Beta-Thalassemia Clinical Trial
Official 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
Verified date | July 2021 |
Source | La Jolla Pharmaceutical Company |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 84 |
Est. completion date | January 14, 2020 |
Est. primary completion date | December 3, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 14 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Australia | Investigative Site | Camperdown | New South Wales |
Australia | Investigative Site | Liverpool | New South Wales |
Greece | Investigative Site | Goudí | Attica |
Greece | Investigative Site | Patra | Peloponnese |
Greece | Investigative Site | Thessaloníki | Macedonia |
Italy | Investigative Site | Cagliari | |
Italy | Investigative Site | Modena | |
Italy | Investigative Site | Napoli | |
Italy | Investigative Site | Orbassano | |
Italy | Investigative Site | Verona | |
Lebanon | Investigative Site | Beirut | |
Thailand | Investigative Site | Bangkok Noi | |
Thailand | Investigative Site | Chiang Mai | |
Turkey | Investigative Site | Adana | |
Turkey | Investigative Site | Ankara | |
Turkey | Investigative Site | Antalya | |
Turkey | Investigative Site | Antalya | |
Turkey | Investigative Site | Izmir | |
United Kingdom | Investigative Site | London | England |
United Kingdom | Investigative Site | London | |
United States | Investigative Site | New York | New York |
United States | Investigative Site | Oakland | California |
Lead Sponsor | Collaborator |
---|---|
La Jolla Pharmaceutical Company |
United States, Australia, Greece, Italy, Lebanon, Thailand, Turkey, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Effect of LJPC-401 on cardiac iron | Change in cardiac T2* magnetic resonance imaging (MRI) | 26 Weeks | |
Secondary | Effect of LJPC-401 on cardiac iron | Change in cardiac T2*MRI | 52 Weeks | |
Secondary | Effect of LJPC-401 on hepatic iron | Change in hepatic T2*MRI | 52 Weeks | |
Secondary | Effect of LJPC-401 on serum iron | Mean change in serum iron | 56 Weeks | |
Secondary | Effect of LJPC-401 on hemoglobin | Mean change in hemoglobin | 52 Weeks | |
Secondary | Effect of LJPC-401 on volume of blood transfused | Change in transfused blood volume | 56 Weeks | |
Secondary | Effect of LJPC-401 on its potential to elicit an immune response | Measured by blood laboratory tests and the presence of anti-drug antibodies | 56 Weeks | |
Secondary | Effect of LJPC-401 on the incidence of treatment-emergent adverse events | 56 Weeks | ||
Secondary | Effect of LJPC-401 on vital signs-heart rate | 56 Weeks | ||
Secondary | Effect of LJPC-401 on vital signs-body temperature | 56 Weeks | ||
Secondary | Effect of LJPC-401 on vital signs-respiratory rate | 56 Weeks | ||
Secondary | Effect of LJPC-401 on vital signs-blood pressure | 56 Weeks | ||
Secondary | Effect of LJPC-401 on body weight | Change in body weight (kilograms) | 56 Weeks |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00069862 -
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
|
Phase 1/Phase 2 | |
Completed |
NCT00733811 -
Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
|
Phase 4 | |
Completed |
NCT05506358 -
Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada
|
N/A | |
Withdrawn |
NCT04938635 -
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
|
Phase 2 | |
Active, not recruiting |
NCT03655678 -
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia
|
Phase 2/Phase 3 | |
Completed |
NCT06239389 -
Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
|
Phase 2 | |
Recruiting |
NCT05635266 -
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
|
||
Completed |
NCT03271541 -
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
|
Phase 2 | |
Terminated |
NCT02274233 -
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
|
Phase 1 | |
Completed |
NCT01206075 -
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
|
N/A | |
Recruiting |
NCT05567458 -
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
|
Phase 2 | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Completed |
NCT03961828 -
Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus
|
Phase 4 | |
Recruiting |
NCT06065189 -
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
|
Early Phase 1 | |
Recruiting |
NCT04143724 -
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
|
Phase 2 | |
Completed |
NCT02268409 -
ACE-536 Extension Study - Beta Thalassemia
|
Phase 2 | |
Not yet recruiting |
NCT01996683 -
Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
|
N/A | |
Active, not recruiting |
NCT01016093 -
Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients
|
Phase 2/Phase 3 | |
Completed |
NCT01039636 -
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
|
Phase 1 | |
Withdrawn |
NCT01927913 -
Treatment of Iron Overload Requiring Chelation Therapy
|
Phase 2 |