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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03381833




Registration number
NCT03381833
Ethics application status
Date submitted
12/12/2017
Date registered
22/12/2017
Date last updated
10/01/2019

Titles & IDs
Public title
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Adult Patients With Transfusion-Dependent Beta Thalassemia
Scientific title
A Multi-Center, Randomized, Open-Label, Parallel Group Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Adult Patients With Transfusion-Dependent Beta Thalassemia
Secondary ID [1] 0 0
LJ401-BT01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Beta-Thalassemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
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 - LJPC-401

Active Comparator: Group A - Delayed therapy - standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks

Active Comparator: Group B - Immediate therapy - standard chelation therapy plus LJPC-401 for 52 weeks


Treatment: Drugs: LJPC-401
subcutaneous injection, up to 40 mg weekly from week 26 to 52

Treatment: Drugs: LJPC-401
subcutaneous injection, up to 40 mg weekly from week 1 to 52

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

Outcomes
Primary outcome [1] 0 0
Effect of LJPC-401 on cardiac iron - Change in cardiac T2* magnetic resonance imagining (MRI)
Timepoint [1] 0 0
52 Weeks
Secondary outcome [1] 0 0
Effect of LJPC-401 on blood iron levels - Change in transferrin saturation (TSAT) as measured by blood laboratory tests
Timepoint [1] 0 0
56 Weeks
Secondary outcome [2] 0 0
Effect of LJPC-401 on clinical chemistry laboratory parameters - Change in clinical chemistry parameters as measured by blood laboratory tests
Timepoint [2] 0 0
56 Weeks
Secondary outcome [3] 0 0
Effect of LJPC-401 on hematology laboratory parameters - Change in hematology parameters as measured by blood laboratory tests
Timepoint [3] 0 0
56 Weeks
Secondary outcome [4] 0 0
Effect of LJPC-401 on iron laboratory parameters - Change in iron parameters as measured by blood laboratory tests
Timepoint [4] 0 0
56 Weeks
Secondary outcome [5] 0 0
Effect of LJPC-401 on endocrine laboratory parameters - Change in endocrine parameters as measured by blood laboratory tests
Timepoint [5] 0 0
56 Weeks
Secondary outcome [6] 0 0
Effect of LJPC-401 on urinalysis laboratory parameters - Change in urinalysis parameters as measured by urine laboratory tests
Timepoint [6] 0 0
56 Weeks
Secondary outcome [7] 0 0
Effect of LJPC-401 on its potential to elicit an immune response - Measured by blood laboratory tests and the presence of anti-drug antibodies
Timepoint [7] 0 0
56 Weeks
Secondary outcome [8] 0 0
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Timepoint [8] 0 0
56 Weeks
Secondary outcome [9] 0 0
Effect of LJPC-401 on blood pressure - Change in diastolic, systolic, and combined diastolic and systolic blood pressure
Timepoint [9] 0 0
56 Weeks
Secondary outcome [10] 0 0
Effect of LJPC-401 on heart rate - Change in heart rate (bpm)
Timepoint [10] 0 0
56 Weeks
Secondary outcome [11] 0 0
Effect of LJPC-401 on body weight - Change in body weight (kilograms)
Timepoint [11] 0 0
56 Weeks
Secondary outcome [12] 0 0
Effect of LJPC-401 on body temperature - Change in body temperature (Celsius)
Timepoint [12] 0 0
56 Weeks
Secondary outcome [13] 0 0
Effect of LJPC-401 on general health - Change in physical examinations (by body system)
Timepoint [13] 0 0
56 Weeks

Eligibility
Key inclusion criteria
- Patients = 18 years of age with transfusion-dependent beta thalassemia.

- Patients must have increased iron blood levels as measured by transferrin saturation
(TSAT)

- Patients must have increased iron levels in the heart as measured by magnetic
resonance imaging (MRI)

- Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a
stable dose prior to study and expected to remain stable during study.

- Female patients of childbearing potential must not be pregnant and must use an
effective birth control method during the study.

- Male patients must be either surgically sterile or use an effective birth control
method during the study.

- 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
- Patients must not have a medical condition that would interfere with the conduct of
the clinical study.

- Pregnant or lactating women.

- Patients taking an immunosuppressive agent (except topical over-the-counter steroids)
or have a planned surgery (except dental surgery or simple dermatologic procedures).

- Patients participating in an investigational clinical trial within 30 days of this
study.

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

- History of hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.

- History of allergic reaction to hepcidin or excipients.

- Unable to undergo MRI.

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
Open (masking not used)
Who is / are masked / blinded?



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

Recruitment
Recruitment status
Recruiting
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,SA
Recruitment hospital [1] 0 0
Investigative Site - Camperdown
Recruitment hospital [2] 0 0
Investigative Site - Liverpool
Recruitment hospital [3] 0 0
Investigative Site - Adelaide
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2170 - Liverpool
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
Greece
State/province [4] 0 0
Attica
Country [5] 0 0
Greece
State/province [5] 0 0
Macedonia
Country [6] 0 0
Greece
State/province [6] 0 0
Peloponnese
Country [7] 0 0
Italy
State/province [7] 0 0
Genova
Country [8] 0 0
Italy
State/province [8] 0 0
Milano
Country [9] 0 0
Italy
State/province [9] 0 0
Modena
Country [10] 0 0
Italy
State/province [10] 0 0
Napoli
Country [11] 0 0
Italy
State/province [11] 0 0
Orbassano
Country [12] 0 0
Italy
State/province [12] 0 0
Palermo
Country [13] 0 0
Lebanon
State/province [13] 0 0
Beirut
Country [14] 0 0
Thailand
State/province [14] 0 0
Bangkok Noi
Country [15] 0 0
Thailand
State/province [15] 0 0
Chiang Mai
Country [16] 0 0
Turkey
State/province [16] 0 0
Adana
Country [17] 0 0
Turkey
State/province [17] 0 0
Ankara
Country [18] 0 0
Turkey
State/province [18] 0 0
Antalya
Country [19] 0 0
Turkey
State/province [19] 0 0
Istanbul
Country [20] 0 0
Turkey
State/province [20] 0 0
Izmir
Country [21] 0 0
United Kingdom
State/province [21] 0 0
England
Country [22] 0 0
United Kingdom
State/province [22] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
La Jolla Pharmaceutical Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The
primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human
hepcidin) on iron levels in adult patients with transfusion-dependent beta thalassemia with
myocardial iron overload.
Trial website
https://clinicaltrials.gov/show/NCT03381833
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
Brian J Byrnes
Address 0 0
Country 0 0
Phone 0 0
+1-858-256-7913
Fax 0 0
Email 0 0
bbyrnes@ljpc.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03381833