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

Sponsor

La Jolla Pharmaceutical Company (Industry)

Overall Status

Terminated

CT.gov ID

NCT03381833

Collaborator

(none)

Enrollment

Locations

Arms

25.5

Actual Duration (Months)

3.8

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Beta-Thalassemia	Drug: LJPC-401 Drug: LJPC-401	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

84 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Nov 30, 2017

Actual Primary Completion Date :

Dec 3, 2019

Actual Study Completion Date :

Jan 14, 2020

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Group A - Delayed therapy standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks	Drug: LJPC-401 subcutaneous injection, up to 20 mg per week from week 26 to 52 Other Names: synthetic human hepcidin
Active Comparator: Group B - Immediate therapy standard chelation therapy plus LJPC-401 for 52 weeks	Drug: LJPC-401 subcutaneous injection, up to 20 mg per week from week 1 to 52 Other Names: synthetic human hepcidin

Arm

Intervention/Treatment

Active Comparator: Group A - Delayed therapy

standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks

Drug: LJPC-401

subcutaneous injection, up to 20 mg per week from week 26 to 52

Other Names:

synthetic human hepcidin

Active Comparator: Group B - Immediate therapy

standard chelation therapy plus LJPC-401 for 52 weeks

Drug: LJPC-401

subcutaneous injection, up to 20 mg per week from week 1 to 52

Other Names:

synthetic human hepcidin

Outcome Measures

Primary Outcome Measures

Effect of LJPC-401 on cardiac iron [26 Weeks]
Change in cardiac T2* magnetic resonance imaging (MRI)

Secondary Outcome Measures

Effect of LJPC-401 on cardiac iron [52 Weeks]
Change in cardiac T2*MRI
Effect of LJPC-401 on hepatic iron [52 Weeks]
Change in hepatic T2*MRI
Effect of LJPC-401 on serum iron [56 Weeks]
Mean change in serum iron
Effect of LJPC-401 on hemoglobin [52 Weeks]
Mean change in hemoglobin
Effect of LJPC-401 on volume of blood transfused [56 Weeks]
Change in transfused blood volume
Effect of LJPC-401 on its potential to elicit an immune response [56 Weeks]
Measured by blood laboratory tests and the presence of anti-drug antibodies
Effect of LJPC-401 on the incidence of treatment-emergent adverse events [56 Weeks]
Effect of LJPC-401 on vital signs-heart rate [56 Weeks]
Effect of LJPC-401 on vital signs-body temperature [56 Weeks]
Effect of LJPC-401 on vital signs-respiratory rate [56 Weeks]
Effect of LJPC-401 on vital signs-blood pressure [56 Weeks]
Effect of LJPC-401 on body weight [56 Weeks]
Change in body weight (kilograms)

Eligibility Criteria

Criteria

Ages Eligible for Study:

14 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigative Site	Oakland	California	United States	94609
2	Investigative Site	New York	New York	United States	10021
3	Investigative Site	Camperdown	New South Wales	Australia	2050
4	Investigative Site	Liverpool	New South Wales	Australia	2170
5	Investigative Site	Goudí	Attica	Greece	115 27
6	Investigative Site	Thessaloníki	Macedonia	Greece	54642
7	Investigative Site	Patra	Peloponnese	Greece	26504
8	Investigative Site	Cagliari		Italy	09121
9	Investigative Site	Modena		Italy	41124
10	Investigative Site	Napoli		Italy	80138
11	Investigative Site	Orbassano		Italy	10043
12	Investigative Site	Verona		Italy	37134
13	Investigative Site	Beirut		Lebanon	1107 2020
14	Investigative Site	Bangkok Noi		Thailand	10700
15	Investigative Site	Chiang Mai		Thailand	50200
16	Investigative Site	Adana		Turkey	01330
17	Investigative Site	Ankara		Turkey	06100
18	Investigative Site	Antalya		Turkey	07059
19	Investigative Site	Antalya		Turkey	07160
20	Investigative Site	İzmir		Turkey	35100
21	Investigative Site	London	England	United Kingdom	E1 1BB
22	Investigative Site	London		United Kingdom	N19 5NF

Sponsors and Collaborators

La Jolla Pharmaceutical Company

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

La Jolla Pharmaceutical Company

ClinicalTrials.gov Identifier:

NCT03381833

Other Study ID Numbers:

LJ401-BT01

First Posted:

Dec 22, 2017

Last Update Posted:

Jul 29, 2021

Last Verified:

Jul 1, 2021

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Study Results

No Results Posted as of Jul 29, 2021