Proof of Concept Study in Patients With Short Bowel Syndrome

Short Bowel Syndrome Clinical Trial

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)

Status Active, not recruiting

Clinical 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. 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Short Bowel Syndrome
• Syndrome

• NCT number: NCT04379856
• Study type: Interventional
• Source: 9 Meters Biopharma, Inc.
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: June 23, 2020
• Completion date: December 31, 2020