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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02725957
Recruitment Status : Completed
First Posted : April 1, 2016
Last Update Posted : November 22, 2016
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
Bioblast Pharma Ltd.

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.


Condition or disease Intervention/treatment Phase
Healthy Volunteer Subjects Drug: Trehalose for IV Infusion Drug: Saline 0.9% IV Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
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
Study Start Date : March 2016
Actual Primary Completion Date : June 2016
Actual Study Completion Date : August 2016

Arm Intervention/treatment
Experimental: Trehalose 9%
Single dose administration of Trehalose 9% for IV infusion.
Drug: Trehalose for IV Infusion
Placebo Comparator: Saline 0.9%
Single dose administration of 0.9% saline in the same volume and duration as Treatment Arm 1 (9% trehalose)
Drug: Saline 0.9% IV



Primary Outcome Measures :
  1. 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 ]
    Safety will be assessed by the incidence of adverse events and serious adverse events, including clinically significant laboratory abnormalities.


Secondary Outcome Measures :
  1. 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 ]
    The maximum-tolerated dose of trehalose will be assessed by evaluating the safety and tolerability of each of the escalating trehalose doses

  2. 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. ]
    To determine the pharmacokinetics (PK) of trehalose following administration of escalating doses of trehalose

  3. 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. ]
    To determine the pharmacokinetics (PK) of glucose following administration of escalating doses of trehalose



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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02725957


Locations
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United States, Maryland
PAREXEL Baltimore Early Phase Clinical Unit; Harbor Hospital
Baltimore, Maryland, United States, 21225
Sponsors and Collaborators
Bioblast Pharma Ltd.
Parexel

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Responsible Party: Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier: NCT02725957    
Other Study ID Numbers: BB-TRE-101
First Posted: April 1, 2016    Key Record Dates
Last Update Posted: November 22, 2016
Last Verified: November 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Bioblast Pharma Ltd.:
Bioblast