EUCTR2017-003372-31-ITActive, not recruitingPhase 1
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 - A multi-center clinical study to determine the effects of LJPC-401 on iron in the heart muscle in pa
A JOLLA PHARMACEUTICAL COMPANY0 sites100 target enrollmentStarted: January 7, 2021Last updated:
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 100
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Patients = 18 years of age with transfusion-dependent beta thalassemia and must have been transfused within 1 month of randomization.
- •2. Patients must have a TSAT = 50% at Screening.
- •3. Two separate cardiac MRIs should be greater than 3 weeks but no more than 3 months apart that both demonstrate cardiac T2* MRI of 6 to 35 (msec) with no more than a 10% difference between the first and second reading.
- •4. Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose of maximally tolerated iron chelation therapy for a minimum of 8 weeks prior to study and expected to remain stable during study.
- •5. Female patients must be of non-childbearing potential, or using a highly effective method of contraception during participation in the study. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly. These include: surgical sterility (vasectomy, bilateral tubal ligation, or hysterectomy); hormonal intrauterine device (IUD); hormonal intrauterine system (IUS), implants, or injections; consistent use of an approved oral contraceptive pill (combined progestin/estrogen pill or progestin-only pill); combined intravaginal or transdermal method; and abstinence.
- •6. Female patients of childbearing potential must have a negative serum pregnancy test at Screening, and negative urine pregnancy test at Week 1 (ie, prior to initial dosing of study drug).
- •7. Male patients must be surgically sterile, or using a highly effective method of contraception during participation in the study. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly.
- •8. Patient must be willing and able to provide written informed consent.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 90
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 10
Exclusion Criteria
- •1. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.
- •2. Pregnant or lactating women.
- •3. Patients taking an immunosuppressive agent (excluding topical over-the-counter steroids) or have a planned surgery (excluding dental surgery or simple dermatologic procedures) during participation in this study or within 2 weeks prior to randomization.
- •4. Patients participating in an unapproved investigational drug or investigational therapeutic device study within 30 days prior to randomization, ie, there must be at least 30 days in between the last dose on a prior study and randomization on this trial.
- •5. Patients with a concomitant disease, disability or condition, including laboratory abnormality, 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 in this study, including, but not limited to, alcohol dependency or abuse, drug dependency or abuse, or psychiatric disease.
- •6. Patients who are unwilling or unable to comply with the study protocol requirements.
- •7. Known hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.
- •8. Patients with Child Pugh class C cirrhosis or liver failure.
- •9. Patients with severe congestive heart failure (New York Heart Association [NYHA] = Class 4).
- •10. Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
- •11. History of allergic reaction to hepcidin or excipients.
- •12. Contraindication to MRI scanning.
Investigators
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