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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

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.

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : November 29, 2017
Estimated Primary Completion Date : October 2018
Estimated Study Completion Date : December 2018

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) single use vial
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

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 on blood iron levels [ Time Frame: 12 weeks ]
    Change in transferrin saturation (TSAT) as measured by blood laboratory tests


Secondary Outcome Measures :
  1. Effect of LJPC-401 on blood iron levels [ Time Frame: 12 weeks ]
    Change in serum ferritin as measured by blood laboratory tests

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. Effect of LJPC-401 on the incidence of treatment-emergent adverse events [ Time Frame: 16 weeks ]
  8. Effect of LJPC-401 on incidence of phlebotomy [ Time Frame: 16 weeks ]
  9. Effect of LJPC-401 on blood pressure [ Time Frame: 16 weeks ]
    Change in blood pressure

  10. Effect of LJPC-401 on heart rate [ Time Frame: 16 weeks ]
    Change in heart rate (bpm)

  11. Effect of LJPC-401 on body weight [ Time Frame: 16 weeks ]
    Change in body weight (kilograms)

  12. Effect of LJPC-401 on body temperature [ Time Frame: 16 weeks ]
    Change in body temperature (Celsius)

  13. Effect of LJPC-401 on general health [ Time Frame: 16 weeks ]
    Change in physical examination (by body system)

  14. 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)



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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 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

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


Contacts
Contact: Brian J Byrnes 858-256-7913 bbyrnes@ljpc.com

Locations
United States, Arkansas
Investigative Site Recruiting
North Little Rock, Arkansas, United States, 72117
United States, California
Investigative Site Recruiting
Palo Alto, California, United States, 94305
Investigative Site Recruiting
Rialto, California, United States, 92377
Investigational Site Recruiting
San Diego, California, United States, 91942
United States, Michigan
Investigative Site Recruiting
Wyoming, Michigan, United States, 49519
United States, Mississippi
Investigative Site Recruiting
Jackson, Mississippi, United States, 39216
United States, New York
Investigative Site Recruiting
East Setauket, New York, United States, 11733
Investigative Site Recruiting
Manhasset, New York, United States, 11030
Investigative Site Recruiting
New York, New York, United States, 10029
United States, Texas
Investigative Site Recruiting
Dallas, Texas, United States, 75246
Investigative Site Recruiting
Fort Worth, Texas, United States, 76104
Investigative Site Recruiting
Houston, Texas, United States, 77058
Investigative Site Recruiting
San Antonio, Texas, United States, 78215
United States, Washington
Investigative Site Recruiting
Seattle, Washington, United States, 98104
Australia, New South Wales
Investigative Site Recruiting
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Investigative Site Recruiting
Brisbane, Queensland, Australia, 4120
Investigative Site Recruiting
Herston, Queensland, Australia, 4029
Australia, Victoria
Investigative Site Recruiting
Melbourne, Victoria, Australia, 3004
France
Investigative Site Recruiting
Bondy, France, 93140
Investigative Site Recruiting
Orléans, France, 45000
Investigative Site Recruiting
Pessac, France, 33604
Investigative Site Recruiting
Rennes Cedex 9, France, 35033
United Kingdom
Investigative Site Recruiting
Bradford, England, United Kingdom, BD9 6RJ
Investigative Site Recruiting
Newcastle Upon Tyne, England, United Kingdom, NE7 7DN
Investigative Site Recruiting
Portsmouth, England, United Kingdom, PO6 3LY
Sponsors and Collaborators
La Jolla Pharmaceutical Company
PRA Health Sciences

Responsible Party: La Jolla Pharmaceutical Company
ClinicalTrials.gov Identifier: NCT03395704     History of Changes
Other Study ID Numbers: LJ401-HH01
First Posted: January 10, 2018    Key Record Dates
Last Update Posted: July 26, 2018
Last Verified: June 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Iron Overload
Hemochromatosis
Iron Metabolism Disorders
Metabolic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn