Trial Review

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR


Registration number
ACTRN12622000933752
Ethics application status
Approved
Date submitted
17/06/2022
Date registered
30/06/2022
Date last updated
28/01/2024
Date data sharing statement initially provided
30/06/2022
Date results information initially provided
28/01/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase 1, First-in-human, Randomized, Double-blind, Placebo-controlled,
Single and Multiple Ascending Intravenous Dose Study to
Assess the Safety, Tolerability, Pharmacokinetics of VGL101 in Healthy
Adults
Scientific title
A Phase 1, First-in-human, Randomized, Double-blind, Placebo-controlled,
Single and Multiple Ascending Intravenous Dose Study to
Assess the Safety, Tolerability, Pharmacokinetics of VGL101 in Healthy
Adults
Secondary ID [1] 307368 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia 326678 0
Condition category
Condition code
Neurological 323919 323919 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
VGL101 will be administered via intravenous infusion once in the single ascending dose (cohorts 1A-8A) part, and three times (every 28 days) in the multiple ascending dose (cohorts 1B-4B) part. The starting dose of VGL101 will be 1mg/kg and each cohort will increase by 2-3x. The doses selected for the multiple ascending dose cohorts will be selected after a review of the single ascending dose data by a safety review committee. The safety review committee will review safety, laboratory and available pharmacokinetic/pharmacodynamic data.
Intervention code [1] 323796 0
Treatment: Drugs
Comparator / control treatment
Placebo controlled (normal saline solution)
Control group
Placebo

Outcomes
Primary outcome [1] 331728 0
To determine the safety and tolerability of single- and multiple-ascending intravenous doses of VGL101 in healthy adult participants. Safety and tolerability will be assessed by reviewing adverse events, physical and neurological exams, vital signs and available pharmacokinetic/ pharmacodynamic data.

As VGL101 has not been administered to humans, no adverse drug reactions have been identified.
Adverse events will be assessed by clinical examination and self report.

Physical and Neurological exams will be a general check-up by a physician.

Vital signs will be measured by blood pressure measured by sphygmomanometer, pulse will be measured by finger pulse oximeter, temperature will be measured by thermometer and respiratory rate will be measured by counting with a stethoscope.

Pharmacokinetic and Pharmacodynamic will be collected by blood and cerebral spinal fluid.
Timepoint [1] 331728 0
Safety and tolerability will be assessed prior to dosing, and post dose at the following timepoints:
single ascending dose- 0, 1, 2, 4, 8, 24 36 and 48 hours, day 8, 15, 29, 47 and 85
multiple ascending dose- 0, 1, 2, 4, 8, 24 36 and 48 hours, day 8, 15: post second dose 0, 1 and 4 hours: post third dose 0, 1, 2, 4, 8, 24 and 48 hours, day 64, 71, 85, 103 and 141
Each component will be assessed at all timepoints.
Secondary outcome [1] 410883 0
To characterize the single-dose and the multiple dose serum of pharmacokientics (PK). The Tmax, AUC and T1/2 will be assessed.
Timepoint [1] 410883 0
The following timepoints will be assessed for pharmacokinetics:
single ascending dose- 0, 1, 2, 4, 8, 24 36 and 48 hours, day 8, 15, 29, 47 and 85
multiple ascending dose- 0, 1, 2, 4, 8, 24 36 and 48 hours, day 8, 15: post second dose 0, 1 and 4 hours: post third dose 0, 1, 2, 4, 8, 24 and 48 hours, day 64, 71, 85, 103 and 141

Eligibility
Key inclusion criteria
Males and females between 18 to 55 years of age, inclusive, at the Screening Visit.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Participant has any concurrent disease or condition that, in the opinion of the PI, would
make the participant unsuitable for participation in the clinical study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Sealed Envelopes
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a randomisation table created by computer software
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
11/07/2022
Actual
22/07/2022
Date of last participant enrolment
Anticipated
3/10/2022
Actual
13/03/2023
Date of last data collection
Anticipated
31/12/2022
Actual
1/08/2023
Sample size
Target
30
Accrual to date
Final
60
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment outside Australia
Country [1] 24841 0
United States of America
State/province [1] 24841 0

Funding & Sponsors
Funding source category [1] 311652 0
Commercial sector/Industry
Name [1] 311652 0
Vigil Neuroscience, Inc
Country [1] 311652 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Vigil Neuroscience, Inc
Address
One Broadway Suite 07-300
Cambridge MA 02142
Country
United States of America
Secondary sponsor category [1] 313096 0
None
Name [1] 313096 0
Address [1] 313096 0
Country [1] 313096 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 311106 0
Alfred Hospital Ethics Committee
Ethics committee address [1] 311106 0
55 Commercial Rd, Melbourne VIC 3004
Ethics committee country [1] 311106 0
Australia
Date submitted for ethics approval [1] 311106 0
Approval date [1] 311106 0
06/06/2022
Ethics approval number [1] 311106 0

Summary
Brief summary
A Phase 1, First-in-human, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Intravenous Dose Study to Assess the Safety, Tolerability and Pharmacokinetics. VGL101 or placebo will be administered to healthy volunteers to test if VGL101 is safe and can be explored as a treatment for Adult onset leukoencephalopathy with axonal spheroids and pigmented glia.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 119966 0
Dr Spyros Papapetropoulos
Address 119966 0
VIgil Neuroscience
One Broadway Suite 07-300
Cambridge MA 02142
Country 119966 0
United States of America
Phone 119966 0
+18572544445
Fax 119966 0
Email 119966 0
[email protected]
Contact person for public queries
Name 119967 0
Mr Andrew Marsh
Address 119967 0
Vigil Neuroscience
One Broadway Suite 07-300
Cambridge MA 02142
Country 119967 0
United States of America
Phone 119967 0
+17862018066
Fax 119967 0
Email 119967 0
[email protected]
Contact person for scientific queries
Name 119968 0
Dr Spyros Papapetropoulos
Address 119968 0
Vigil Neuroscience
One Broadway Suite 07-300
Cambridge MA 02142
Country 119968 0
United States of America
Phone 119968 0
+18572544445
Fax 119968 0
Email 119968 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.