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

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ClinicalTrials.gov Identifier: NCT03381833
Recruitment Status : Recruiting
First Posted : December 22, 2017
Last Update Posted : January 10, 2019
Sponsor:
Information provided by (Responsible Party):
La Jolla Pharmaceutical Company

Study Description
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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.

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Drug: LJPC-401 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : November 30, 2017
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : May 2020

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Arms and Interventions
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Arm Intervention/treatment
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 40 mg weekly from week 26 to 52
Other Name: 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 40 mg weekly from week 1 to 52
Other Name: synthetic human hepcidin


Outcome Measures
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Primary Outcome Measures :
  1. Effect of LJPC-401 on cardiac iron [ Time Frame: 52 Weeks ]
    Change in cardiac T2* magnetic resonance imagining (MRI)


Secondary Outcome Measures :
  1. Effect of LJPC-401 on blood iron levels [ Time Frame: 56 Weeks ]
    Change in transferrin saturation (TSAT) as measured by blood laboratory tests

  2. Effect of LJPC-401 on clinical chemistry laboratory parameters [ Time Frame: 56 Weeks ]
    Change in clinical chemistry parameters as measured by blood laboratory tests

  3. Effect of LJPC-401 on hematology laboratory parameters [ Time Frame: 56 Weeks ]
    Change in hematology parameters as measured by blood laboratory tests

  4. Effect of LJPC-401 on iron laboratory parameters [ Time Frame: 56 Weeks ]
    Change in iron parameters as measured by blood laboratory tests

  5. Effect of LJPC-401 on endocrine laboratory parameters [ Time Frame: 56 Weeks ]
    Change in endocrine parameters as measured by blood laboratory tests

  6. Effect of LJPC-401 on urinalysis laboratory parameters [ Time Frame: 56 Weeks ]
    Change in urinalysis parameters as measured by urine laboratory tests

  7. 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

  8. Effect of LJPC-401 on the incidence of treatment-emergent adverse events [ Time Frame: 56 Weeks ]
  9. Effect of LJPC-401 on blood pressure [ Time Frame: 56 Weeks ]
    Change in diastolic, systolic, and combined diastolic and systolic blood pressure

  10. Effect of LJPC-401 on heart rate [ Time Frame: 56 Weeks ]
    Change in heart rate (bpm)

  11. Effect of LJPC-401 on body weight [ Time Frame: 56 Weeks ]
    Change in body weight (kilograms)

  12. Effect of LJPC-401 on body temperature [ Time Frame: 56 Weeks ]
    Change in body temperature (Celsius)

  13. Effect of LJPC-401 on general health [ Time Frame: 56 Weeks ]
    Change in physical examinations (by body system)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03381833


Contacts
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Contact: Brian J Byrnes +1-858-256-7913 bbyrnes@ljpc.com

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Sponsors and Collaborators
La Jolla Pharmaceutical Company
More Information
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Responsible Party: La Jolla Pharmaceutical Company
ClinicalTrials.gov Identifier: NCT03381833     History of Changes
Other Study ID Numbers: LJ401-BT01
First Posted: December 22, 2017    Key Record Dates
Last Update Posted: January 10, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
 Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Hepcidins
Anti-Infective Agents