LJPC-401 for iron overload in adults with TDT
Research type
Research Study
Full 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’
IRAS ID
232937
Contact name
Farrukh Shah
Contact email
Sponsor organisation
La Jolla Pharmaceutical Company
Eudract number
2017-003372-31
Clinicaltrials.gov Identifier
118188, IND Number
Duration of Study in the UK
2 years, 1 months, 29 days
Research summary
Patients with beta-thalassaemia have an increased amount of iron in their bodies (iron overload) as a result of blood transfusions and increased absorption of iron from food. Once the body’s ability to store iron is exceeded, iron levels in the blood increases and iron is stored in vital organs like the liver, heart, spleen and pancreas. Increased iron in these organs can cause significant damage and even result in death.
The disorder affects all genders but is more prevalent in certain ethnicities and age groups. 20 people die per year causing thalassaemia to be listed as a “rare disease”. There have been 4,000 hospitalised cases in England in 2002 and 9,233 consultant episodes for thalassaemia.
LJPC-401 is a synthetic human hepcidin. Hepcidin is a naturally occurring protein made by the body that controls iron. It lowers blood iron levels and decreases iron storage in organs. Hepcidin levels are lower than normal in patients with beta-thalassaemia. Synthetic human hepcidin is a chemically made version of the naturally occurring human hepcidin that helps to regulate how iron is distributed in the body.
Approximately 100 participants who have been diagnosed with beta–thalassaemia and increased iron in the heart muscle will be enrolled in this research study. The study will be conducted in up to 40 different clinical research sites or centres in Europe, the United States, Australia and Asia.
Patients will be assigned to Group A or Group B. Both Group A and Group B participants will continue to receive their standard therapy, but each group receives LJPC-401 for different periods of time: Group A – Delayed Treatment (26 weeks of standard therapy, followed by 26 weeks of LJPC-401 treatment and standard therapy); or Group B – Immediate Treatment (52 weeks) of LJPC-401 treatment and standard therapy).The length of involvement would be up to approximately 62 weeks (up to 6 weeks of screening, 52 weeks of treatment, and 4 weeks for safety follow-up after the study treatment period).
REC name
London - Brent Research Ethics Committee
REC reference
18/LO/0079
Date of REC Opinion
20 Mar 2018
REC opinion
Further Information Favourable Opinion