A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Primary Purpose
Hereditary Hemochromatosis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LJPC-401
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Hemochromatosis
Eligibility Criteria
Inclusion Criteria:
- Patients with clinical diagnosis of hereditary hemochromatosis
- Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis
- Patients with serum ferritin and TSAT levels above treatment guidelines
- Female patients of child bearing potential must have a negative pregnancy test and must be using a highly effective method of contraception during participation in the study, and for 30 days after the last dose of study drug
- Males must be surgically sterile (vasectomy), or using a highly effective method of contraception during participation in the study, and for 30 days after the last dose of study drug
- Patient must be willing and able to provide written informed consent
Exclusion Criteria:
- Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug
- Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug
- Pregnant or lactating women
- Patients taking an immunosuppressive agent without prior Sponsor approval
- Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug
- Patients who are unwilling or unable to comply with the study protocol requirements
- Patients with type 1 or poorly controlled type 2 diabetes
- Patients with a concomitant disease, disability or condition, including laboratory abnormality and ECG findings, 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 in this study, including, but not limited to, clinically significant arrhythmias, alcohol dependency or abuse, drug dependency or abuse, or psychiatric disease
Sites / Locations
- Investigative Site
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- Investigative Site
- Investigative Site
- Investigative Site
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- Investigative Site
- Investigative Site
- Investigative Site
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- Investigative Site
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- Investigative Site
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- Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
LJPC-401
Placebo
Arm Description
LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
0.9% Sodium Chloride Injection, USP, or equivalent
Outcomes
Primary Outcome Measures
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Secondary Outcome Measures
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Change in serum ferritin as measured by blood laboratory tests
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Full Information
NCT ID
NCT03395704
First Posted
December 12, 2017
Last Updated
May 18, 2022
Sponsor
La Jolla Pharmaceutical Company
Collaborators
PRA Health Sciences
1. Study Identification
Unique Protocol Identification Number
NCT03395704
Brief Title
A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Official Title
A Phase 2, Multi-Center, Randomized, Placebo Controlled, Single-Blind Study With LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
November 29, 2017 (Actual)
Primary Completion Date
October 28, 2019 (Actual)
Study Completion Date
October 28, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
La Jolla Pharmaceutical Company
Collaborators
PRA Health Sciences
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, placebo controlled, single-blind study. The primary objective of the study is to compare the effect of weekly dosing of LJPC-401 (synthetic human hepcidin) versus placebo on transferrin saturation (TSAT) in an adult hereditary hemochromatosis patient population.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Hemochromatosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
70 (Actual)
8. Arms, Groups, and Interventions
Arm Title
LJPC-401
Arm Type
Active Comparator
Arm Description
LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
0.9% Sodium Chloride Injection, USP, or equivalent
Intervention Type
Drug
Intervention Name(s)
LJPC-401
Other Intervention Name(s)
synthetic human hepcidin
Intervention Description
LJPC-401 subcutaneous injection, up to 20 mg weekly for 16 weeks. The minimum weekly dose will be 5 mg and the maximum weekly dose of LJPC-401 will be 20 mg.
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Normal saline
Intervention Description
0.9% Sodium Chloride Injection, USP, or equivalent
Primary Outcome Measure Information:
Title
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Description
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Time Frame
16 Weeks
Secondary Outcome Measure Information:
Title
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Time Frame
16 Weeks
Title
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Description
Change in serum ferritin as measured by blood laboratory tests
Time Frame
16 Weeks
Title
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Time Frame
20 Weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with clinical diagnosis of hereditary hemochromatosis
Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis
Patients with serum ferritin and TSAT levels above treatment guidelines
Female patients of child bearing potential must have a negative pregnancy test and must be using a highly effective method of contraception during participation in the study, and for 30 days after the last dose of study drug
Males must be surgically sterile (vasectomy), or using a highly effective method of contraception during participation in the study, and for 30 days after the last dose of study drug
Patient must be willing and able to provide written informed consent
Exclusion Criteria:
Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug
Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug
Pregnant or lactating women
Patients taking an immunosuppressive agent without prior Sponsor approval
Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug
Patients who are unwilling or unable to comply with the study protocol requirements
Patients with type 1 or poorly controlled type 2 diabetes
Patients with a concomitant disease, disability or condition, including laboratory abnormality and ECG findings, 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 in this study, including, but not limited to, clinically significant arrhythmias, alcohol dependency or abuse, drug dependency or abuse, or psychiatric disease
Facility Information:
Facility Name
Investigative Site
City
North Little Rock
State/Province
Arkansas
ZIP/Postal Code
72117
Country
United States
Facility Name
Investigative Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90036
Country
United States
Facility Name
Investigative Site
City
Palo Alto
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Investigative Site
City
Rialto
State/Province
California
ZIP/Postal Code
92377
Country
United States
Facility Name
Investigational Site
City
San Diego
State/Province
California
ZIP/Postal Code
91942
Country
United States
Facility Name
Investigative Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
Investigative Site
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32204
Country
United States
Facility Name
Investigative Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Investigative Site
City
Wyoming
State/Province
Michigan
ZIP/Postal Code
49519
Country
United States
Facility Name
Investigative Site
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Investigative Site
City
East Setauket
State/Province
New York
ZIP/Postal Code
11733
Country
United States
Facility Name
Investigative Site
City
Manhasset
State/Province
New York
ZIP/Postal Code
11030
Country
United States
Facility Name
Investigative Site
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Investigative Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75246
Country
United States
Facility Name
Investigative Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Investigative Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77058
Country
United States
Facility Name
Investigative Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78215
Country
United States
Facility Name
Investigative Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Facility Name
Investigative Site
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Investigative Site
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Investigative Site
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4120
Country
Australia
Facility Name
Investigative Site
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Investigative Site
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Investigative Site
City
Murdoch
State/Province
Western Australia
ZIP/Postal Code
6150
Country
Australia
Facility Name
Investigative Site
City
Bondy
ZIP/Postal Code
93140
Country
France
Facility Name
Investigative Site
City
Orléans
ZIP/Postal Code
45000
Country
France
Facility Name
Investigative Site
City
Pessac
ZIP/Postal Code
33604
Country
France
Facility Name
Investigative Site
City
Rennes Cedex 9
ZIP/Postal Code
35033
Country
France
Facility Name
Investigative Site
City
Bradford
State/Province
England
ZIP/Postal Code
BD9 6RJ
Country
United Kingdom
Facility Name
Investigative Site
City
Newcastle Upon Tyne
State/Province
England
ZIP/Postal Code
NE7 7DN
Country
United Kingdom
Facility Name
Investigative Site
City
Portsmouth
State/Province
England
ZIP/Postal Code
PO6 3LY
Country
United Kingdom
12. IPD Sharing Statement
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A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
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