A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
Primary Purpose
Beta-Thalassemia
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LJPC-401
LJPC-401
Sponsored by
About this trial
This is an interventional treatment trial for Beta-Thalassemia
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.
Sites / Locations
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
- Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Active Comparator
Arm Label
Group A - Delayed therapy
Group B - Immediate therapy
Arm Description
standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
standard chelation therapy plus LJPC-401 for 52 weeks
Outcomes
Primary Outcome Measures
Effect of LJPC-401 on cardiac iron
Change in cardiac T2* magnetic resonance imaging (MRI)
Secondary Outcome Measures
Effect of LJPC-401 on cardiac iron
Change in cardiac T2*MRI
Effect of LJPC-401 on hepatic iron
Change in hepatic T2*MRI
Effect of LJPC-401 on serum iron
Mean change in serum iron
Effect of LJPC-401 on hemoglobin
Mean change in hemoglobin
Effect of LJPC-401 on volume of blood transfused
Change in transfused blood volume
Effect of LJPC-401 on its potential to elicit an immune response
Measured by blood laboratory tests and the presence of anti-drug antibodies
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Effect of LJPC-401 on vital signs-heart rate
Effect of LJPC-401 on vital signs-body temperature
Effect of LJPC-401 on vital signs-respiratory rate
Effect of LJPC-401 on vital signs-blood pressure
Effect of LJPC-401 on body weight
Change in body weight (kilograms)
Full Information
NCT ID
NCT03381833
First Posted
December 12, 2017
Last Updated
July 28, 2021
Sponsor
La Jolla Pharmaceutical Company
1. Study Identification
Unique Protocol Identification Number
NCT03381833
Brief Title
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
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
Terminated
Why Stopped
Terminated early for lack of efficacy as determined by interim endpoint analyses
Study Start Date
November 30, 2017 (Actual)
Primary Completion Date
December 3, 2019 (Actual)
Study Completion Date
January 14, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
La Jolla Pharmaceutical Company
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-Thalassemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
84 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group A - Delayed therapy
Arm Type
Active Comparator
Arm Description
standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
Arm Title
Group B - Immediate therapy
Arm Type
Active Comparator
Arm Description
standard chelation therapy plus LJPC-401 for 52 weeks
Intervention Type
Drug
Intervention Name(s)
LJPC-401
Other Intervention Name(s)
synthetic human hepcidin
Intervention Description
subcutaneous injection, up to 20 mg per week from week 26 to 52
Intervention Type
Drug
Intervention Name(s)
LJPC-401
Other Intervention Name(s)
synthetic human hepcidin
Intervention Description
subcutaneous injection, up to 20 mg per week from week 1 to 52
Primary Outcome Measure Information:
Title
Effect of LJPC-401 on cardiac iron
Description
Change in cardiac T2* magnetic resonance imaging (MRI)
Time Frame
26 Weeks
Secondary Outcome Measure Information:
Title
Effect of LJPC-401 on cardiac iron
Description
Change in cardiac T2*MRI
Time Frame
52 Weeks
Title
Effect of LJPC-401 on hepatic iron
Description
Change in hepatic T2*MRI
Time Frame
52 Weeks
Title
Effect of LJPC-401 on serum iron
Description
Mean change in serum iron
Time Frame
56 Weeks
Title
Effect of LJPC-401 on hemoglobin
Description
Mean change in hemoglobin
Time Frame
52 Weeks
Title
Effect of LJPC-401 on volume of blood transfused
Description
Change in transfused blood volume
Time Frame
56 Weeks
Title
Effect of LJPC-401 on its potential to elicit an immune response
Description
Measured by blood laboratory tests and the presence of anti-drug antibodies
Time Frame
56 Weeks
Title
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Time Frame
56 Weeks
Title
Effect of LJPC-401 on vital signs-heart rate
Time Frame
56 Weeks
Title
Effect of LJPC-401 on vital signs-body temperature
Time Frame
56 Weeks
Title
Effect of LJPC-401 on vital signs-respiratory rate
Time Frame
56 Weeks
Title
Effect of LJPC-401 on vital signs-blood pressure
Time Frame
56 Weeks
Title
Effect of LJPC-401 on body weight
Description
Change in body weight (kilograms)
Time Frame
56 Weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Investigative Site
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Investigative Site
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Investigative Site
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Investigative Site
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Investigative Site
City
Goudí
State/Province
Attica
ZIP/Postal Code
115 27
Country
Greece
Facility Name
Investigative Site
City
Thessaloníki
State/Province
Macedonia
ZIP/Postal Code
54642
Country
Greece
Facility Name
Investigative Site
City
Patra
State/Province
Peloponnese
ZIP/Postal Code
26504
Country
Greece
Facility Name
Investigative Site
City
Cagliari
ZIP/Postal Code
09121
Country
Italy
Facility Name
Investigative Site
City
Modena
ZIP/Postal Code
41124
Country
Italy
Facility Name
Investigative Site
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Facility Name
Investigative Site
City
Orbassano
ZIP/Postal Code
10043
Country
Italy
Facility Name
Investigative Site
City
Verona
ZIP/Postal Code
37134
Country
Italy
Facility Name
Investigative Site
City
Beirut
ZIP/Postal Code
1107 2020
Country
Lebanon
Facility Name
Investigative Site
City
Bangkok Noi
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Investigative Site
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
Facility Name
Investigative Site
City
Adana
ZIP/Postal Code
01330
Country
Turkey
Facility Name
Investigative Site
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
Investigative Site
City
Antalya
ZIP/Postal Code
07059
Country
Turkey
Facility Name
Investigative Site
City
Antalya
ZIP/Postal Code
07160
Country
Turkey
Facility Name
Investigative Site
City
İzmir
ZIP/Postal Code
35100
Country
Turkey
Facility Name
Investigative Site
City
London
State/Province
England
ZIP/Postal Code
E1 1BB
Country
United Kingdom
Facility Name
Investigative Site
City
London
ZIP/Postal Code
N19 5NF
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
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