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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

Tracking Information
First Submitted Date  ICMJE December 12, 2017
First Posted Date  ICMJE December 22, 2017
Last Update Posted Date January 10, 2019
Actual Study Start Date  ICMJE November 30, 2017
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 20, 2017)
Effect of LJPC-401 on cardiac iron [ Time Frame: 52 Weeks ]
Change in cardiac T2* magnetic resonance imagining (MRI)
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03381833 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 21, 2017)
  • Effect of LJPC-401 on blood iron levels [ Time Frame: 56 Weeks ]
    Change in transferrin saturation (TSAT) as measured by blood laboratory tests
  • Effect of LJPC-401 on clinical chemistry laboratory parameters [ Time Frame: 56 Weeks ]
    Change in clinical chemistry parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on hematology laboratory parameters [ Time Frame: 56 Weeks ]
    Change in hematology parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on iron laboratory parameters [ Time Frame: 56 Weeks ]
    Change in iron parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on endocrine laboratory parameters [ Time Frame: 56 Weeks ]
    Change in endocrine parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on urinalysis laboratory parameters [ Time Frame: 56 Weeks ]
    Change in urinalysis parameters as measured by urine laboratory tests
  • 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
  • Effect of LJPC-401 on the incidence of treatment-emergent adverse events [ Time Frame: 56 Weeks ]
  • Effect of LJPC-401 on blood pressure [ Time Frame: 56 Weeks ]
    Change in diastolic, systolic, and combined diastolic and systolic blood pressure
  • Effect of LJPC-401 on heart rate [ Time Frame: 56 Weeks ]
    Change in heart rate (bpm)
  • Effect of LJPC-401 on body weight [ Time Frame: 56 Weeks ]
    Change in body weight (kilograms)
  • Effect of LJPC-401 on body temperature [ Time Frame: 56 Weeks ]
    Change in body temperature (Celsius)
  • Effect of LJPC-401 on general health [ Time Frame: 56 Weeks ]
    Change in physical examinations (by body system)
Original Secondary Outcome Measures  ICMJE
 (submitted: December 20, 2017)
  • Effect of LJPC-401 on blood iron levels [ Time Frame: 56 Weeks ]
    Change in transferrin saturation (TSAT) as measured by blood laboratory tests
  • Effect of LJPC-401 on clinical chemistry laboratory parameters [ Time Frame: 56 Weeks ]
    Change in clinical chemistry parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on hematology laboratory parameters [ Time Frame: 56 Weeks ]
    Change in hematology parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on iron laboratory parameters [ Time Frame: 56 Weeks ]
    Change in iron parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on endocrine laboratory parameters [ Time Frame: 56 Weeks ]
    Change in endocrine parameters as measured by blood laboratory tests
  • Effect of LJPC-401 on urinalysis laboratory parameters [ Time Frame: 56 Weeks ]
    Change in urinalysis parameters as measured by urine laboratory tests
  • 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
  • Effect of LJPC-401 on the incidence of treatment-emergent adverse events [ Time Frame: 56 Weeks ]
  • Effect of LJPC-401 on blood pressure [ Time Frame: 56 Weeks ]
    Change in blood pressure
  • Effect of LJPC-401 on heart rate [ Time Frame: 56 Weeks ]
    Change in heart rate (bpm)
  • Effect of LJPC-401 on body weight [ Time Frame: 56 Weeks ]
    Change in body weight (kilograms)
  • Effect of LJPC-401 on body temperature [ Time Frame: 56 Weeks ]
    Change in body temperature (Celsius)
  • Effect of LJPC-401 on general health [ Time Frame: 56 Weeks ]
    Change in physical examinations (by body system)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Adult Patients With Transfusion-Dependent Beta Thalassemia
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Beta-Thalassemia
Intervention  ICMJE
  • Drug: LJPC-401
    subcutaneous injection, up to 40 mg weekly from week 26 to 52
    Other Name: synthetic human hepcidin
  • Drug: LJPC-401
    subcutaneous injection, up to 40 mg weekly from week 1 to 52
    Other Name: synthetic human hepcidin
Study Arms  ICMJE
  • Active Comparator: Group A - Delayed therapy
    standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
    Intervention: Drug: LJPC-401
  • Active Comparator: Group B - Immediate therapy
    standard chelation therapy plus LJPC-401 for 52 weeks
    Intervention: Drug: LJPC-401
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 20, 2017)
100
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2020
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Brian J Byrnes +1-858-256-7913 bbyrnes@ljpc.com
Listed Location Countries  ICMJE Australia,   Greece,   Italy,   Lebanon,   Thailand,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03381833
Other Study ID Numbers  ICMJE LJ401-BT01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party La Jolla Pharmaceutical Company
Study Sponsor  ICMJE La Jolla Pharmaceutical Company
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account La Jolla Pharmaceutical Company
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP