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

ClinicalTrials.gov processed this data on May 18, 2022. Link to the current ClinicalTrials.gov record.

Recruitment Status

COMPLETED - HAS RESULTS
 (See Contacts and Locations)
Verified May 2022 by La Jolla Pharmaceutical Company, PRA Health Sciences

Sponsor

La Jolla Pharmaceutical Company

Information Provided by (Responsible Party)

La Jolla Pharmaceutical Company

Clinicaltrials.gov Identifier

NCT03395704
Other Study ID Numbers: LJ401-HH01
First Submitted: December 12, 2017
First Posted: January 10, 2018
Results First Posted: June 9, 2022
Last Update Posted: June 9, 2022
Last Verified: May 2022
History of Changes

Listing a study on this site does not mean it has been evaluated by the U.S. Federal Government. The safety and scientific validity of a study listed on ClinicalTrials.gov is the responsibility of the study sponsor and investigators. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating.

ClinicalTrials.gov, a resource provided by the U.S. National Library of Medicine (NLM), is a registry and results information database of clinical research studies sponsored or funded by a broad range of public and private organizations around the world. Not all studies listed on ClinicalTrials.gov are funded by the National Institutes of Health (NIH) or other agencies of the U.S. Federal Government. Not all listed studies are regulated and/or reviewed by the U.S. Food and Drug Administration or other governmental entities.

Information on ClinicalTrials.gov is provided by study sponsors and investigators, and they are responsible for ensuring that the studies follow all applicable laws and regulations. NLM staff do not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.

Choosing to participate in a study is an important personal decision. Before you participate in a study, discuss all options with your health care provider and other trusted advisors. For more information about participating in clinical studies, see Learn About Clinical Studies, which includes questions that you might want to ask before deciding to participate in a study.

For more information about using the information on ClinicalTrials.gov, please also see Terms and Conditions.

See also the Web Policies and Notices for the NIH web site.

Study Description

Not Provided
Condition or Disease Intervention/Treatment
  • Hereditary Hemochromatosis
  • Drug: LJPC-401
  • Drug: Placebo

Study Design

Study TypeInterventional
Actual Enrollment70 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingSingle
Primary PurposeTreatment
Official TitleA 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 Start DateNovember 29, 2017
Actual Primary Completion DateOctober 28, 2019
Actual Study Completion DateOctober 28, 2019

Groups and Cohorts

Group/ CohortIntervention/ Treatment
  • 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.
  • Placebo
    • 0.9% Sodium Chloride Injection, USP, or equivalent
  • Drug: Placebo

    Outcome Measures

    Primary Outcome Measures

    1. Effect of LJPC-401 Versus Placebo on Blood Iron Levels [16 Weeks]
      Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.

    Secondary Outcome Measures

    1. Effect of LJPC-401 Versus Placebo on Number of Phlebotomies [16 Weeks]
    2. Effect of LJPC-401 Versus Placebo on Blood Iron Levels [16 Weeks]
      Change in serum ferritin as measured by blood laboratory tests
    3. Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events [20 Weeks]

    Eligibility Criteria

    Ages Eligible for Study 18 Years and Older (Adult, Older Adult)
    Sexes Eligible for Study All
    Accepts Healthy Volunteers No
    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

    Contacts and Locations

    Sponsors and Collaborators La Jolla Pharmaceutical Company, PRA Health Sciences
    PRA Health Sciences
    Locations
    • Investigative Site | North Little Rock, Arkansas, United States, 72117
    • Investigative Site | Los Angeles, California, United States, 90036
    • Investigative Site | Palo Alto, California, United States, 94305
    • Investigative Site | Rialto, California, United States, 92377
    • Investigational Site | San Diego, California, United States, 91942
    • Investigative Site | San Francisco, California, United States, 94115
    • Investigative Site | Jacksonville, Florida, United States, 32204
    • Investigative Site | Indianapolis, Indiana, United States, 46202
    • Investigative Site | Wyoming, Michigan, United States, 49519
    • Investigative Site | Jackson, Mississippi, United States, 39216
    • Investigative Site | 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 | 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 | Seattle, Washington, United States, 98104
    • Investigative Site | Liverpool, New South Wales, Australia, 2170
    • Investigative Site | Westmead, New South Wales, Australia, 2145
    • Investigative Site | Brisbane, Queensland, Australia, 4120
    • Investigative Site | Herston, Queensland, Australia, 4029
    • Investigative Site | Melbourne, Victoria, Australia, 3004
    • Investigative Site | Murdoch, Western Australia, Australia, 6150
    • Investigative Site | Bondy, France, 93140
    • Investigative Site | OrlĂ©ans, France, 45000
    • Investigative Site | Pessac, France, 33604
    • Investigative Site | Rennes Cedex 9, France, 35033
    • Investigative Site | Bradford, England, United Kingdom, BD9 6RJ
    • Investigative Site | Newcastle Upon Tyne, England, United Kingdom, NE7 7DN
    • Investigative Site | Portsmouth, England, United Kingdom, PO6 3LY

    Study Documents (Full Text)

    More Information

    Additional Relevant MeSH Terms

    • Hemochromatosis
    • Iron Overload
    • Hemosiderosis
    • Iron Metabolism Disorders
    • Metabolic Diseases
    • Metal Metabolism, Inborn Errors
    • Metabolism, Inborn Errors
    • Genetic Diseases, Inborn