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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03395704




Registration number
NCT03395704
Ethics application status
Date submitted
12/12/2017
Date registered
10/01/2018
Date last updated
4/03/2020

Titles & IDs
Public title
A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Scientific title
A Phase 2, Multi-Center, Randomized, Placebo Controlled, Single-Blind Study With LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Secondary ID [1] 0 0
LJ401-HH01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Hemochromatosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LJPC-401
Treatment: Drugs - Placebo

Active Comparator: LJPC-401 - LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial

Placebo Comparator: Placebo - 0.9% Sodium Chloride Injection, USP, or equivalent


Treatment: Drugs: LJPC-401
LJPC-401 subcutaneous injection, up to 20 mg weekly for 16 weeks. The minimum weekly dose will be 5 mg and the maximum weekly dose of LJPC-401 will be 20 mg.

Treatment: Drugs: Placebo
0.9% Sodium Chloride Injection, USP, or equivalent

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Effect of LJPC-401 versus placebo on blood iron levels - Change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Timepoint [1] 0 0
16 Weeks
Secondary outcome [1] 0 0
Effect of LJPC-401 versus placebo on incidence of phlebotomy
Timepoint [1] 0 0
16 Weeks
Secondary outcome [2] 0 0
Effect of LJPC-401 versus placebo on blood iron levels - Change in serum ferritin as measured by blood laboratory tests
Timepoint [2] 0 0
16 Weeks
Secondary outcome [3] 0 0
Effect of LJPC-401 versus placebo on the incidence of treatment-emergent adverse events
Timepoint [3] 0 0
20 Weeks
Secondary outcome [4] 0 0
Effect of LJPC-401 versus placebo on clinical chemistry laboratory parameters - Safety measure for clinical chemistry parameters including serum creatinine, uric acid, alkaline phosphatase, blood urea nitrogen, albumin, bilirubin (total), phosphate, bicarbonate, lactate dehydrogenase, glucose, calcium, magnesium, chloride, sodium, potassium, alanine aminotransferase, aspartate aminotransferase, and total protein will be assessed. In addition, serum pregnancy test will be assessed.
Timepoint [4] 0 0
20 Weeks
Secondary outcome [5] 0 0
Effect of LJPC-401 versus placebo on hematology laboratory parameters - Safety measure for laboratory hematology parameters including platelet count, red blood cell count, absolute white blood cell count, hemoglobin, hematocrit, reticulocyte count, red cell distribution width, mean corpuscular hemoglobin, mean corpuscular volume, and mean corpuscular hemoglobin concentration will be assessed.
Timepoint [5] 0 0
20 Weeks
Secondary outcome [6] 0 0
Effect of LJPC-401 versus placebo on blood iron parameters - Exploratory measure of serum iron, serum transferrin, total iron-binding capacity, unsaturation iron-binding capacity will be assessed to evaluate iron parameters.
Timepoint [6] 0 0
20 Weeks
Secondary outcome [7] 0 0
Effect of LJPC-401 versus placebo on endocrine laboratory parameters - Exploratory measure for endocrine laboratory parameters, including thyroid-stimulating hormone, thyroxine (T4), and triiodothyronine (T3) will be assessed to evaluate changes in thyroid function.
Timepoint [7] 0 0
16 Weeks
Secondary outcome [8] 0 0
Effect of LJPC-401 versus placebo on glucose levels - Exploratory measure for change in hemoglobin A1c will be assessed to evaluate blood sugar levels.
Timepoint [8] 0 0
16 Weeks
Secondary outcome [9] 0 0
Effect of LJPC-401 versus placebo on urinalysis laboratory parameters - Safety measure for urinalysis laboratory parameters including specific gravity, bilirubin, glucose, pH, protein, nitrite, blood, ketones will be assessed. In addition, urine pregnancy test will be assessed at Week 1 (Day 1) to reconfirm eligibility predose and at again at Week 20 for additional safety assessment.
Timepoint [9] 0 0
20 Weeks
Secondary outcome [10] 0 0
Effect of LJPC-401 versus placebo on blood pressure - Safety measure for change in blood pressure.
Timepoint [10] 0 0
20 Weeks
Secondary outcome [11] 0 0
Effect of LJPC-401 versus placebo on heart rate - Safety measure for change in heart rate (bpm).
Timepoint [11] 0 0
20 Weeks
Secondary outcome [12] 0 0
Effect of LJPC-401 versus placebo on body weight - Safety measure for change in body weight (kilograms).
Timepoint [12] 0 0
20 Weeks
Secondary outcome [13] 0 0
Effect of LJPC-401 versus placebo on body temperature - Safety measure for change in body temperature (Celsius).
Timepoint [13] 0 0
20 Weeks
Secondary outcome [14] 0 0
Effect of LJPC-401 versus placebo on general health - Safety measure for change in physical examination (by body system).
Timepoint [14] 0 0
20 Weeks
Secondary outcome [15] 0 0
Effect of LJPC-401 versus placebo on electrical activity of the heart - Safety measure for change in the electrical activity of the heart over a period of time using electrodes placed on the skin and recorded by electrocardiogram (ECG).
Timepoint [15] 0 0
20 Weeks
Secondary outcome [16] 0 0
Effect of LJPC-401 versus placebo on its potential to elicit an immune response - Safety measure of blood samples collected to assess the presence of anti-drug antibodies.
Timepoint [16] 0 0
20 Weeks
Secondary outcome [17] 0 0
Effect of LJPC-401 versus placebo on quality of life - Exploratory measure for change in quality of life as measured by the Short Form Health Survey version 2.0 questionnaire. The scale measures physical functioning; role-physical, bodily pain; general health; vitality; social functioning; role-emotional; mental health and reported health transition. The responses vary depending on the question: Range 1-6 (none to very severe); range 1-5 (all of the time to none of the time or definitely true to definitely false); range 1-4 not at all to quite a bit; range 1-3 (yes, limited a lot to no, not limited at all). All scoring will be tallied by a validated central system (Optum) and provided to the sponsor at the end of the study. Scoring will be analyzed as a change from baseline to end of treatment (Week 16).
Timepoint [17] 0 0
16 Weeks
Secondary outcome [18] 0 0
Effect of LJPC-401 versus placebo on quality of life - Exploratory measure for change in quality of life as measured by the Western Ontario and McMaster Universities Osteoarthritis Index questionnaire. The scale ranges from 1-5 (none to extreme in regards to pain and difficulty during day to day activity. Responses will be analyzed as a change from baseline to end of treatment (Week 16).
Timepoint [18] 0 0
16 Weeks
Secondary outcome [19] 0 0
Exploratory measure for change in quality of life - Exploratory measure for change in quality of life as measured by the Modified Dreisler indicies (FIOHA-Functional Index for Hand OsteoArthritis) questionnaire. The scale ranges from 1-4 (Possible without difficulty to impossible in regards to activities that use hand movement. The results are totaled and scores will be analyzed as a change from baseline to end of treatment (Week 16).
Timepoint [19] 0 0
16 Weeks

Eligibility
Key inclusion criteria
1. Patients with clinical diagnosis of hereditary hemochromatosis

2. Patients who are prescribed therapeutic phlebotomy for treatment of hereditary
hemochromatosis

3. Patients with serum ferritin and TSAT levels above treatment guidelines

4. Female patients of child bearing potential must have a negative pregnancy test and
must be using a highly effective method of contraception during participation in the
study, and for 30 days after the last dose of study drug

5. Males must be surgically sterile (vasectomy), or using a highly effective method of
contraception during participation in the study, and for 30 days after the last dose
of study drug

6. Patient must be willing and able to provide written informed consent
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Patients receiving iron chelation therapy within 7 days prior to the first dose of
study drug

2. Patients initiating phlebotomy treatments less than 3 months prior to the first dose
of study drug

3. Pregnant or lactating women

4. Patients taking an immunosuppressive agent without prior Sponsor approval

5. Patients participating in an unapproved investigational drug or investigational
therapeutic device within 30 days of study drug

6. Patients who are unwilling or unable to comply with the study protocol requirements

7. Patients with type 1 or poorly controlled type 2 diabetes

8. Patients with a concomitant disease, disability or condition, including laboratory
abnormality and ECG findings, which may interfere with the conduct of the study, or
which would, in the opinion of the Investigator, pose an unacceptable risk to the
patient in this study, including, but not limited to, clinically significant
arrhythmias, alcohol dependency or abuse, drug dependency or abuse, or psychiatric
disease

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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
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)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Investigative Site - Liverpool
Recruitment hospital [2] 0 0
Investigative Site - Westmead
Recruitment hospital [3] 0 0
Investigative Site - Brisbane
Recruitment hospital [4] 0 0
Investigative Site - Herston
Recruitment hospital [5] 0 0
Investigative Site - Melbourne
Recruitment hospital [6] 0 0
Investigative Site - Murdoch
Recruitment postcode(s) [1] 0 0
2170 - Liverpool
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4120 - Brisbane
Recruitment postcode(s) [4] 0 0
4029 - Herston
Recruitment postcode(s) [5] 0 0
3004 - Melbourne
Recruitment postcode(s) [6] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Mississippi
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
France
State/province [10] 0 0
Bondy
Country [11] 0 0
France
State/province [11] 0 0
Orléans
Country [12] 0 0
France
State/province [12] 0 0
Pessac
Country [13] 0 0
France
State/province [13] 0 0
Rennes Cedex 9
Country [14] 0 0
United Kingdom
State/province [14] 0 0
England

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
La Jolla Pharmaceutical Company
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
PRA Health Sciences
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
This study is a Phase 2 multicenter, randomized, placebo controlled, single-blind study. The
primary objective of the study is to compare the effect of weekly dosing of LJPC-401
(synthetic human hepcidin) versus placebo on transferrin saturation (TSAT) in an adult
hereditary hemochromatosis patient population.
Trial website
https://clinicaltrials.gov/show/NCT03395704
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications