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 :
Completed
First Posted : January 10, 2018
Results First Posted : June 9, 2022
Last Update Posted : June 9, 2022
|
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hereditary Hemochromatosis | Drug: LJPC-401 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 70 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 |
Actual Primary Completion Date : | October 28, 2019 |
Actual Study Completion Date : | October 28, 2019 |

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 |
- Effect of LJPC-401 Versus Placebo on Blood Iron Levels [ Time Frame: 16 Weeks ]Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
- Effect of LJPC-401 Versus Placebo on Number of Phlebotomies [ 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 Total Number of Treatment-emergent Adverse Events [ Time Frame: 20 Weeks ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients with clinical diagnosis of hereditary hemochromatosis
- Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis
- Patients with serum ferritin and TSAT levels above treatment guidelines
- 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
- 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
- Patient must be willing and able to provide written informed consent
Exclusion Criteria:
- Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug
- Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug
- Pregnant or lactating women
- Patients taking an immunosuppressive agent without prior Sponsor approval
- Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug
- Patients who are unwilling or unable to comply with the study protocol requirements
- Patients with type 1 or poorly controlled type 2 diabetes
- 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

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
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 |
Documents provided by La Jolla Pharmaceutical Company:
Responsible Party: | La Jolla Pharmaceutical Company |
ClinicalTrials.gov Identifier: | NCT03395704 |
Other Study ID Numbers: |
LJ401-HH01 |
First Posted: | January 10, 2018 Key Record Dates |
Results First Posted: | June 9, 2022 |
Last Update Posted: | June 9, 2022 |
Last Verified: | May 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Hemochromatosis Iron Overload Hemosiderosis Iron Metabolism Disorders Metabolic Diseases |
Metal Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Hepcidins Anti-Infective Agents |