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

NCT ID: NCT02725957

Last Updated: 2016-11-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-03-31
• Study Completion Date: 2016-08-31

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.

Detailed 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.

Conditions

Healthy Volunteer Subjects

Keywords

Bioblast

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Blinding Strategy: TRIPLE

Study Groups

Trehalose 9%

Single dose administration of Trehalose 9% for IV infusion.

Group Type: EXPERIMENTAL

Trehalose for IV Infusion

Intervention Type: DRUG

Saline 0.9%

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

Group Type: PLACEBO_COMPARATOR

Saline 0.9% IV

Intervention Type: DRUG

Interventions

Trehalose for IV Infusion

Intervention Type: DRUG

Saline 0.9% IV

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Parexel

INDUSTRY

Sponsor Role: collaborator

Bioblast Pharma Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

PAREXEL Baltimore Early Phase Clinical Unit; Harbor Hospital

Baltimore, Maryland, United States

Countries

United States

Other Identifiers

BB-TRE-101

Identifier Type: -

Identifier Source: org_study_id