A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

May 18, 2022 updated by: La Jolla Pharmaceutical Company

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
70

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Liverpool, New South Wales, Australia, 2170
        • Investigative Site
      • Westmead, New South Wales, Australia, 2145
        • Investigative Site
    • Queensland
      • Brisbane, Queensland, Australia, 4120
        • Investigative Site
      • Herston, Queensland, Australia, 4029
        • Investigative Site
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Investigative Site
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Investigative Site
  • France
      • Bondy, France, 93140
        • Investigative Site
      • Orléans, France, 45000
        • Investigative Site
      • Pessac, France, 33604
        • Investigative Site
      • Rennes Cedex 9, France, 35033
        • Investigative Site
  • United Kingdom
    • England
      • Bradford, England, United Kingdom, BD9 6RJ
        • Investigative Site
      • Newcastle Upon Tyne, England, United Kingdom, NE7 7DN
        • Investigative Site
      • Portsmouth, England, United Kingdom, PO6 3LY
        • Investigative Site
  • United States
    • Arkansas
      • North Little Rock, Arkansas, United States, 72117
        • Investigative Site
    • California
      • Los Angeles, California, United States, 90036
        • Investigative Site
      • Palo Alto, California, United States, 94305
        • Investigative Site
      • Rialto, California, United States, 92377
        • Investigative Site
      • San Diego, California, United States, 91942
        • Investigational Site
      • San Francisco, California, United States, 94115
        • Investigative Site
    • Florida
      • Jacksonville, Florida, United States, 32204
        • Investigative Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Investigative Site
    • Michigan
      • Wyoming, Michigan, United States, 49519
        • Investigative Site
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • Investigative Site
    • New York
      • East Setauket, New York, United States, 11733
        • Investigative Site
      • Manhasset, New York, United States, 11030
        • Investigative Site
      • New York, New York, United States, 10029
        • Investigative Site
    • Texas
      • Dallas, Texas, United States, 75246
        • Investigative Site
      • Fort Worth, Texas, United States, 76104
        • Investigative Site
      • Houston, Texas, United States, 77058
        • Investigative Site
      • San Antonio, Texas, United States, 78215
        • Investigative Site
    • Washington
      • Seattle, Washington, United States, 98104
        • Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Patients with clinical diagnosis of hereditary hemochromatosis
  2. Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis
  3. Patients with serum ferritin and TSAT levels above treatment guidelines
  4. 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
  5. 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
  6. Patient must be willing and able to provide written informed consent

Exclusion Criteria:

  1. Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug
  2. Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug
  3. Pregnant or lactating women
  4. Patients taking an immunosuppressive agent without prior Sponsor approval
  5. Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug
  6. Patients who are unwilling or unable to comply with the study protocol requirements
  7. Patients with type 1 or poorly controlled type 2 diabetes
  8. 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: LJPC-401
LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
Drug: LJPC-401
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.
Other Names:
  • synthetic human hepcidin
Placebo Comparator: Placebo
0.9% Sodium Chloride Injection, USP, or equivalent
Drug: Placebo
0.9% Sodium Chloride Injection, USP, or equivalent
Other Names:
  • Normal saline

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Time Frame: 16 Weeks
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
16 Weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Time Frame: 16 Weeks
16 Weeks
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Time Frame: 16 Weeks
Change in serum ferritin as measured by blood laboratory tests
16 Weeks
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Time Frame: 20 Weeks
20 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
La Jolla Pharmaceutical Company

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
PRA Health Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 29, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 28, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 28, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 12, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 3, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 10, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 9, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 18, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • LJ401-HH01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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