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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03381833
Ethics application status
Date submitted
12/12/2017
Date registered
22/12/2017

Titles & IDs
Public title
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in 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 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 20 mg per week from week 26 to 52

Treatment: Drugs: LJPC-401
subcutaneous injection, up to 20 mg per week 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
Timepoint [1] 0 0
26 Weeks
Secondary outcome [1] 0 0
Effect of LJPC-401 on cardiac iron
Timepoint [1] 0 0
52 Weeks
Secondary outcome [2] 0 0
Effect of LJPC-401 on hepatic iron
Timepoint [2] 0 0
52 Weeks
Secondary outcome [3] 0 0
Effect of LJPC-401 on serum iron
Timepoint [3] 0 0
56 Weeks
Secondary outcome [4] 0 0
Effect of LJPC-401 on hemoglobin
Timepoint [4] 0 0
52 Weeks
Secondary outcome [5] 0 0
Effect of LJPC-401 on volume of blood transfused
Timepoint [5] 0 0
56 Weeks
Secondary outcome [6] 0 0
Effect of LJPC-401 on its potential to elicit an immune response
Timepoint [6] 0 0
56 Weeks
Secondary outcome [7] 0 0
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Timepoint [7] 0 0
56 Weeks
Secondary outcome [8] 0 0
Effect of LJPC-401 on vital signs-heart rate
Timepoint [8] 0 0
56 Weeks
Secondary outcome [9] 0 0
Effect of LJPC-401 on vital signs-body temperature
Timepoint [9] 0 0
56 Weeks
Secondary outcome [10] 0 0
Effect of LJPC-401 on vital signs-respiratory rate
Timepoint [10] 0 0
56 Weeks
Secondary outcome [11] 0 0
Effect of LJPC-401 on vital signs-blood pressure
Timepoint [11] 0 0
56 Weeks
Secondary outcome [12] 0 0
Effect of LJPC-401 on body weight
Timepoint [12] 0 0
56 Weeks

Eligibility
Key inclusion criteria
* Patients = 14 years of age with transfusion-dependent beta thalassemia.
* Patients must have increased iron levels in the heart as measured by magnetic resonance imaging (MRI). Two separate cardiac T2*MRI from 6 to 35 msec; <= 15% difference between the two.
* 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, must have negative pregnancy tests, 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. Parent of legal guardian to patients younger than age of majority must be willing and able to provide informed consent.
Minimum age
14 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Any significant medical condition or lab abnormality that would prevent the patient from participating in the study.
* Pregnant or lactating women.
* Patients taking an immunosuppressive agent (except topical over-the-counter steroids, inhaled steroid medications, and non-steroidal anti-inflammatory drugs) or have a planned surgery (except dental surgery or simple dermatologic procedures).
* Patients participating in an unapproved investigational clinical trial within 30 days of this study.
* Patients with a disease, disability or condition 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.
* Patients who are unwilling or unable to comply with the study requirements.
* Patients with known hepatitis B or hepatitis C, or being treated for a positive viral load or are noncompliant with hepatitis medications.
* Known and active human immunodeficiency virus (HIV) infection.
* Patients with Child Pugh class C cirrhosis or liver failure.
* Patients with severe congestive heart failure (NYHA Class 4).
* Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
* History of allergic reaction to hepcidin or excipients.
* Contraindication to MRI scanning.

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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/11/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
14/01/2020
Sample size
Target
Accrual to date
Final
84
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Investigative Site - Camperdown
Recruitment hospital [2] 0 0
Investigative Site - Liverpool
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2170 - Liverpool
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
New York
Country [3] 0 0
Greece
State/province [3] 0 0
Attica
Country [4] 0 0
Greece
State/province [4] 0 0
Macedonia
Country [5] 0 0
Greece
State/province [5] 0 0
Peloponnese
Country [6] 0 0
Italy
State/province [6] 0 0
Cagliari
Country [7] 0 0
Italy
State/province [7] 0 0
Modena
Country [8] 0 0
Italy
State/province [8] 0 0
Napoli
Country [9] 0 0
Italy
State/province [9] 0 0
Orbassano
Country [10] 0 0
Italy
State/province [10] 0 0
Verona
Country [11] 0 0
Lebanon
State/province [11] 0 0
Beirut
Country [12] 0 0
Thailand
State/province [12] 0 0
Bangkok Noi
Country [13] 0 0
Thailand
State/province [13] 0 0
Chiang Mai
Country [14] 0 0
Turkey
State/province [14] 0 0
Adana
Country [15] 0 0
Turkey
State/province [15] 0 0
Ankara
Country [16] 0 0
Turkey
State/province [16] 0 0
Antalya
Country [17] 0 0
Turkey
State/province [17] 0 0
Izmir
Country [18] 0 0
United Kingdom
State/province [18] 0 0
England
Country [19] 0 0
United Kingdom
State/province [19] 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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03381833