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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 10, 2019
Sponsor:
Collaborator:
PRA Health Sciences
Information provided by (Responsible Party):
La Jolla Pharmaceutical Company

Tracking Information
First Submitted Date  ICMJE December 12, 2017
First Posted Date  ICMJE January 10, 2018
Last Update Posted Date July 10, 2019
Actual Study Start Date  ICMJE November 29, 2017
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 8, 2019)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 3, 2018)
Effect of LJPC-401 on blood iron levels [ Time Frame: 12 weeks ]
Change in transferrin saturation (TSAT) as measured by blood laboratory tests
Change History Complete list of historical versions of study NCT03395704 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 8, 2019)
  • Effect of LJPC-401 versus placebo on incidence of phlebotomy [ Time Frame: 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
  • Effect of LJPC-401 versus placebo on the incidence of treatment-emergent adverse events [ Time Frame: 20 Weeks ]
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • Effect of LJPC-401 versus placebo on blood pressure [ Time Frame: 20 Weeks ]
    Safety measure for change in blood pressure.
  • Effect of LJPC-401 versus placebo on heart rate [ Time Frame: 20 Weeks ]
    Safety measure for change in heart rate (bpm).
  • Effect of LJPC-401 versus placebo on body weight [ Time Frame: 20 Weeks ]
    Safety measure for change in body weight (kilograms).
  • Effect of LJPC-401 versus placebo on body temperature [ Time Frame: 20 Weeks ]
    Safety measure for change in body temperature (Celsius).
  • Effect of LJPC-401 versus placebo on general health [ Time Frame: 20 Weeks ]
    Safety measure for change in physical examination (by body system).
  • 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).
  • 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.
  • 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 SF-36v2 (Short Form Health Survey version 2.0) questionnaire.
  • 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 WOMAC (Western Ontario and McMaster Universities Osteoarthritis Index) questionnaire.
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: January 3, 2018)
  • Effect of LJPC-401 on blood iron levels [ Time Frame: 12 weeks ]
    Change in serum ferritin as measured by blood laboratory tests
  • Effect of LJPC-401 on clinical chemistry laboratory parameters [ Time Frame: 16 weeks ]
    Safety data 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 at screening to confirm eligibility.
  • Effect of LJPC-401 on hematology laboratory parameters [ Time Frame: 16 weeks ]
    Safety data 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.
  • Effect of LJPC-401 on endocrine laboratory parameters [ Time Frame: 16 Weeks ]
    Safety data for endocrine laboratory parameters, including thyroid-stimulating hormone, thyroxine (T4), and triiodothyronine (T3) will be assessed to evaluate changes in thyroid function.
  • Effect of LJPC-401 on urinalysis laboratory parameters [ Time Frame: 16 weeks ]
    Safety data 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 12/end of treatment for additional safety assessment.
  • Effect of LJPC-401 on its potential to elicit an immune response [ Time Frame: 16 weeks ]
    Blood samples will be collected to assess the presence of anti-drug antibodies.
  • Effect of LJPC-401 on the incidence of treatment-emergent adverse events [ Time Frame: 16 weeks ]
  • Effect of LJPC-401 on incidence of phlebotomy [ Time Frame: 12 weeks ]
  • Effect of LJPC-401 on blood pressure [ Time Frame: 16 weeks ]
    Change in blood pressure
  • Effect of LJPC-401 on heart rate [ Time Frame: 16 weeks ]
    Change in heart rate (bpm)
  • Effect of LJPC-401 on body weight [ Time Frame: 16 weeks ]
    Change in body weight (kilograms)
  • Effect of LJPC-401 on body temperature [ Time Frame: 16 weeks ]
    Change in body temperature (Celsius)
  • Effect of LJPC-401 on general health [ Time Frame: 16 weeks ]
    Change in physical examination (by body system)
  • Effect of LJPC-401 on electrical activity of the heart [ Time Frame: 16 weeks ]
    Change in the electrical activity of the heart over a period of time using electrodes placed on the skin and recorded by electrocardiogram (ECG)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Hemochromatosis
Intervention  ICMJE
  • 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
  • Drug: Placebo
    0.9% Sodium Chloride Injection, USP, or equivalent
    Other Name: Normal saline
Study Arms  ICMJE
  • Active Comparator: LJPC-401
    LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
    Intervention: Drug: LJPC-401
  • Placebo Comparator: Placebo
    0.9% Sodium Chloride Injection, USP, or equivalent
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 8, 2019)
69
Original Estimated Enrollment  ICMJE
 (submitted: January 3, 2018)
60
Estimated Study Completion Date  ICMJE October 2019
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   France,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03395704
Other Study ID Numbers  ICMJE LJ401-HH01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party La Jolla Pharmaceutical Company
Study Sponsor  ICMJE La Jolla Pharmaceutical Company
Collaborators  ICMJE PRA Health Sciences
Investigators  ICMJE Not Provided
PRS Account La Jolla Pharmaceutical Company
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP