NCT03381833 is a Phase 2 clinical trial of LJPC-401 in Beta-Thalassemia, sponsored by La Jolla Pharmaceutical Company.

Who is sponsoring NCT03381833?

NCT03381833 is sponsored by La Jolla Pharmaceutical Company.

What drugs are being tested in NCT03381833?

Interventions in NCT03381833: LJPC-401, LJPC-401.

What condition does NCT03381833 study?

NCT03381833 studies Beta-Thalassemia.

Is NCT03381833 still recruiting?

NCT03381833 is currently terminated. Last updated 29 July 2021.

Last reviewed 2021-07-29 · How we verify

NCT03381833

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

Terminated Phase 2 Last updated 29 July 2021

What this trial tests

Phase 2 trial testing LJPC-401 in Beta-Thalassemia in 84 participants. Terminated before completion.

Timeline

30 November 2017

Primary endpoint
3 December 2019

14 January 2020

Quick facts

Lead sponsor	La Jolla Pharmaceutical Company
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	84
Start date	30 November 2017
Primary completion	3 December 2019
Estimated completion	14 January 2020
Sites	22 locations across Italy, Greece, United Kingdom, Thailand, Australia, Lebanon, United States, Turkey (Türkiye)

Drugs / interventions tested

LJPC-401 — full drug profile →
LJPC-401 — full drug profile →

Conditions studied

Beta-Thalassemia — all drugs for Beta-Thalassemia →

Sponsor

La Jolla Pharmaceutical Company — full company profile →

Who can join

14 and older, any sex, with Beta-Thalassemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with transfusion-dependent beta thalassemia with myocardial iron overload.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Hepcidin Therapeutics.
Katsarou A, Pantopoulos K. · · 2018 · cited 85× · PMID 30469435 · DOI 10.3390/ph11040127
Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation.
Motta I, Bou-Fakhredin R, Taher AT, Cappellini MD. · · 2020 · cited 54× · PMID 32557398 · DOI 10.1007/s40265-020-01341-9
Physiological and pathophysiological mechanisms of hepcidin regulation: clinical implications for iron disorders.
Xu Y, Alfaro-Magallanes VM, Babitt JL. · · 2021 · cited 50× · PMID 33316086 · DOI 10.1111/bjh.17252
Iron Metabolism: An Under Investigated Driver of Renal Pathology in Lupus Nephritis.
Wlazlo E, Mehrad B, Morel L, Scindia Y. · · 2021 · cited 36× · PMID 33912577 · DOI 10.3389/fmed.2021.643686
Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs.
Longo F, Piolatto A, Ferrero GB, Piga A. · · 2021 · cited 28× · PMID 34281283 · DOI 10.3390/ijms22137229
Innovative Treatments for Rare Anemias.
Cappellini MD, Marcon A, Fattizzo B, Motta I. · · 2021 · cited 18× · PMID 34095760 · DOI 10.1097/hs9.0000000000000576
The Role of Iron in Benign and Malignant Hematopoiesis.
Sinha S, Pereira-Reis J, Guerra A, Rivella S, et al · · 2021 · cited 18× · PMID 33231101 · DOI 10.1089/ars.2020.8155
Dyserythropoiesis evaluated by the RED score and hepcidin:ferritin ratio predicts response to erythropoietin in lower-risk myelodysplastic syndromes.
Park S, Kosmider O, Maloisel F, Drenou B, et al · · 2019 · cited 18× · PMID 30287621 · DOI 10.3324/haematol.2018.203158

Verify or expand the search:

Other trials of LJPC-401

Trials testing the same drug.

NCT03395704 — A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis · Phase 2 · completed

Other recruiting trials for Beta-Thalassemia

Currently open trials in the same condition.

NCT06364774 — ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia · Phase 1, PHASE2 · recruiting
NCT06568926 — Adherence of Beta Thalssemia Patients to Oral Chelation Therapy · NA · recruiting
NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
NCT06308159 — An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia · Phase 1, PHASE2 · recruiting
NCT06065189 — Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major · EARLY_PHASE1 · active not recruiting

Other La Jolla Pharmaceutical Company trials

Trials by the same sponsor.

NCT03431077 — A Study of LJPC-501 in Pediatric Patients With Hypotension · Phase 2 · completed
NCT03395704 — A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis · Phase 2 · completed
NCT03245528 — Expanded Access for LJPC-501 · approved for marketing

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03381833 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by La Jolla Pharmaceutical Company
Last refreshed: 29 July 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03381833.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing