Healthy Volunteer Subjects Clinical Trial
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
| Verified date | November 2016 |
| Source | Bioblast Pharma Ltd. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 24 |
| Est. completion date | August 2016 |
| Est. primary completion date | June 2016 |
| Accepts healthy volunteers | Accepts Healthy Volunteers |
| Gender | All |
| Age group | 18 Years to 55 Years |
| Eligibility |
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 |
| Country | Name | City | State |
|---|---|---|---|
| United States | PAREXEL Baltimore Early Phase Clinical Unit; Harbor Hospital | Baltimore | Maryland |
| Lead Sponsor | Collaborator |
|---|---|
| Bioblast Pharma Ltd. | Parexel |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | Maximum-tolerated dose (MTD) of trehalose administered intravenously (Averse events, vitals signs) | The maximum-tolerated dose of trehalose will be assessed by evaluating the safety and tolerability of each of the escalating trehalose doses | 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 | Pharmacokinetics (PK) of plasma and urine trehalose | To determine the pharmacokinetics (PK) of trehalose following administration of escalating doses of trehalose | Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration. | |
| Secondary | Pharmacokinetics (PK) of serum and urine glucose | To determine the pharmacokinetics (PK) of glucose following administration of escalating doses of trehalose | Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration. |