Transfusion-dependent beta thalassemia with myocardial iron overload MedDRA version: 20.0 Level: SOC Classification code 10005329 Term: Blood and lymphatic system disorders System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Product Name: LJPC-401
Product Code: LJPC-401
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Hepcidin-25 (human)
CAS N
Sponsors
La Jolla Pharmaceutical Company
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients = 14 years of age with transfusion-dependent beta thalassemia and must have been transfused within 1 month of randomisation. 2. Two separate MRIs that both demonstrate cardiac T2* MRI from 6 to 35 msec (= 6 to = 35 msec) with no more than a 15% difference between the first (MRI 1) and second (MRI 2) reading. MRI 1 and MRI 2 must be at least 3 weeks apart. MRI 1 may be a historical SOC cardiac T2* MRI performed within 12 months of randomisation. 3. Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable, appropriate dose of iron chelation therapy for a minimum of 8 weeks prior to randomisation and expected to remain stable during study participation. 4. Female patients must be of non-childbearing potential, or using a highly effective method of contraception during participation in the study and for 30 days after the last dose of study drug. 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. 5. Female patients of childbearing potential must have a negative serum pregnancy test at Screening, negative pregnancy test predose on the first day study drug is administered, and negative pregnancy tests during the study. 6. Male patients must be surgically sterile, or using a highly effective method of contraception during participation in the study and for 30 days after the last dose of study drug. 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. 7. Patient must be willing and able to provide written informed consent and/or assent. Parent(s) or legal guardian(s) to patients younger than the local age of majority (ie, adult) is willing and able to provide informed consent and assist the patient in complying with all protocol requirements. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 85 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
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, inhaled steroid medications, and nonsteroidal anti-inflammatory drugs) or have a planned surgery (excluding dental surgery or simple dermatologic procedures). 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 and ECG findings, 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, clinically significant arrhythmias, 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. Patients with known hepatitis B, hepatitis C who are being treated for a positive viral load or are noncompliant with their hepatitis medication. 8. Known and active human immunodeficiency virus (HIV) infection. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Efficacy on cardiac iron as measured by cardiac T2* MRI in patients with transfusion-dependent beta thalassemia ; Secondary Objective: •Safety and tolerability in patients with transfusion-dependent beta thalassemia •Proportion of patients who demonstrate an improvement in cardiac T2* MRI •Hemoglobin level •Volume of blood transfused •Hepatic iron levels as measured by T2* MRI •Total serum iron •Immunogenicity (anti-drug antibodies) ;Primary end point(s): The mean change from baseline (Screening) in cardiac iron level as measured by T2* MRI (comparing 26 weeks of SOC to 26 weeks of SOC and LJPC-401 [Group A versus Group B]).;Timepoint(s) of evaluation of this end point: Week 26 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Mean change from baseline in cardiac iron level as measured by cardiac T2* MRI at week 52 2. Mean change in hepatic iron level as measured by cardiac T2* MRI. 3. Mean change in total serum iron 4. Mean change in haemoglobin 5. Change in the volume of blood transfused 6. The proportion of patients with an increase of at least 10% compared to baseline (Screening) in the T2* level as measured by cardiac MRI. 7. The proportion of patients with an increase of at least 20% at Week 52 compared to baseline (Screening) in the T2* level as measured by cardiac MRI ;Timepoint(s) of evaluation of this end point: Week 26 and Week 52 | — |
Countries
Australia, Cyprus, Greece, Italy, Lebanon, Thailand, Turkey, United Kingdom, United States
Contacts
Public ContactRegulatory Affairs
La Jolla Pharmaceutical Company
Outcome results
None listed