Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12616000132448
Ethics application status
Approved
Date submitted
26/01/2016
Date registered
4/02/2016
Date last updated
4/05/2018
Type of registration
Retrospectively registered

Titles & IDs
Public title
A Phase I study of LJPC-401 in healthy adult volunteers
Scientific title
A Phase 1, Placebo-Controlled Study to Determine the Safety, Tolerability and Pharmacokinetics of Escalating Subcutaneous Doses of LJPC-401 in Healthy Adult Volunteers
Secondary ID [1] 288417 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemochromatosis 297432 0
Thalassemia 297476 0
Sickle Cell Disease 297477 0
Myelodysplasia 297478 0
Condition category
Condition code
Blood 297616 297616 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Single dose of LJPC-401 delivered subcutaneously. Planned ascending dose cohorts at 5mg, 18mg, 36, and 72mg. All doses will be administered on-site by study staff. A Data Monitoring Committee (DMC) comprised of the Principal Investigator (PI), the Sponsor’s medical representatives, and others as applicable, will meet periodically to review all treatment-emergent adverse events (TEAEs), and to make dose-escalation decisions.
Intervention code [1] 293731 0
Treatment: Drugs
Comparator / control treatment
Normal saline
Control group
Placebo

Outcomes
Primary outcome [1] 297160 0
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, ECG).
Timepoint [1] 297160 0
Day 1: -30- 0 minutes pre-dose, 15-30 minutes, 2 hours, 4 hours, and 8 hours post dose; Day 2, 3, and 8: once daily
Primary outcome [2] 297161 0
Changes in clinical outcomes: clinical laboratory evaluations including serum iron levels, vital signs, ECGs, physical examinations
Timepoint [2] 297161 0
Clinical laboratory evaluations once daily at Day -1 or Day 1 (pre-dose/baseline), Day 2, Day 3, Day 8

Total serum iron at Day 1 pre-dose/baseline (-30 to 0 minutes pre-dose), 2 hours, 4 hours, and 8 hours post-dose; once daily at Day 2, Day 3, and Day 8. Additional serum iron testing at Day 1 pre-dose and Day 8, including ferritin, transferrin, total iron binding capacity (TIBC), and unsaturated iron binding capacity (UIBC).

Vital signs once at Day -1 or Day 1 (pre-dose/baseline); 15-30 minutes, 2 hours, 4 hours, and 8 hours post-dose; once daily at Day 2, Day 3, and Day 8

ECG once at Day -1 or Day 1 (pre-dose/baseline); 4 hours post-dose; once daily at Day 3, and Day 8 (if abnormal at Day 3 only)

Physical examination once at Day -1 or Day 1 (pre-dose/baseline); 2 hours, 4 hours, and 8 hours post-dose; once daily at Day 2, Day 2, and Day 8
Primary outcome [3] 297201 0
Immunogenicity testing, assessed by serum assay
Timepoint [3] 297201 0
Once daily at Day -1 or Day 1 pre-dose/baseline, Day 8, and Day 22.
Secondary outcome [1] 320202 0
Serum iron parameters: total serum iron, ferritin, transferrin, TIBC, and UIBC
Timepoint [1] 320202 0
Total serum iron at Day 1 pre-dose/baseline (-30 to 0 minutes pre-dose), 2 hours, 4 hours, and 8 hours post-dose; once daily at Day 2, Day 3, and Day 8. Additional serum iron testing at Day 1 pre-dose and Day 8, including ferritin, TIBC, and UIBC.
Secondary outcome [2] 320329 0
Pharmacokinetics assessed by blood sample: t1/2, Tmax, Cmax, Kel, AUClast, AUC0-8
Timepoint [2] 320329 0
Day 1 pre-dose/baseline (-30 to 0 minutes pre-dose); 15-30 minutes, 2 hours, 4 hours, and 8 hours post-dose; once daily at Day 2, Day 3, and Day 8

Eligibility
Key inclusion criteria
Normal, healthy volunteers, negative drug and alcohol tests, BMI 18-32 inclusive
Minimum age
18 Years
Maximum age
65 Years
Sex
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
2/02/2016
Date of last participant enrolment
Anticipated
31/03/2016
Actual
14/03/2016
Date of last data collection
Anticipated
Actual
4/04/2016
Sample size
Target
32
Accrual to date
Final
32
Recruitment in Australia
Recruitment state(s)
WA

Funding & Sponsors
Funding source category [1] 292766 0
Commercial sector/Industry
Name [1] 292766 0
La Jolla Pharmaceutical Company
Country [1] 292766 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
La Jolla Pharmaceutical Company
Address
10182 Telesis Court, Suite 600
San Diego, CA 92121
Country
United States of America
Secondary sponsor category [1] 291500 0
None
Name [1] 291500 0
None
Address [1] 291500 0
None
Country [1] 291500 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 294256 0
Bellberry Human Research Ethics Committee
Ethics committee address [1] 294256 0
Ethics committee country [1] 294256 0
Australia
Date submitted for ethics approval [1] 294256 0
25/11/2015
Approval date [1] 294256 0
06/01/2016
Ethics approval number [1] 294256 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 63050 0
Dr Sam Salman
Address 63050 0
Linear Clinical Research
Level 1, B Block, Hospital Avenue
Nedlands, WA 6009
Country 63050 0
Australia
Phone 63050 0
+61 (8) 6382 5100
Fax 63050 0
Email 63050 0
[email protected]
Contact person for public queries
Name 63051 0
Brian Byrnes
Address 63051 0
La Jolla Pharmaceutical Company
10182 Telesis Court, Suite 600
San Diego, CA 92121
Country 63051 0
United States of America
Phone 63051 0
+1-858-207-4264
Fax 63051 0
Email 63051 0
[email protected]
Contact person for scientific queries
Name 63052 0
Brian Byrnes
Address 63052 0
La Jolla Pharmaceutical Company
10182 Telesis Court, Suite 600
San Diego, CA 92121
Country 63052 0
United States of America
Phone 63052 0
+1-858-207-4264
Fax 63052 0
Email 63052 0
[email protected]

No information has been provided regarding IPD availability


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.