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
129 Glen Osmond Rd
Eastwood, SA 3222
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
A 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. Bellberry Human Research Ethics Committee approval has been received.
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
Mr 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
Mr 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.