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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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ClinicalTrials.gov Identifier: NCT03395704
Recruitment Status : Recruiting
First Posted : January 10, 2018
Last Update Posted : July 26, 2018
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 26, 2018
Actual Study Start Date  ICMJE November 29, 2017
Estimated Primary Completion Date October 2018   (Final data collection date for primary outcome measure)
Current 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
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03395704 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 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: 16 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)
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, Double-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, double-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: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Hemochromatosis
Intervention  ICMJE
  • Drug: LJPC-401
    LJPC-401 subcutaneous injection, up to 20 mg weekly for 12 weeks. The minimum weekly dose will be 1 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) 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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 3, 2018)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2018
Estimated Primary Completion Date October 2018   (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 elevated serum ferritin and TSAT levels
  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
  2. Patients initiating phlebotomy treatments less than 6 months prior to the first dose of study drug
  3. Pregnant or lactating women
  4. Patients taking an immunosuppressive agent
  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 must not have a medical condition that will interfere with the conduct of the clinical study
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: Brian J Byrnes 858-256-7913 bbyrnes@ljpc.com
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 2018

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