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Trial registered on ANZCTR


Registration number
ACTRN12617000689370
Ethics application status
Approved
Date submitted
10/05/2017
Date registered
15/05/2017
Date last updated
4/05/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase 1, Randomized, Double-Blind, Two-Arm, Placebo-Controlled, Single and Multiple Dose Escalation Study to Assess the Safety and Tolerability of LJPC-401 in Healthy Adults
Scientific title
A Phase 1, Randomized, Double-Blind, Two-Arm, Placebo-Controlled, Single and Multiple Dose Escalation Study to Assess the Safety and Tolerability of LJPC-401 in Healthy Adults
Secondary ID [1] 291776 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
hereditary haemochromatosis 303002 0
thalassemia 303136 0
sickle cell disease 303137 0
myelodysplasia (MDS) 303138 0
Condition category
Condition code
Blood 302466 302466 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Single and multiple doses of LJPC-401 delivered subcutaneously by site staff. Ascending-dose cohorts at 4 mg, 10 mg, and 20 mg per dose in single-dose cohorts. One dose cohort at 10 mg per dose administered twice weekly for two weeks in a multiple-dose cohort (Days 1, 4, 8 and 11). A Data Monitoring Committee (DMC) comprised of the Principal Investigator (PI), the Sponsor’s medical representatives, and other site, sponsor, and contract research organization representatives, as applicable, to include at least one independent member, met prior to the start of the multiple-dose cohort to review all treatment-emergent adverse events (TEAEs), and to make study-related decisions (i.e. escalation, dose level, etc.). The DMC met after the last subject in the single-dose cohorts had completed their Day 8 visit. The proposed doses had been tested in two previous clinical studies and were below the maximum tested dose of 30 mg. The DMC was also responsible for making study decisions regarding stopping, interrupting subject recruitment, or expanding enrollment in the study or cohort based upon review of the study data.
Intervention code [1] 297887 0
Treatment: Drugs
Comparator / control treatment
Normal saline
Control group
Placebo

Outcomes
Primary outcome [1] 301882 0
Safety and tolerability assessed by treatment emergent adverse events, as reported by subjects and/or in response to an open question from study personnel or revealed by observation, physical examination or other diagnostic procedures (i.e., vital signs, clinical laboratory evaluations, ECGs).
Timepoint [1] 301882 0
Single-dose cohorts:
Day 1: pre-dose, 30 minutes, 2 hours, 4 hours, 8 hours and 12 hours post dose; Day 2, Day 3, Day 8 and Day 22: once daily
Multiple-dose cohort:
Day 1 and Day 11: pre-dose, 30 minutes, 2 hours, 4 hours, 8 hours and 12 hours post dose; Day 4 and Day 8: pre-dose, 2 hours, 4 hours, and 8 hours post dose. Day 2, Day 3, Day 5, Day 6, Day 7, Day 9, Day 10, Day 12, Day 13, Day 18, and Day 32: once daily
Primary outcome [2] 301883 0
Changes in clinical outcomes: clinical laboratory evaluations including serum iron parameters, vital signs, ECGs, physical examinations
Timepoint [2] 301883 0
Single-dose cohorts:
Day 1: pre-dose, 30 minutes, 2 hours, 4 hours, 8 hours and 12 hours post dose; Day 2, Day 3, Day 8 and Day 22: once daily
Multiple-dose cohort:
Day 1 and Day 11: pre-dose, 30 minutes, 2 hours, 4 hours, 8 hours and 12 hours post dose; Day 4 and Day 8: pre-dose, 2 hours, 4 hours, and 8 hours post dose. Day 2, Day 3, Day 5, Day 6, Day 7, Day 9, Day 10, Day 12, Day 13, Day 18, and Day 32: once daily
Primary outcome [3] 301884 0
Immunogenicity testing, assessed by serum assay
Timepoint [3] 301884 0
Single-dose cohorts:
Day 1 predose, Day 8, Day 22
Multiple-dose cohort:
Day 1 predose, Day 8 predose, Day 12, Day 18, Day 32
Secondary outcome [1] 334189 0
Serum iron parameters: total serum iron, ferritin, transferrin, TIBC, UIBC, and TSAT %
Timepoint [1] 334189 0
Single-dose cohorts:
Screening, Day 1: predose, 2, 4 , 8 and 12 hours postdose, Day 2, Day 3, and Day 8
Multiple-dose cohort:
Screening, Day 1: predose, 2, 4, 8 and 12 hours postdose, Day 2, Day 3, Day 4: predose, 2, 4, and 8 hours postdose, Day 5, Day 8: predose, 2, 4, and 8 hours postdose, Day 9, Day 10, Day 11: predose, 2, 4, 8 and 12 hours postdose, Day 12, Day 13 and Day 18.
Secondary outcome [2] 334190 0
Pharmacokinetics assessed by blood sample: t1/2, Tmax, Cmax, Kel, AUClast, AUC 0 to infinity
Timepoint [2] 334190 0
Single-dose cohorts:
Screening, Day -1 , Day 1 predose, 30 minutes postdose, 2 hours postdose, 4 hours postdose, 8 hours postdose, 12 hours postdose, Day 2; Day 8; and Day 22 Multiple-dose cohort:
Screening; Day -1; Day 1 predose, 30 minutes postdose, 2 hours postdose, 4 hours postdose, 8 hours postdose, 12 hours postdose; Day 2; Day 4 and Day 8: predose, 2 hours postdose; Day 11 predose, 30 minutes postdose, 2 hours postdose, 4 hours postdose, 8 hours postdose, 12 hours postdose; Day 12; Day 13; Day 18; and Day 32.

Eligibility
Key inclusion criteria
Healthy volunteers, negative drug and alcohol tests, BMI 18-32 inclusive
Minimum age
18 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Blood pressure less than 90/60 or greater than 160/100, limited alcohol and tobacco use, infection or other serious underlying medical condition

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
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

Funding & Sponsors
Funding source category [1] 296281 0
Commercial sector/Industry
Name [1] 296281 0
La Jolla Pharmaceutical Australia Pty Ltd, an affiliate of La Jolla Pharmaceutical Company
Address [1] 296281 0
58 Gipps Street, Collingwood, VIC 3066

Country [1] 296281 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
La Jolla Pharmaceutical Australia Pty Ltd, an affiliate of La Jolla Pharmaceutical Company
Address
58 Gipps Street, Collingwood, VIC 3066
Country
Australia
Secondary sponsor category [1] 295204 0
None
Name [1] 295204 0
None
Address [1] 295204 0
None
Country [1] 295204 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 297514 0
Bellberry Human Research Ethics Committee
Ethics committee address [1] 297514 0
129 Glen Osmond Rd
Eastwood, SA 3222
Ethics committee country [1] 297514 0
Australia
Date submitted for ethics approval [1] 297514 0
29/03/2017
Approval date [1] 297514 0
28/04/2017
Ethics approval number [1] 297514 0

Summary
Brief summary
A single- and multiple-dose Phase I study of LJPC-401 in healthy adult volunteers. LJPC-401 is being developed to treat conditions characterized by iron overload, such as hemochromatosis and beta thalassemia.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 74282 0
Dr Sam Salman
Address 74282 0
Linear Clinical Research
Level 1, B Block, Hospital Avenue
Nedlands, WA 6009
Country 74282 0
Australia
Phone 74282 0
+61 8 6382 5100
Fax 74282 0
Email 74282 0
SSalman@linear.org.au
Contact person for public queries
Name 74283 0
Mr Brian Byrnes
Address 74283 0
La Jolla Pharmaceutical Company 4550 Towne Centre Court San Diego, CA 92121
Country 74283 0
United States of America
Phone 74283 0
+1-858-207-4264
Fax 74283 0
Email 74283 0
bbyrnes@ljpc.com
Contact person for scientific queries
Name 74284 0
Mr Brian Byrnes
Address 74284 0
La Jolla Pharmaceutical Company 4550 Towne Centre Court San Diego, CA 92121
Country 74284 0
United States of America
Phone 74284 0
+1-858-207-4264
Fax 74284 0
Email 74284 0
bbyrnes@ljpc.com

No data has been provided for results reporting
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary