A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

Recruitment Status
COMPLETED - HAS RESULTS
(See Contacts and Locations)Verified April 2022 by La Jolla Pharmaceutical Company
Sponsor
La Jolla Pharmaceutical Company
Information Provided by (Responsible Party)
La Jolla Pharmaceutical Company
Clinicaltrials.gov Identifier
NCT03395704
Other Study ID Numbers:
LJ401-HH01
First Submitted
December 11, 2017
First Posted
January 9, 2018
Results First Posted
December 14, 2021
Last Update Posted
June 8, 2022
Last Verified
April 2022

ClinicalTrials.gov processed this data on May 2022. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Hereditary Hemochromatosis
Drug: LJPC-401Drug: Placebo

Study Design

Study TypeInterventional
Actual Enrollment70 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingSingle
Primary PurposeTreatment
Official TitleA 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
Study Start DateNovember 28, 2017
Actual Primary Completion DateOctober 27, 2019
Actual Study Completion DateOctober 27, 2019

Groups and Cohorts

Group/CohortIntervention/Treatment
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.
Placebo
0.9% Sodium Chloride Injection, USP, or equivalent
Drug: Placebo
0.9% Sodium Chloride Injection, USP, or equivalent

Outcome Measures

Primary Outcome Measures
  1. Effect of LJPC-401 Versus Placebo on Blood Iron Levels
    Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Secondary Outcome Measures
  1. Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
  2. Effect of LJPC-401 Versus Placebo on Blood Iron Levels
    Change in serum ferritin as measured by blood laboratory tests
  3. Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events

Eligibility Criteria

Ages Eligible for Study(Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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

Contacts and Locations

Sponsors and CollaboratorsLa Jolla Pharmaceutical Company
Locations
Investigative Site | North Little Rock Arkansas, United States, 72117Investigative Site | Los Angeles California, United States, 90036Investigative Site | Palo Alto California, United States, 94305Investigative Site | Rialto California, United States, 92377Investigational Site | San Diego California, United States, 91942Investigative Site | San Francisco California, United States, 94115Investigative Site | Jacksonville Florida, United States, 32204Investigative Site | Indianapolis Indiana, United States, 46202Investigative Site | Wyoming Michigan, United States, 49519Investigative Site | Jackson Mississippi, United States, 39216Investigative Site | East Setauket New York, United States, 11733Investigative Site | Manhasset New York, United States, 11030Investigative Site | New York New York, United States, 10029Investigative Site | Dallas Texas, United States, 75246Investigative Site | Fort Worth Texas, United States, 76104Investigative Site | Houston Texas, United States, 77058Investigative Site | San Antonio Texas, United States, 78215Investigative Site | Seattle Washington, United States, 98104Investigative Site | Liverpool New South Wales, Australia, 2170Investigative Site | Westmead New South Wales, Australia, 2145Investigative Site | Brisbane Queensland, Australia, 4120Investigative Site | Herston Queensland, Australia, 4029Investigative Site | Melbourne Victoria, Australia, 3004Investigative Site | Murdoch Western Australia, Australia, 6150Investigative Site | Bondy , France, 93140Investigative Site | Orléans , France, 45000Investigative Site | Pessac , France, 33604Investigative Site | Rennes , France, 35033Investigative Site | Bradford England, United Kingdom, BD9 6RJInvestigative Site | Newcastle upon Tyne England, United Kingdom, NE7 7DNInvestigative Site | Portsmouth England, United Kingdom, PO6 3LY
Study Documents (Full Text)
Documents provided by La Jolla Pharmaceutical Company: Study Protocol  December 13, 2018Documents provided by La Jolla Pharmaceutical Company: Statistical Analysis Plan  March 21, 2019