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 |
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Recruitment Status : Unknown
Verified January 2019 by La Jolla Pharmaceutical Company.
Recruitment status was: Recruiting
First Posted : December 22, 2017
Last Update Posted : January 10, 2019
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Sponsor:
La Jolla Pharmaceutical Company
Information provided by (Responsible Party):
La Jolla Pharmaceutical Company
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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 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 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Beta thalassemia
| Arm | Intervention/treatment |
|---|---|
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Active Comparator: Group A - Delayed therapy
standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
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Drug: LJPC-401
subcutaneous injection, up to 40 mg weekly from week 26 to 52
Other Name: synthetic human hepcidin |
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Active Comparator: Group B - Immediate therapy
standard chelation therapy plus LJPC-401 for 52 weeks
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Drug: LJPC-401
subcutaneous injection, up to 40 mg weekly from week 1 to 52
Other Name: synthetic human hepcidin |
Primary Outcome Measures :
- Effect of LJPC-401 on cardiac iron [ Time Frame: 52 Weeks ]Change in cardiac T2* magnetic resonance imagining (MRI)
Secondary Outcome Measures :
- 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)
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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.
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
| Contact: Brian J Byrnes | +1-858-256-7913 | bbyrnes@ljpc.com |
Locations
| United States, California | |
| Investigative Site | Recruiting |
| Los Angeles, California, United States, 90027 | |
| Investigative Site | Recruiting |
| Oakland, California, United States, 94609 | |
| Investigational Site | Recruiting |
| San Diego, California, United States, 91942 | |
| United States, Illinois | |
| Investigative Site | Recruiting |
| Chicago, Illinois, United States, 60611-2605 | |
| United States, New York | |
| Investigative Site | Recruiting |
| New Hyde Park, New York, United States, 11040 | |
| Investigative Site | Recruiting |
| New York, New York, United States, 10021 | |
| Australia, New South Wales | |
| Investigative Site | Recruiting |
| Camperdown, New South Wales, Australia, 2050 | |
| Investigative Site | Recruiting |
| Liverpool, New South Wales, Australia, 2170 | |
| Australia, South Australia | |
| Investigative Site | Recruiting |
| Adelaide, South Australia, Australia, 5000 | |
| Greece | |
| Investigative Site | Recruiting |
| Goudí, Attica, Greece, 115 27 | |
| Investigative Site | Recruiting |
| Thessaloníki, Macedonia, Greece, 54642 | |
| Investigative Site | Recruiting |
| Patra, Peloponnese, Greece, 26504 | |
| Italy | |
| Investigative Site | Recruiting |
| Genova, Italy, 16128 | |
| Investigative Site | Recruiting |
| Milano, Italy, 20122 | |
| Investigative Site | Recruiting |
| Modena, Italy, 41124 | |
| Investigative Site | Recruiting |
| Napoli, Italy, 80131 | |
| Investigative Site | Recruiting |
| Napoli, Italy, 80138 | |
| Investigative Site | Recruiting |
| Orbassano, Italy, 10043 | |
| Investigative Site | Recruiting |
| Palermo, Italy, 90146 | |
| Lebanon | |
| Investigative Site | Recruiting |
| Beirut, Lebanon, 1107 2020 | |
| Thailand | |
| Investigative Site | Recruiting |
| Bangkok Noi, Thailand, 10700 | |
| Investigative Site | Recruiting |
| Chiang Mai, Thailand, 50200 | |
| Turkey | |
| Investigative Site | Recruiting |
| Adana, Turkey, 01330 | |
| Investigative Site | Recruiting |
| Ankara, Turkey, 06100 | |
| Investigative Site | Recruiting |
| Antalya, Turkey, 07059 | |
| Investigative Site | Recruiting |
| Antalya, Turkey, 07160 | |
| Investigative Site | Recruiting |
| Istanbul, Turkey, 34093 | |
| Investigative Site | Recruiting |
| İzmir, Turkey, 35100 | |
| United Kingdom | |
| Investigative Site | Recruiting |
| London, England, United Kingdom, E1 1BB | |
| Investigative Site | Recruiting |
| London, United Kingdom, N19 5NF | |
| Investigative Site | Recruiting |
| London, United Kingdom, NW1 2BU | |
Sponsors and Collaborators
La Jolla Pharmaceutical Company
| Responsible Party: | La Jolla Pharmaceutical Company |
| ClinicalTrials.gov Identifier: | NCT03381833 |
| 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 |
| 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 |