A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

July 28, 2021 updated by: La Jolla Pharmaceutical Company

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

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 patients with transfusion-dependent beta thalassemia with myocardial iron overload.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
84

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Investigative Site
      • Liverpool, New South Wales, Australia, 2170
        • Investigative Site
  • Greece
    • Attica
      • Goudí, Attica, Greece, 115 27
        • Investigative Site
    • Macedonia
      • Thessaloníki, Macedonia, Greece, 54642
        • Investigative Site
    • Peloponnese
      • Patra, Peloponnese, Greece, 26504
        • Investigative Site
  • Italy
      • Cagliari, Italy, 09121
        • Investigative Site
      • Modena, Italy, 41124
        • Investigative Site
      • Napoli, Italy, 80138
        • Investigative Site
      • Orbassano, Italy, 10043
        • Investigative Site
      • Verona, Italy, 37134
        • Investigative Site
  • Lebanon
      • Beirut, Lebanon, 1107 2020
        • Investigative Site
  • Thailand
      • Bangkok Noi, Thailand, 10700
        • Investigative Site
      • Chiang Mai, Thailand, 50200
        • Investigative Site
  • Turkey
      • Adana, Turkey, 01330
        • Investigative Site
      • Ankara, Turkey, 06100
        • Investigative Site
      • Antalya, Turkey, 07059
        • Investigative Site
      • Antalya, Turkey, 07160
        • Investigative Site
      • İzmir, Turkey, 35100
        • Investigative Site
  • United Kingdom
      • London, United Kingdom, N19 5NF
        • Investigative Site
    • England
      • London, England, United Kingdom, E1 1BB
        • Investigative Site
  • United States
    • California
      • Oakland, California, United States, 94609
        • Investigative Site
    • New York
      • New York, New York, United States, 10021
        • Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Group A - Delayed therapy
standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
Drug: LJPC-401
subcutaneous injection, up to 20 mg per week from week 26 to 52
Other Names:
  • synthetic human hepcidin
Drug: LJPC-401
subcutaneous injection, up to 20 mg per week from week 1 to 52
Other Names:
  • synthetic human hepcidin
Active Comparator: Group B - Immediate therapy
standard chelation therapy plus LJPC-401 for 52 weeks
Drug: LJPC-401
subcutaneous injection, up to 20 mg per week from week 26 to 52
Other Names:
  • synthetic human hepcidin
Drug: LJPC-401
subcutaneous injection, up to 20 mg per week from week 1 to 52
Other Names:
  • synthetic human hepcidin

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Effect of LJPC-401 on cardiac iron
Time Frame: 26 Weeks
Change in cardiac T2* magnetic resonance imaging (MRI)
26 Weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Effect of LJPC-401 on cardiac iron
Time Frame: 52 Weeks
Change in cardiac T2*MRI
52 Weeks
Effect of LJPC-401 on hepatic iron
Time Frame: 52 Weeks
Change in hepatic T2*MRI
52 Weeks
Effect of LJPC-401 on serum iron
Time Frame: 56 Weeks
Mean change in serum iron
56 Weeks
Effect of LJPC-401 on hemoglobin
Time Frame: 52 Weeks
Mean change in hemoglobin
52 Weeks
Effect of LJPC-401 on volume of blood transfused
Time Frame: 56 Weeks
Change in transfused blood volume
56 Weeks
Effect of LJPC-401 on its potential to elicit an immune response
Time Frame: 56 Weeks
Measured by blood laboratory tests and the presence of anti-drug antibodies
56 Weeks
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Time Frame: 56 Weeks
56 Weeks
Effect of LJPC-401 on vital signs-heart rate
Time Frame: 56 Weeks
56 Weeks
Effect of LJPC-401 on vital signs-body temperature
Time Frame: 56 Weeks
56 Weeks
Effect of LJPC-401 on vital signs-respiratory rate
Time Frame: 56 Weeks
56 Weeks
Effect of LJPC-401 on vital signs-blood pressure
Time Frame: 56 Weeks
56 Weeks
Effect of LJPC-401 on body weight
Time Frame: 56 Weeks
Change in body weight (kilograms)
56 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
La Jolla Pharmaceutical Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 30, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 3, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 14, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 12, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 20, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 22, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 29, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 28, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • LJ401-BT01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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