LJPC-401 for Iron Overload in Adults with Hereditary Hemochromatosis
Research type
Research Study
Full title
A Phase 2, Multi-center, Randomized, Placebo Controlled, Double-Blind Study with LJPC-401 for the Treatment of Iron Overload in Adult Patients with Hereditary Hemochromatosis
IRAS ID
237518
Contact name
Steven Masson
Contact email
Sponsor organisation
La Jolla Pharmaceutical Company
Eudract number
2017-003598-33
Clinicaltrials.gov Identifier
118188, IND Number
Duration of Study in the UK
0 years, 8 months, 20 days
Research summary
This is a phase 2, multi-centre, randomised, placebo-controlled, double-blind study of LJPC-401 for the treatment of iron overload in adults with Hereditary Haemochromatosis (HH).
HH is an autosomal (a chromosome which isn’t a sex chromosome) recessive (two copies required for effects to manifest) disorder that causes excessive amounts of iron to be absorbed and stored in the body. The excess can overload and eventually damage tissues and organs. It is the most common hereditary (inherited) disorder observed in Caucasians. Mutations in several genes can cause HH. Approximately 80% of HH cases are caused by high iron (HFE) mutations.
The primary treatment for HH is phlebotomy (removal of blood). Phlebotomy is inconvenient and tedious, and compliance decreases over time. Other treatments for iron overload, including chelation therapy (binding and removal of iron) have significant and undesirable side effects.
The iron-regulating effects of hepcidin (a naturally occurring protein made by the body that controls iron), which is reduced in patients with HH, make it a potentially beneficial therapeutic treatment of HH. LJPC-401, a synthetic equivalent of naturally circulating hepcidin, is expected to reduce iron overload in individuals with HH. The safety profile to date indicates that LJPC-401 is well tolerated. In addition, the single, weekly subcutaneous (SC [just below the skin]) injection(s) is expected to be less burdensome than phlebotomy, and the effects of a single injection last up to 1 week. Up to 4 injections may be required dependent on the dose, which will be adjusted in relation to transferrin saturation (TSAT [iron levels in blood]) results.
Approximately 60 male and female participants aged 18 or older will participate in this study over a 20 week period. Participants will be randomly assigned 1:1 to receive either study medication or placebo, according to TSAT results and phlebotomy frequency over the last year.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
18/NE/0035
Date of REC Opinion
3 Apr 2018
REC opinion
Further Information Favourable Opinion