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

Bioblast Pharma

Status and phase: Completed; Phase 1

Conditions: Healthy Volunteer Subjects

Treatments: Drug: Trehalose for IV Infusion; Drug: Saline 0.9% IV

Study type: Interventional

Funder types: Industry

Identifiers

NCT02725957

BB-TRE-101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 24 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

24 participants in 2 patient groups, including a placebo group

Trehalose 9%

Experimental group

Description:

Single dose administration of Trehalose 9% for IV infusion.

Treatment:

Drug: Trehalose for IV Infusion

Saline 0.9%

Placebo Comparator group

Description:

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

Treatment:

Drug: Saline 0.9% IV