Proof of Concept Study in Patients With Short Bowel Syndrome

NCT04379856 | Unknown Phase 1 FDA Drug

• 1 other identifier: NMSBS01-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Short Bowel Syndrome

Outcome Measures

Primary Outcomes (1)

• 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

  56 days

Study Arms (3)

Dose 1

EXPERIMENTAL

NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15.

Drug: NM-002

Dose 2

EXPERIMENTAL

NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15.

Drug: NM-002

Dose 3

EXPERIMENTAL

NM-002 will be administered at the indicated dose by subcutaneous injection on Days 1 and 15.

Drug: NM-002

Interventions

NM-002 DRUG

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)

Dose 1; Dose 2; Dose 3

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females with SBS secondary to surgical resection of small intestine, with or without an intact colon
• 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.
• 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.

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• 9 Meters Biopharma, Inc.: lead

Study Sites (1)

Research Facility One

Los Angeles, California, 90048, United States

Location

MeSH Terms

Conditions

Short Bowel Syndrome

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2020
• First Posted: May 8, 2020
• Study Start: June 23, 2020
• Primary Completion: December 11, 2020
• Study Completion: December 31, 2020
• Last Updated: November 20, 2020

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)