Hereditary Haemochromatosis

LJPC-401 Trial

Do you have Hereditary Haemochromatosis?

Symptoms such as weakness and joint pain present special challenges in the daily lives of patients with hereditary haemochromatosis (HH). Treatment options for HH are often difficult and tiring, so additional ways to treat HH are being studied.

Doctors are looking for volunteers who have been diagnosed with HH to help evaluate a study drug. The primary purpose of this research study is to measure how well and how safe the study drug is in reducing the amount of iron in the blood.


You may qualify to enrol in this study if you are at least 18 years old and:

  • Diagnosed with hereditary haemochromatosis
  • Have elevated iron levels
  • On prescribed phlebotomy for at least three months
  • Do not have type 1 diabetes

Additional criteria will apply.


If you agree to participate in this study, you may be involved for up to 32 weeks. If the doctor determines that you are eligible to remain in the study, you will be placed in one of two groups:

  • Group 1: Receives the study drug.
  • Group 2: Receives a placebo. The placebo contains no active medication.

Participants are placed into these groups randomly (like the flip of a coin), so each participant has an equal 50 percent chance of being placed into the study drug or the placebo group.


Prequalified participants will go through a screening process, which includes medical tests and procedures such as:

  • Physical exam with height, weight, and vital signs.
  • Electrocardiogram, also called ECG—a painless test that measures the electrical activity of your heart.
  • Talking to the doctor about what medicines you take and your health history.
  • Getting your blood drawn to check your iron levels plus other routine blood tests to check your current health. For some tests, you will need to fast for at least six hours prior, which means you will not eat or drink anything but water for at least six hours prior to having your blood drawn.
  • Urinating in a cup for routine testing.

Treatment and Follow-up

Participants will come into the study clinic to receive a study drug injection once a week for 16 weeks. At the first treatment period visit, patients have a blood test, complete a quality of life questionnaire, and undergo procedures like those at screening. Approximately 30 days after your last dose of study drug, you will come to the study clinic for a final visit.


As with any research study, there may be side effects or risks. For this study, common side effects may include injection site reactions, headache, nausea and decreased appetite. You will be monitored carefully by the study doctor at the study clinic visits.

While you may experience decreased iron levels during the study, it is not guaranteed. Your condition may worsen, improve, or stay the same. Your participation in this study will provide important information to health researchers that may potentially help future patients with HH.

You will receive the study drug and all study-related care at no cost. Phlebotomy visits are considered standard care and will not be paid for by the study sponsor.


Patients have certain responsibilities when enrolled in the study, both for your safety and for study outcomes. You must:

  1. Attend all scheduled study visits.
  2. Tell the doctor about any changes to your medications.
  3. Tell the doctor about any side effects, even if you think they aren’t important.
  4. Inform the study staff if you decide you no longer wish to participate in the study.

Thank you for considering this important research study. For more information or to find out if you might be eligible to participate, please contact our nursing staff on (08) 9242 7640 or fill out this form