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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03395704
Recruitment Status : Active, not recruiting
First Posted : January 10, 2018
Last Update Posted : July 30, 2019
Sponsor:
Collaborator:
PRA Health Sciences
Information provided by (Responsible Party):
La Jolla Pharmaceutical Company

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.

Condition or disease Intervention/treatment Phase
Hereditary Hemochromatosis Drug: LJPC-401 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 69 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
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
Actual Study Start Date : November 29, 2017
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : October 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemochromatosis Iron

Arm Intervention/treatment
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 Name: 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 Name: Normal saline




Primary Outcome Measures :
  1. Effect of LJPC-401 versus placebo on blood iron levels [ Time Frame: 16 Weeks ]
    Change in transferrin saturation (TSAT) as measured by blood laboratory tests.


Secondary Outcome Measures :
  1. Effect of LJPC-401 versus placebo on incidence of phlebotomy [ Time Frame: 16 Weeks ]
  2. Effect of LJPC-401 versus placebo on blood iron levels [ Time Frame: 16 Weeks ]
    Change in serum ferritin as measured by blood laboratory tests

  3. Effect of LJPC-401 versus placebo on the incidence of treatment-emergent adverse events [ Time Frame: 20 Weeks ]
  4. Effect of LJPC-401 versus placebo on clinical chemistry laboratory parameters [ Time Frame: 20 Weeks ]
    Safety measure for clinical chemistry parameters including serum creatinine, uric acid, alkaline phosphatase, blood urea nitrogen, albumin, bilirubin (total), phosphate, bicarbonate, lactate dehydrogenase, glucose, calcium, magnesium, chloride, sodium, potassium, alanine aminotransferase, aspartate aminotransferase, and total protein will be assessed. In addition, serum pregnancy test will be assessed.

  5. Effect of LJPC-401 versus placebo on hematology laboratory parameters [ Time Frame: 20 Weeks ]
    Safety measure for laboratory hematology parameters including platelet count, red blood cell count, absolute white blood cell count, hemoglobin, hematocrit, reticulocyte count, red cell distribution width, mean corpuscular hemoglobin, mean corpuscular volume, and mean corpuscular hemoglobin concentration will be assessed.

  6. Effect of LJPC-401 versus placebo on blood iron parameters [ Time Frame: 20 Weeks ]
    Exploratory measure of serum iron, serum transferrin, total iron-binding capacity, unsaturation iron-binding capacity will be assessed to evaluate iron parameters.

  7. Effect of LJPC-401 versus placebo on endocrine laboratory parameters [ Time Frame: 16 Weeks ]
    Exploratory measure for endocrine laboratory parameters, including thyroid-stimulating hormone, thyroxine (T4), and triiodothyronine (T3) will be assessed to evaluate changes in thyroid function.

  8. Effect of LJPC-401 versus placebo on glucose levels [ Time Frame: 16 Weeks ]
    Exploratory measure for change in hemoglobin A1c will be assessed to evaluate blood sugar levels.

  9. Effect of LJPC-401 versus placebo on urinalysis laboratory parameters [ Time Frame: 20 Weeks ]
    Safety measure for urinalysis laboratory parameters including specific gravity, bilirubin, glucose, pH, protein, nitrite, blood, ketones will be assessed. In addition, urine pregnancy test will be assessed at Week 1 (Day 1) to reconfirm eligibility predose and at again at Week 20 for additional safety assessment.

  10. Effect of LJPC-401 versus placebo on blood pressure [ Time Frame: 20 Weeks ]
    Safety measure for change in blood pressure.

  11. Effect of LJPC-401 versus placebo on heart rate [ Time Frame: 20 Weeks ]
    Safety measure for change in heart rate (bpm).

  12. Effect of LJPC-401 versus placebo on body weight [ Time Frame: 20 Weeks ]
    Safety measure for change in body weight (kilograms).

  13. Effect of LJPC-401 versus placebo on body temperature [ Time Frame: 20 Weeks ]
    Safety measure for change in body temperature (Celsius).

  14. Effect of LJPC-401 versus placebo on general health [ Time Frame: 20 Weeks ]
    Safety measure for change in physical examination (by body system).

  15. Effect of LJPC-401 versus placebo on electrical activity of the heart [ Time Frame: 20 Weeks ]
    Safety measure for change in the electrical activity of the heart over a period of time using electrodes placed on the skin and recorded by electrocardiogram (ECG).

  16. Effect of LJPC-401 versus placebo on its potential to elicit an immune response [ Time Frame: 20 Weeks ]
    Safety measure of blood samples collected to assess the presence of anti-drug antibodies.

  17. Effect of LJPC-401 versus placebo on quality of life [ Time Frame: 16 Weeks ]
    Exploratory measure for change in quality of life as measured by the Short Form Health Survey version 2.0 questionnaire. The scale measures physical functioning; role-physical, bodily pain; general health; vitality; social functioning; role-emotional; mental health and reported health transition. The responses vary depending on the question: Range 1-6 (none to very severe); range 1-5 (all of the time to none of the time or definitely true to definitely false); range 1-4 not at all to quite a bit; range 1-3 (yes, limited a lot to no, not limited at all). All scoring will be tallied by a validated central system (Optum) and provided to the sponsor at the end of the study. Scoring will be analyzed as a change from baseline to end of treatment (Week 16).

  18. Effect of LJPC-401 versus placebo on quality of life [ Time Frame: 16 Weeks ]
    Exploratory measure for change in quality of life as measured by the Western Ontario and McMaster Universities Osteoarthritis Index questionnaire. The scale ranges from 1-5 (none to extreme in regards to pain and difficulty during day to day activity. Responses will be analyzed as a change from baseline to end of treatment (Week 16).

  19. Exploratory measure for change in quality of life [ Time Frame: 16 Weeks ]
    Exploratory measure for change in quality of life as measured by the Modified Dreisler indicies (FIOHA-Functional Index for Hand OsteoArthritis) questionnaire. The scale ranges from 1-4 (Possible without difficulty to impossible in regards to activities that use hand movement. The results are totaled and scores will be analyzed as a change from baseline to end of treatment (Week 16).



Information from the National Library of Medicine

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03395704


Locations
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United States, Arkansas
Investigative Site
North Little Rock, Arkansas, United States, 72117
United States, California
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
United States, Florida
Investigative Site
Jacksonville, Florida, United States, 32204
United States, Indiana
Investigative Site
Indianapolis, Indiana, United States, 46202
United States, Michigan
Investigative Site
Wyoming, Michigan, United States, 49519
United States, Mississippi
Investigative Site
Jackson, Mississippi, United States, 39216
United States, New York
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
United States, Texas
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
United States, Washington
Investigative Site
Seattle, Washington, United States, 98104
Australia, New South Wales
Investigative Site
Liverpool, New South Wales, Australia, 2170
Investigative Site
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Investigative Site
Brisbane, Queensland, Australia, 4120
Investigative Site
Herston, Queensland, Australia, 4029
Australia, Victoria
Investigative Site
Melbourne, Victoria, Australia, 3004
Australia, Western Australia
Investigative Site
Murdoch, Western Australia, Australia, 6150
France
Investigative Site
Bondy, France, 93140
Investigative Site
Orléans, France, 45000
Investigative Site
Pessac, France, 33604
Investigative Site
Rennes Cedex 9, France, 35033
United Kingdom
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
Sponsors and Collaborators
La Jolla Pharmaceutical Company
PRA Health Sciences

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Responsible Party: La Jolla Pharmaceutical Company
ClinicalTrials.gov Identifier: NCT03395704    
Other Study ID Numbers: LJ401-HH01
First Posted: January 10, 2018    Key Record Dates
Last Update Posted: July 30, 2019
Last Verified: July 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemochromatosis
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hepcidins
Anti-Infective Agents