Overview

This trial has been completed.

Condition healthy volunteer subjects
Treatments trehalose for iv infusion, saline 0.9% iv
Phase phase 1
Sponsor Bioblast Pharma Ltd.
Collaborator Parexel
Start date March 2016
End date June 2016
Trial size 24 participants
Trial identifier NCT02725957, BB-TRE-101

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification pharmacokinetics study
Intervention model parallel assignment
Masking double blind (subject, investigator, outcomes assessor)
Arm
(Experimental)
Single dose administration of Trehalose 9% for IV infusion.
trehalose for iv infusion
(Placebo Comparator)
Single dose administration of 0.9% saline in the same volume and duration as Treatment Arm 1 (9% trehalose)
saline 0.9% iv

Primary Outcomes

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

Measure
Maximum-tolerated dose (MTD) of trehalose administered intravenously (Averse events, vitals signs)
time frame: 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
time frame: 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
time frame: Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.

Eligibility Criteria

Male or female participants from 18 years up to 55 years old.

Inclusion Criteria: 1. Healthy men and women between 18 and 55 years (inclusive) of age 2. Body Mass Index (BMI) 19 to 29.9 kg/m2 (inclusive) and weighing at least 55 kg. 3. Subjects in general good health in the opinion of the investigator 4. Blood pressure and heart rate within normal limits 5. 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. Exclusion Criteria: 1. Diabetes mellitus type 1 or 2 or HbA1c > 5.6 % at Screening 2. History of significant medical disorder 3. Any clinically significant abnormality in safety laboratory tests during the Screening period (Day -28 to -1) 4. Known contraindication, hypersensitivity and/or allergy to trehalose 5. Any acute illness (e.g. acute infection) within 72 hours 6. Participation in another clinical trial with drugs received within 3 months prior to dosing 7. Positive serum pregnancy test determined during the Screening period or currently lactating women 8. ECG with clinically significant finding recorded during the Screening period 9. Positive HIV, Hepatitis B or Hepatitis C serology at Screening 10. Known history of alcohol or drug abuse in the past 5 years 11. Positive urinary drug screen determined during the Screening period

Additional Information

Official title A Single Center, Single Ascending Dose, Double-Blind, Randomized, Placebo-Controlled Trial to Establish Safety and the Maximum Tolerated Dose of Intravenous Trehalose Solution in Healthy Subjects
Description This is 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. Cohorts 1 to 3 After all subjects in a given cohort complete their 1-week follow-up visit (Visit 4), a Safety Review Committee (SRC) will review the safety and PK data of that cohort. If no safety concerns are identified, and the exposure data supports a higher dose is acceptable, the SRC will approve continuation into the next cohort (dose level). Cohort 4 Cohort 4 will be initiated based on review of the safety and exposure data from the first 3 cohorts by the SRC. This cohort will only be performed if there is a suggestion that exposure can be safely increased.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Bioblast Pharma Ltd..