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

NCT02725957 Phase 1 COMPLETED

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.

Details

Lead sponsor	Bioblast Pharma Ltd.
Phase	Phase 1
Status	COMPLETED
Enrolment	24
Start date	2016-03
Completion	2016-08

Conditions

• Healthy Volunteer Subjects

Interventions

• Trehalose for IV Infusion
• Saline 0.9% IV

Primary outcomes

• Safety and tolerability of escalating doses of intravenously administered trehalose (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
  Safety will be assessed by the incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities.

Countries

United States