A multi-center clinical study to determine the effects of LJPC-401 on iron in the heart muscle in patients with beta-thalassemia who are treated with routine blood transfusions

Phase 1

• Conditions: 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; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-003372-31-GB
• Lead Sponsor: a Jolla Pharmaceutical Company

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-12-11
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 100

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

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified