Clinical trial NCT03395704: A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Hemochromatosis
Lead sponsor: La Jolla Pharmaceutical Company
Sponsor class: INDUSTRY
Collaborators: name: PRA Health Sciences
First posted: 2018-01-10
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is a Phase 2 multicenter, randomized, placebo controlled, single-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.


Eligibility 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 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

Primary outcomes:
measure: Effect of LJPC-401 Versus Placebo on Blood Iron Levels
timeFrame: 16 Weeks
Secondary outcomes:
measure: Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
timeFrame: 16 Weeks
measure: Effect of LJPC-401 Versus Placebo on Blood Iron Levels
timeFrame: 16 Weeks
measure: Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
timeFrame: 20 Weeks
Other outcomes:
Locations:
city: North Little Rock
country: United States
facility: Investigative Site
state: Arkansas
city: Los Angeles
country: United States
facility: Investigative Site
state: California
city: Palo Alto
country: United States
facility: Investigative Site
state: California
city: Rialto
country: United States
facility: Investigative Site
state: California
city: San Diego
country: United States
facility: Investigational Site
state: California
city: San Francisco
country: United States
facility: Investigative Site
state: California
city: Jacksonville
country: United States
facility: Investigative Site
state: Florida
city: Indianapolis
country: United States
facility: Investigative Site
state: Indiana
city: Wyoming
country: United States
facility: Investigative Site
state: Michigan
city: Jackson
country: United States
facility: Investigative Site
state: Mississippi
city: East Setauket
country: United States
facility: Investigative Site
state: New York
city: Manhasset
country: United States
facility: Investigative Site
state: New York
city: New York
country: United States
facility: Investigative Site
state: New York
city: Dallas
country: United States
facility: Investigative Site
state: Texas
city: Fort Worth
country: United States
facility: Investigative Site
state: Texas
city: Houston
country: United States
facility: Investigative Site
state: Texas
city: San Antonio
country: United States
facility: Investigative Site
state: Texas
city: Seattle
country: United States
facility: Investigative Site
state: Washington
city: Liverpool
country: Australia
facility: Investigative Site
state: New South Wales
city: Westmead
country: Australia
facility: Investigative Site
state: New South Wales
city: Brisbane
country: Australia
facility: Investigative Site
state: Queensland
city: Herston
country: Australia
facility: Investigative Site
state: Queensland
city: Melbourne
country: Australia
facility: Investigative Site
state: Victoria
city: Murdoch
country: Australia
facility: Investigative Site
state: Western Australia
city: Bondy
country: France
facility: Investigative Site
city: Orléans
country: France
facility: Investigative Site
city: Pessac
country: France
facility: Investigative Site
city: Rennes Cedex 9
country: France
facility: Investigative Site
city: Bradford
country: United Kingdom
facility: Investigative Site
state: England
city: Newcastle Upon Tyne
country: United Kingdom
facility: Investigative Site
state: England
city: Portsmouth
country: United Kingdom
facility: Investigative Site
state: England
Study officials:
References: