Hepcidin

Hepcidin trials – TRANSCEND

 

Update: 27 September 2019

No update available

 

Update: 30 July 2019

No update available.

 

Update: 30 May 2019

No update available.

 

Update: 01 February 2019

Hepcidin is a hormone that performs various functions related to iron absorption and distribution in the body.  Thalassaemia patients have low hepcidin levels due to iron overload.

Abnormally low hepcidin levels caused by genetic mutations or secondary pathology can be replaced by a hepcidin mimetic to restore iron balance in the body. Increasing hepcidin levels can reduce iron overload and improve anaemia.

PTG-300 is an injectable hepcidin mimetic peptide, that has been granted Orphan Drug designation in the U.S. and EU and has received Fast Track designation by the FDA for development in the treatment of β-thalassaemia.

A Phase 1 study in healthy volunteers showed that administration of PTG-300 was well tolerated and demonstrated a dose-related and sustained reduction in serum iron levels.

As such a global Phase 2 study (TRANSCEND) with an open label and multiple-ascending dose design has commenced to evaluate the safety and preliminary efficacy of PTG-300 in approximately 84 adolescent and adult patients with non-transfusion-dependent or transfusion-dependent β-thalassemia.

Preliminary results are to be announced in the second half of 2019.

Sources:

http://investors.protagonist-inc.com/news-releases/news-release-details/protagonist-therapeutics-initiates-phase-2-trial-novel-hepcidin

https://www.clinicaltrials.gov/ct2/show/NCT03802201

 

Hepcidin trials – LJPC-401

 

 

Update: 27 September 2019

No update available

 

Update: 30 July 2019

No update available.

 

Update: 30 May 2019

No update available.

 

Update: 29 March 2019

No update available.

 

Update: 01 February 2019

No update available.

 

Update: 20 December 2018

No update available.

 

Update: 02 October 2018

Hepcidin is a hormone that performs various functions related to iron absorption and distribution in the body.  Thalassaemia patients have low hepcidin levels due to iron overload.

A Phase 2 multi-centre, randomized, open-label, parallel-group study is underway at 22 sites across the world.

The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in adult patients with transfusion-dependent beta thalassemia with myocardial iron overload.

The purpose is to increase the level of hepcidin in the body in order to help it absorb less iron from the gut and transfer this iron to various organs. Hepcidin also plays a role in the regulation of RBC production. Therefore, the trial will also determine whether increasing hepcidin can increase haemoglobin in thalassaemia patients.

Preliminary results are expected to be announced in 2019.

 

Sources:

https://clinicaltrials.gov/ct2/show/NCT03381833

http://www.bloodjournal.org/content/early/2018/03/08/blood-2017-11-737411?sso-checked=true