Safety and Tolerability of SYNB1020-CP-001 (SYNB1020CP001)

A Phase 1, First-in-human, Oral Single and Multiple Dose-Escalation, Randomized, Double-blinded, Placebo-controlled Study of SYNB1020 in Healthy Adult Volunteers to Evaluate Safety, Tolerability, Dosing, and Pharmacodynamics

A Phase 1, First-in-human, Oral Single and Multiple Dose-Escalation, Randomized, Double-blinded, Placebo-controlled Study of SYNB1020 in Healthy Adult Volunteers to Evaluate Safety, Tolerability, Dosing, and Pharmacodynamics

Study Overview

• Status: Completed
• Conditions: Healthy Volunteer; Urea Cycle Disorder
• Intervention / Treatment: Drug: Placebo; Drug: SYNB1020

Detailed Description

This Phase 1, dose-escalating, randomized, double-blinded study will evaluate SYNB1020 in placebo-controlled cohorts within the following 2 study parts:

Part 1: A single-ascending dose (SAD) study conducted in an inpatient setting over 6 days in healthy volunteer male and female subjects evaluated in up to 7 dose cohorts to identify the maximum tolerated dose (MTD) within the single dose range studied; and Part 2: A multiple-ascending dose (MAD) study conducted in an inpatient setting over 22 days in healthy volunteer male and female subjects evaluated in up to 4 dose cohorts that were proven tolerable in the SAD part of the study to identify the MTD of SYNB1020 within the multiple-dose range studied. Up to 48 subjects may be enrolled in this part of the study.

Subjects will be screened for eligibility within 30 days prior to enrollment, with evaluations of exercise habits, gastrointestinal (GI) signs and symptoms, fecal patterns (frequency, consistency), recent or current antibiotic exposure, and laboratory measurements. Eligible patients will be admitted to an inpatient facility for investigational product (IP) administration, safety monitoring, and collection of blood, urine, and fecal samples for pharmacokinetic and pharmacodynamic evaluations.

• Study Type: Interventional
• Enrollment (Actual): 52
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Brooklyn, Maryland, United States, 21225
      • Parexel

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 64 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Age 18 to 64 years
• Healthy volunteer Males and Females; Females must be of non childbearing potential
• Able and willing to complete informed consent process
• Available for and agree to all study procedures
• Screening Labs within normal range

Key Exclusion Criteria:

• Acute or chronic medical, surgical, psychiatric, social or laboratory abnormality
• Body mass index < 18.5 or ≥ 30 kg/m2
• Intolerance of or allergic reaction to E. coli Nissle or any of the ingredients in SYNB1020 or placebo formulations; allergies to common foods (e.g., eggs, milk, soy, nuts).
• Prior participation in a study with SYNB1020
• Evidence or history of clinical signification hematological, renal, endocrine, pulmonary, GI cardiovascular, hepatic, psychiatric, neurologic or allergic disease
• Personal or family history of UCD

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: SYNB1020	Drug: SYNB1020; Investigational Product
PLACEBO_COMPARATOR: Placebo; 100 mL masking solution	Drug: Placebo; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events, Laboratory Assessments and ECGs to measure Safety and Tolerability of SYNB1020	Will be measured by assessing nature and frequency of AEs, Laboratory Assessments, and ECGs	3 months from study entry

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
GI tolerability measured using the Gastrointestinal Symptom Rating Scale (GSRS)	Will be measured using the Gastrointestinal Symptom Rating Scale (GSRS)	1 month of study entry
SYNB1020 kinetics measured by qPCR fecal assays	Will be measured by qPCR fecal assays	3 months from study entry

Collaborators and Investigators

• Sponsor: Synlogic

Study record dates

Study Major Dates

• Study Start (ACTUAL): June 16, 2017
• Primary Completion (ACTUAL): November 2, 2017
• Study Completion (ACTUAL): April 11, 2018

Study Registration Dates

• First Submitted: June 6, 2017
• First Submitted That Met QC Criteria: June 6, 2017
• First Posted (ACTUAL): June 7, 2017

Study Record Updates

• Last Update Posted (ACTUAL): May 12, 2021
• Last Update Submitted That Met QC Criteria: May 11, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Urea Cycle Disorders, Inborn
• Other Study ID Numbers: SYNB1020-CP-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No