analysis and for PK. Patients will continue to make daily diary entries through Day 55. Telephone follow-ups will be conducted by the investigational site on Days 35±1 and 42±1 to assess any requirement for PS adjustment.
Patients will return to the clinic on Day 56 ± 2 for a final safety assessment.
- Conditions
- Short Bowel Syndrome
- Being tested
- NM-002
- Medical subject headings
- Short Bowel Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NM-002Drug
NM-002 is a fusion protein, of which the pharmacological active portion is the glucagon-like peptide-1 (GLP-1) analog domain with the same amino acid sequence as the active pharmaceutical ingredient (exenatide)
| Study group | Role | What they receive |
|---|---|---|
| Dose 1 | Experimental | NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15. Receives: Drug: NM-002 |
| Dose 2 | Experimental | NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15. Receives: Drug: NM-002 |
| Dose 3 | Experimental | NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15. Receives: Drug: NM-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the safety and tolerability of repeated doses of NM002 three different dose levels
Assess adverse event profile of each dose and dose level
Measured: 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2020
- Study start23 Jun 2020
- Primary completion11 Dec 2020 (estimated)
- Study completion31 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Short Bowel SyndromeStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- 9 Meters Biopharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04379856
- Sponsor's protocol ID
- NMSBS01-001
- Status last verified
- Nov 2020
- First submitted
- 2 Apr 2020
- Last updated
- 20 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Proof of Concept, Open-Label, Repeat Dose, Dose Escalation Study of NM-002 in Adult Patients With Short Bowel Syndrome (SBS)
- ClinicalTrials.gov ID
- NCT04379856
- Acronym / sponsor ID
- NMSBS01-001
- Conditions
- Short Bowel Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- 9 Meters Biopharma, Inc.Industry (pharma, biotech, devices)
- Study start
- 23 Jun 2020 (actual)
- Primary completion
- 11 Dec 2020 (estimated)
- Study completion
- 31 Dec 2020 (estimated)
- First posted
- 8 May 2020 (actual)
- 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.
This is a Phase I/II. proof of concept, open label, two-dose, dose escalation study of NM-002 in adult patients with SBS who previously responded to exenatide. NM-002 is planned to be administered twice, at up to 3 different dose levels, in up to 3 cohorts, each consisting of 3-4 patients. Doses will be administered on Days 1 and 15 by subcutaneous injection. Patients will be monitored for their usage of parenteral supplementation, and will fill out a daily diary for their symptoms of SBS. Urine output will be measured on a daily basis. Patients will be followed for 6 weeks after the second dose.
Detailed description
During a baseline period, all subjects will remain on their current prescription for administration of PS. Between Days -7 to -5 patients who have met initial screening criteria will return to the clinic and be enrolled into the study. Patients will be trained on diary usage and patients receiving the SBS meds will be instructed to discontinue use of their medications; Imodium, lomotil, and opium tincture.
On Day -4, subjects will begin diary entries. For 48 hours, on Days -1 and 0, patients will record in their diaries total urine output, PS usage, stool output and oral liquid intake.
On Study Day -1, patients will visit the clinic for baseline safety assessment, and review of symptoms of malabsorption and PS usage. Baseline SF36 and Bowel Symptom questionnaires will be administered.
On Day 1 of treatment, patients will return to the clinic with a sample of urine collected during the baseline run-in for urinalysis. They will have blood samples drawn for clinical laboratory assessment and for pre-dose PK levels, anti-drug antibody, and AE assessments. Following administration of study drug on Day 1, subjects will undergo additional serial PK blood draws.
Serial blood samples for the pharmacokinetic evaluation of NM-002 will be drawn on Days 1 and 15, with single samples obtained on 2, 3, 4, 5, 8, 16, 17, 18, 19, 22 and 29. Additional blood samples will be taken for clinical laboratory evaluations.
Blood glucose meters will be provided to patients to record their daily blood glucose levels, and to check their blood glucose should any symptoms related to hypoglycemia occur. Patients will receive written instructions regarding the symptoms of hypoglycemia and how to manage them at home. They will be provided with glucose gel tubes and instructed in their use.
At any time on Days 6 through 24, the investigator may call for a reduction or increase in PS, based on patient diary data and his/her clinical judgment. If a change in PS occurs, serum electrolytes will be assessed within 48 hours of implementing the change in PS.
If patients have experienced a reduction in PS administration during the study, on Day 25 they will be returned to their pre-study, standard PS regimen. On Day 27, patients will be asked to return to their individual oral liquid intake established during the baseline run-in period, and they will record their urine output volume for 48 hours (on Days 27 and 28).
Any patients who experience an increase in PS administration over the original baseline level will remain on the new level of PS on Days 27 and 28.
Patients will return to the clinic on Day 29. Blood samples will be drawn for clinical laboratory analysis and for PK. Patients will continue to make daily diary entries through Day 55. Telephone follow-ups will be conducted by the investigational site on Days 35±1 and 42±1 to assess any requirement for PS adjustment.
Patients will return to the clinic on Day 56 ± 2 for a final safety assessment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose 1 | Experimental | Drug: NM-002NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15. |
| Dose 2 | Experimental | Drug: NM-002NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15. |
| Dose 3 | Experimental | Drug: NM-002NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males and females with SBS secondary to surgical resection of small intestine, with or without an intact colon
- 18-75 years of age at the time of screening
- Female subjects must be postmenopausal (at least 2 years prior to dosing) or surgically sterile or agree to use an acceptable form of birth control from screening until 30 days after last dose. If oral contraceptives are used, Subject must have been on a stable dose for ≥6 months. (See below, "Participation of Women", for additional detail.)
- Male subjects must agree to use an acceptable form of birth control during the study and for 90 days after the last dose. Male subjects may not donate sperm for 90 days after last dose.
- At least 6 months since last surgical bowel resection
Key exclusion criteria (23 listed)
- Positive results on the HIV, Hepatitis, or drug screens.
- Pregnancy or lactation
- Body mass index <18 or >30 kg/m2
- Clinically significant intestinal adhesions and/or chronic abdominal pain
- Active Crohn's disease or IBD (as evaluated by standard procedures employed by the investigator/institution). If in remission, must be ≥12 weeks of remission prior to enrollment.
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 |
|---|---|---|
| NM-002NM-002 is a fusion protein, of which the pharmacological active portion is the glucagon-like peptide-1 (GLP-1) analog domain with the same amino acid sequence as the active pharmaceutical ingredient (exenatide) | Drug | Dose 1, Dose 2, Dose 3 |
Primary outcome
To assess the safety and tolerability of repeated doses of NM002 three different dose levels
Time frame: 56 days
Assess adverse event profile of each dose and dose level
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2020.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NM-002 in people with Short Bowel Syndrome, run by 9 Meters Biopharma, Inc. 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 9 inclusion and 23 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: status not confirmed, as of the sponsor's last update on 20 Nov 2020. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Results are not available in the source record.
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?
To assess the safety and tolerability of repeated doses of NM002 three different dose levels (time frame: 56 days). 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 20 Nov 2020.