Clinical trial NCT03381833: A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
Official 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Beta-Thalassemia
Lead sponsor: La Jolla Pharmaceutical Company
Sponsor class: INDUSTRY
First posted: 2017-12-22
Enrollment: 84
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* Patients ≥ 14 years of age with transfusion-dependent beta thalassemia.
* Patients must have increased iron levels in the heart as measured by magnetic resonance imaging (MRI). Two separate cardiac T2\*MRI from 6 to 35 msec; \<= 15% difference between the two.
* Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose prior to study and expected to remain stable during study.
* Female patients of childbearing potential must not be pregnant, must have negative pregnancy tests, and must use an effective birth control method during the study.
* Male patients must be either surgically sterile or use an effective birth control method during the study.
* Patient must be willing and able to provide written informed consent. Parent of legal guardian to patients younger than age of majority must be willing and able to provide informed consent.

Exclusion Criteria:

* Any significant medical condition or lab abnormality that would prevent the patient from participating in the study.
* Pregnant or lactating women.
* Patients taking an immunosuppressive agent (except topical over-the-counter steroids, inhaled steroid medications, and non-steroidal anti-inflammatory drugs) or have a planned surgery (except dental surgery or simple dermatologic procedures).
* Patients participating in an unapproved investigational clinical trial within 30 days of this study.
* Patients with a disease, disability or condition 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.
* Patients who are unwilling or unable to comply with the study requirements.
* Patients with known hepatitis B or hepatitis C, or being treated for a positive viral load or are noncompliant with hepatitis medications.
* Known and active human immunodeficiency virus (HIV) infection.
* Patients with Child Pugh class C cirrhosis or liver failure.
* Patients with severe congestive heart failure (NYHA Class 4).
* Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
* History of allergic reaction to hepcidin or excipients.
* Contraindication to MRI scanning.

Primary outcomes:
measure: Effect of LJPC-401 on cardiac iron
timeFrame: 26 Weeks
Secondary outcomes:
measure: Effect of LJPC-401 on cardiac iron
timeFrame: 52 Weeks
measure: Effect of LJPC-401 on hepatic iron
timeFrame: 52 Weeks
measure: Effect of LJPC-401 on serum iron
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on hemoglobin
timeFrame: 52 Weeks
measure: Effect of LJPC-401 on volume of blood transfused
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on its potential to elicit an immune response
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on the incidence of treatment-emergent adverse events
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on vital signs-heart rate
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on vital signs-body temperature
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on vital signs-respiratory rate
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on vital signs-blood pressure
timeFrame: 56 Weeks
measure: Effect of LJPC-401 on body weight
timeFrame: 56 Weeks
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Investigative Site
state: California
city: New York
country: United States
facility: Investigative Site
state: New York
city: Camperdown
country: Australia
facility: Investigative Site
state: New South Wales
city: Liverpool
country: Australia
facility: Investigative Site
state: New South Wales
city: Goudí
country: Greece
facility: Investigative Site
state: Attica
city: Thessaloníki
country: Greece
facility: Investigative Site
state: Macedonia
city: Patra
country: Greece
facility: Investigative Site
state: Peloponnese
city: Cagliari
country: Italy
facility: Investigative Site
city: Modena
country: Italy
facility: Investigative Site
city: Napoli
country: Italy
facility: Investigative Site
city: Orbassano
country: Italy
facility: Investigative Site
city: Verona
country: Italy
facility: Investigative Site
city: Beirut
country: Lebanon
facility: Investigative Site
city: Bangkok Noi
country: Thailand
facility: Investigative Site
city: Chiang Mai
country: Thailand
facility: Investigative Site
city: Adana
country: Turkey
facility: Investigative Site
city: Ankara
country: Turkey
facility: Investigative Site
city: Antalya
country: Turkey
facility: Investigative Site
city: Antalya
country: Turkey
facility: Investigative Site
city: İzmir
country: Turkey
facility: Investigative Site
city: London
country: United Kingdom
facility: Investigative Site
state: England
city: London
country: United Kingdom
facility: Investigative Site
Study officials:
References: