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Trial registered on ANZCTR


Registration number
ACTRN12620001156976
Ethics application status
Approved
Date submitted
4/09/2020
Date registered
4/11/2020
Date last updated
4/11/2020
Date data sharing statement initially provided
4/11/2020
Date results information initially provided
4/11/2020
Type of registration
Retrospectively registered

Titles & IDs
Public title
A study to evaluate the safety, tolerability, pharmacokinetics (PK – how your body uses the study drug) and pharmacodynamics (PD – how the study drug carries out its actions on the body) profile of Inclacumab following a single intravenous infusion in healthy participants
Scientific title
An Open-label, Single-ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Inclacumab Following Intravenous Infusion in Healthy Participants
Secondary ID [1] 302024 0
GBT2104-111
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Sickle Cell Disease 318601 0
Condition category
Condition code
Blood 316620 316620 0 0
Haematological diseases
Human Genetics and Inherited Disorders 317255 317255 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Inclacumab is a recombinant human monoclonal antibody (huMAb) being developed for the treatment of sickle cell disease (SCD).

15 eligible participants will be enrolled in 2 dose cohorts and receive a single intravenous dose of 20 mg/kg or 40 mg/kg of inclacumab over a period of 1 hour. Doses will be administered by the study nurse.
Intervention code [1] 318324 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 324746 0
To assess the safety and tolerability of inclacumab after a single dose at different dose levels in healthy participants. Safety and tolerability will be assessed through review of ongoing safety assessments including clinical laboratory testing, physical examination, ECG and vitals as well as recording of any side effects..
Timepoint [1] 324746 0
Safety and tolerability will be assessed during the inpatient period following dosing (daily assessments for up to 4 days post-dose) and during the outpatient follow-up period, up to Day 203 (14 outpatient visits, 1-3 weeks apart)
Secondary outcome [1] 385627 0
To characterize the plasma pharmacokinetics (PK) of inclacumab in healthy participants including but not limited to, Cmax, AUC, and T1/2
Timepoint [1] 385627 0
Blood samples for PK will be collected at the following timepoint:
Day 1: pre-dose, end of infusion and 2, 6, & 12 hrs post end of infusion
Day 2, 3, 4, 8, 15, 22, 29, 43, 57, 71, 85, 99, 120, 141, 162, 183, 203
Secondary outcome [2] 387475 0
To characterize the effect of inclacumab on pharmacodynamics (PD) in healthy participants including including P-selectin inhibition, platelet-leukocyte aggregation, and plasma soluble P-selectin
Timepoint [2] 387475 0
Blood samples for PD will be collected at the following timepoint:
Day 1: pre-dose, end of infusion and 2, 6, & 12 hrs post end of infusion
Day 2, 3, 4, 8, 15, 29, 57, 71, 85, 99, 120, 141, 162, 183, 203

Eligibility
Key inclusion criteria
1. Healthy male or female participants between 18 and 65 years of age, inclusive.
2. Participants in good health as determined by the Investigator’s review of medical history,
physical examination, vital sign measurements, 12-lead ECG, and clinical laboratory
tests.
3. Body weight equal to or greater than 50 kg (male) or equal to or greater than 45 kg (female), and equal to or less than 110 kg and a body mass index (BMI) between 18.0 and 30.0 kg/m2, inclusive.
4. Ability to comply with all the requirements of the study.
Minimum age
18 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1.. Use of prescription medications or herbal remedies within 14 days of the Day -1 (with the
exceptions of hormonal contraceptives and other medications at the discretion of the
Investigator and/or Sponsor).
2. Use of over-the-counter medication within 7 days of the Day -1. Medications such as
paracetamol or non-steroidal anti-inflammatories and routinely taken dietary supplements
are allowed at the discretion of the Investigator and/or Sponsor.
3. Clinically significant abnormalities in laboratory results including:
a. Absolute neutrophil count (ANC) < 1.5 × 10^9/L;
b. Platelet count < 150 × 10^9/L;
c. Creatinine clearance (calculated by Cockcroft-Gault) < 60 mL/min;
d. Direct conjugated bilirubin >1.5 × upper limit of normal (ULN);
e. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >1.5 × ULN;
f. Hemoglobin < 12 g/dL.
4. History of alcohol abuse or use of drugs of abuse in the 12 months prior to the Day -1.
Excess alcohol is defined as more than 14 drinks/week for women and 21 drinks/week for
men. A drink is defined as 360 mL of beer, 150 mL of wine, or 45 mL of spirits.
5. Females who are pregnant or breastfeeding.
6. Prior exposure to inclacumab or crizanlizumab.
7. Received an investigational drug within 30 days or 5 half-lives, whichever is longer, of
the Day -1, or is currently participating in another trial of an investigational or marketed
drug (or medical device).
8. Vaccination with live vaccine (with the exception of seasonal flu vaccine) within 6 weeks of Day -1 or planning for vaccination while participating in the study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Single study drug dose will be administered on Day 1 only
Phase
Phase 1
Type of endpoint(s)
Safety
Statistical methods / analysis
15 healthy volunteers between 18-65 years of age will be enrolled in this study.
Due to the exploratory nature of this study, no formal power or sample size calculations were used to determine cohort size. A sufficient number of healthy volunteers will be screened to ensure at least 6 participants are enrolled and dosed in each cohort.

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 17252 0
Linear Clinical Research - Nedlands
Recruitment postcode(s) [1] 30964 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 306447 0
Commercial sector/Industry
Name [1] 306447 0
Global Blood Therapeutics, Inc.
Address [1] 306447 0
181 Oyster Point Blvd.
South San Francisco
CA 94080
Country [1] 306447 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Linear Clinical Research Ltd
Address
Level 1, B Block
Hospital Ave
Nedlands WA 6009
Country
Australia
Secondary sponsor category [1] 306963 0
Commercial sector/Industry
Name [1] 306963 0
Global Blood Therapeutics, Inc.
Address [1] 306963 0
181 Oyster Point Blvd.
South San Francisco
CA 94080
Country [1] 306963 0
United States of America

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 306653 0
Bellberry Limited
Ethics committee address [1] 306653 0
123 Glen Osmond Rd
Eastwood SA 5063
Ethics committee country [1] 306653 0
Australia
Date submitted for ethics approval [1] 306653 0
15/07/2020
Approval date [1] 306653 0
25/08/2020
Ethics approval number [1] 306653 0
2020-07-658

Summary
Brief summary
The purpose of this study is to assess the safety and tolerability of inclacumab, as well as the pharmacokinetics (PK – how your body uses the study drug) and pharmacodynamics (PD – how the study drug carries out its actions on the body).

Inclacumab will be tested in 15 healthy participants aged 18 to 65 years. Participants will be administered a single doses of inclacumab as an intravenous infusion over 1 hour. Participants will then be followed up for 203 days for safety and PK/PD assessments
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 104566 0
Dr Andrew Redfern
Address 104566 0
Linear Clinical Research
Level 1, B Block, Hospital Ave
Nedlands, WA 6009
Country 104566 0
Australia
Phone 104566 0
+61 433 078 719
Fax 104566 0
Email 104566 0
aredfern@linear.org.au
Contact person for public queries
Name 104567 0
Ms Margot Hottmann
Address 104567 0
Global Blood Therapeutics, Inc.
181 Oyster Point Blvd.
South San Francisco, CA 94080
Country 104567 0
United States of America
Phone 104567 0
+1 650 822 8728
Fax 104567 0
Email 104567 0
mhottmann@gbt.com
Contact person for scientific queries
Name 104568 0
Dr Dan Cooper
Address 104568 0
Global Blood Therapeutics, Inc.
181 Oyster Point Blvd.
South San Francisco, CA 94080
Country 104568 0
United States of America
Phone 104568 0
+1 650 888 9896
Fax 104568 0
Email 104568 0
dcooper@gbt.com

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
IPD will not be available.
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
No
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary