Healthy Volunteer SubjectsPhase 1NCT02725957
A Study to Establish Safety and Maximum Tolerated Dose of IV Trehalose in Healthy Subjects
A Phase 1 study testing Trehalose for IV Infusion and Saline 0.9% IV in people with Healthy Volunteer Subjects, run by Bioblast Pharma Ltd. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteer Subjects
- What is being tested?
- Trehalose for IV Infusion and Saline 0.9% IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2016to Aug 2016
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy men and women between 18 and 55 years (inclusive) of age
- Body Mass Index (BMI) 19 to 29.9 kg/m2 (inclusive) and weighing at least 55 kg.
- Subjects in general good health in the opinion of the investigator
- Blood pressure and heart rate within normal limits
- 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.
Who cannot join
- Diabetes mellitus type 1 or 2 or HbA1c > 5.6 % at Screening
- History of significant medical disorder
- Any clinically significant abnormality in safety laboratory tests during the Screening period (Day -28 to -1)
- Known contraindication, hypersensitivity and/or allergy to trehalose
- Any acute illness (e.g. acute infection) within 72 hours
- Participation in another clinical trial with drugs received within 3 months prior to dosing
Show all 11Show fewer
- Positive serum pregnancy test determined during the Screening period or currently lactating women
- ECG with clinically significant finding recorded during the Screening period
- Positive HIV, Hepatitis B or Hepatitis C serology at Screening
- Known history of alcohol or drug abuse in the past 5 years
- Positive urinary drug screen determined during the Screening period
Trial locations and contacts
1 site in 1 country.
United States1 site
PAREXEL Baltimore Early Phase Clinical Unit; Harbor Hospital
Baltimore, Maryland, 21225
About this study
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
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.
Read the 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
- Being tested
- Trehalose for IV Infusion, Saline 0.9% IV
- Keywords
- Bioblast
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Trehalose for IV InfusionDrug
Saline 0.9% IVDrug
| Study group | Role | What they receive |
|---|---|---|
| Trehalose 9% | Experimental | Single dose administration of Trehalose 9% for IV infusion. Receives: Drug: Trehalose for IV Infusion |
| Saline 0.9% | Placebo comparator | Single dose administration of 0.9% saline in the same volume and duration as Treatment Arm 1 (9% trehalose) Receives: Drug: Saline 0.9% IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 outcomes (3)
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
Measured: 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
Pharmacokinetics (PK) of plasma and urine trehalose
To determine the pharmacokinetics (PK) of trehalose following administration of escalating doses of trehalose
Measured: Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.
Pharmacokinetics (PK) of serum and urine glucose
To determine the pharmacokinetics (PK) of glucose following administration of escalating doses of trehalose
Measured: Will be assessed on the day of drug administration, before drug administration and up to 12hours following administration.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2016 (estimated)
- Study startMar 2016
- Primary completionJun 2016
- Study completionAug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy Volunteer SubjectsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioblast Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02725957
- Sponsor's protocol ID
- BB-TRE-101
- Status last verified
- Nov 2016
- First submitted
- 15 Mar 2016
- Last updated
- 22 Nov 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02725957
- Acronym / sponsor ID
- BB-TRE-101
- Conditions
- Healthy Volunteer Subjects
- Keywords
- Bioblast
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Bioblast Pharma Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Mar 2016
- Primary completion
- Jun 2016 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 1 Apr 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Trehalose 9% | Experimental | Drug: Trehalose for IV InfusionSingle dose administration of Trehalose 9% for IV infusion. |
| Saline 0.9% | Placebo comparator | Drug: Saline 0.9% IVSingle dose administration of 0.9% saline in the same volume and duration as Treatment Arm 1 (9% trehalose) |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Healthy men and women between 18 and 55 years (inclusive) of age
- Body Mass Index (BMI) 19 to 29.9 kg/m2 (inclusive) and weighing at least 55 kg.
- Subjects in general good health in the opinion of the investigator
- Blood pressure and heart rate within normal limits
- 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.
Key exclusion criteria (11 listed)
- Diabetes mellitus type 1 or 2 or HbA1c > 5.6 % at Screening
- History of significant medical disorder
- Any clinically significant abnormality in safety laboratory tests during the Screening period (Day -28 to -1)
- Known contraindication, hypersensitivity and/or allergy to trehalose
- Any acute illness (e.g. acute infection) within 72 hours
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Trehalose for IV Infusion | Drug | Trehalose 9% |
| Saline 0.9% IV | Drug | Saline 0.9% |
Primary outcome
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.
3 secondary outcomes
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
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.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2016.
- Individual participant dataSharing planned: Undecided
Related trials for Healthy Volunteer Subjects
Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH
RecruitingPhase 1
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
RecruitingPhase 1
A Phase 1 Study of GTX-B001 in Healthy Subjects and Patients With Chronic Inducible Urticaria
Healthy Subjects, Cold Urticaria
RecruitingPhase 1/2
A Study of CYT-1010 in Healthy Volunteers and Participants With Acute Pain After Third Molar Extraction
Acute Postoperative Pain, Dental Pain
RecruitingPhase 1
A Single Ascending Dose, and Food Effect Study of ABBV-7828 in Healthy Adults
Healthy Subjects
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Trehalose for IV Infusion and Saline 0.9% IV in people with Healthy Volunteer Subjects, run by Bioblast Pharma Ltd. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2016.