A Study to Establish Safety and Maximum Tolerated Dose of IV Trehalose in Healthy Subjects

NCT02725957 | Completed Phase 1 DMC

• 1 other identifier: BB-TRE-101
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This will be a double-blind, randomized, placebo-controlled, single ascending dose study performed in healthy subjects. The study will include up to four escalating dose cohorts with eight (8) subjects in each cohort. In each cohort, eligible subjects will be randomized in a 3:1 ratio to receive single IV administration of 9% trehalose (Treatment Arm 1) or placebo (Treatment Arm 2). All subjects, regardless of their treatment arm assignment, will undergo the same evaluations and will receive the study drug at the clinic. Each subject will continue to be followed for one week post dosing.

Conditions

Healthy Volunteer Subjects

Keywords

Bioblast

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of escalating doses of intravenously administered trehalose (incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities)

  Safety will be assessed by the incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities.

  Will be assessed during the entire study. At screening, at day -1 before drug administration, and day 1 of drug administration before, during and after drug administration, and at day 8 the follow up visit

Secondary Outcomes (3)

• Maximum-tolerated dose (MTD) of trehalose administered intravenously (Averse events, vitals signs)

  Will be assessed during the entire study. At screening, at day -1 before drug administration, and day 1 of drug administration before, during and after drug administration, and at day 8 the follow up visit

• Pharmacokinetics (PK) of plasma and urine trehalose

  Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.

• Pharmacokinetics (PK) of serum and urine glucose

  Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.

Study Arms (2)

Trehalose 9%

EXPERIMENTAL

Single dose administration of Trehalose 9% for IV infusion.

Drug: Trehalose for IV Infusion

Saline 0.9%

PLACEBO COMPARATOR

Single dose administration of 0.9% saline in the same volume and duration as Treatment Arm 1 (9% trehalose)

Drug: Saline 0.9% IV

Interventions

Trehalose for IV Infusion DRUG

Trehalose 9%

Saline 0.9% IV DRUG

Saline 0.9%

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy men and women between 18 and 55 years (inclusive) of age
• Body Mass Index (BMI) 19 to 29.9 kg/m2 (inclusive) and weighing at least 55 kg.
• Subjects in general good health in the opinion of the investigator
• Blood pressure and heart rate within normal limits
• Female subjects must have a negative serum pregnancy test during the Screening period (Day -28 to -1) and be willing and able to use a medically acceptable method of birth control or be postmenopausal.

You may not qualify if:

• Diabetes mellitus type 1 or 2 or HbA1c \> 5.6 % at Screening
• History of significant medical disorder
• Any clinically significant abnormality in safety laboratory tests during the Screening period (Day -28 to -1)
• Known contraindication, hypersensitivity and/or allergy to trehalose
• Any acute illness (e.g. acute infection) within 72 hours
• Participation in another clinical trial with drugs received within 3 months prior to dosing
• Positive serum pregnancy test determined during the Screening period or currently lactating women
• ECG with clinically significant finding recorded during the Screening period
• Positive HIV, Hepatitis B or Hepatitis C serology at Screening
• Known history of alcohol or drug abuse in the past 5 years
• Positive urinary drug screen determined during the Screening period

Sponsors & Collaborators

• Bioblast Pharma Ltd.: lead
• Parexel: collaborator

Study Sites (1)

PAREXEL Baltimore Early Phase Clinical Unit; Harbor Hospital

Baltimore, Maryland, 21225, United States

Location

MeSH Terms

Interventions

Trehalose; Infusions, Intravenous; Sodium Chloride

Intervention Hierarchy (Ancestors)

Glucans; Polysaccharides; Carbohydrates; Disaccharides; Oligosaccharides; Sugars; Administration, Intravenous; Drug Administration Routes; Drug Therapy; Therapeutics; Infusions, Parenteral; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2016
• First Posted: April 1, 2016
• Study Start: March 1, 2016
• Primary Completion: June 1, 2016
• Study Completion: August 1, 2016
• Last Updated: November 22, 2016

Record last verified: 2016-11

Locations

US (1)