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CompletedNCT03395704Updated Jun 9, 2022Results posted

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

A Phase 2 interventional study of LJPC-401 and Placebo in Hereditary Hemochromatosis, sponsored by La Jolla Pharmaceutical Company. Completed at 31 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-06-09.

Sponsored by La Jolla Pharmaceutical Company · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
70
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Hereditary Hemochromatosis
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Single (Participant)
Enrollment
70 participants (actual)

Study arms

  • 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

  • Placebo comparator
    Placebo

    0.9% Sodium Chloride Injection, USP, or equivalent

    Drug: Placebo

Interventions

  • DrugLJPC-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.

    Also known as: synthetic human hepcidin

  • DrugPlacebo

    0.9% Sodium Chloride Injection, USP, or equivalent

    Also known as: Normal saline

05

What researchers measure

Primary outcomes

  1. Effect of LJPC-401 Versus Placebo on Blood Iron Levels

    Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.

    Time frame: 16 Weeks

Secondary outcomes

  1. Effect of LJPC-401 Versus Placebo on Number of Phlebotomies

    Time frame: 16 Weeks

  2. Effect of LJPC-401 Versus Placebo on Blood Iron Levels

    Change in serum ferritin as measured by blood laboratory tests

    Time frame: 16 Weeks

  3. Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events

    Time frame: 20 Weeks

06

Results

Posted Jun 9, 2022

Participant flow

Participant flow — Overall Study
MilestoneLJPC-401Placebo
Started3436
Safety population3435
Analysis population2128
Completed3033
Not completed43
Withdrew: Adverse event21
Withdrew: Withdrawal by subject21
Withdrew: Physician decision01

Outcome measures

PrimaryEffect of LJPC-401 Versus Placebo on Blood Iron Levels

Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.

Time frame:
16 Weeks
Reported as:
Mean · Percent Change
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Percent ChangeLJPC-401Placebo
Effect of LJPC-401 Versus Placebo on Blood Iron Levels-32.8 ± 20.53-2.5 ± 18.50
Statistical analysis
  • LJPC-401 vs Placebo · General Linear Model · p = < 0.0001
SecondaryEffect of LJPC-401 Versus Placebo on Number of Phlebotomies
Time frame:
16 Weeks
Reported as:
Mean · Phlebotomies
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
PhlebotomiesLJPC-401Placebo
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies0.52 ± 0.812.96 ± 1.90
SecondaryEffect of LJPC-401 Versus Placebo on Blood Iron Levels

Change in serum ferritin as measured by blood laboratory tests

Time frame:
16 Weeks
Reported as:
Mean · Percent Change
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Percent ChangeLJPC-401Placebo
Effect of LJPC-401 Versus Placebo on Blood Iron Levels-18.0 ± 25.81-18.5 ± 21.98
SecondaryEffect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Time frame:
20 Weeks
Reported as:
Number · Events
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
EventsLJPC-401Placebo
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events317101

Adverse events

Collected over 20 weeks. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
LJPC-4010/34 (0%)2/34 (5.9%)33/34 (97.1%)
Placebo0/35 (0%)2/35 (5.7%)30/35 (85.7%)
Most frequent serious events
Most frequent serious events
EventLJPC-401Placebo
HyponatraemiaMetabolism and nutrition disorders1/340/35
Arteriospasm coronaryCardiac disorders1/340/35
HypertensionVascular disorders1/340/35
SeizureNervous system disorders0/341/35
Acute myocardial infarctionCardiac disorders0/341/35
Hernia painGeneral disorders0/341/35
Most frequent other events
Showing 10 of 33
Most frequent other events
EventLJPC-401Placebo
Injection site erythemaGeneral disorders17/340/35
Injection site pruritusGeneral disorders14/341/35
Injection site painGeneral disorders8/340/35
ArthralgiaMusculoskeletal and connective tissue disorders6/344/35
Injection site bruisingGeneral disorders6/340/35
DiarrhoeaGastrointestinal disorders5/344/35
FatigueGeneral disorders2/345/35
NauseaGastrointestinal disorders4/341/35
PruritisSkin and subcutaneous tissue disorders4/341/35
Back painMusculoskeletal and connective tissue disorders4/342/35

Baseline characteristics

Safety Population

Age, Categorical
Age, Categorical(Participants)LJPC-401PlaceboTotal
<=18 years000
Between 18 and 65 years232043
>=65 years111526
Sex: Female, Male
Sex: Female, Male(Participants)LJPC-401PlaceboTotal
Female91827
Male251742
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)LJPC-401PlaceboTotal
Hispanic or Latino112
Not Hispanic or Latino333467
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)LJPC-401PlaceboTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American000
White343569
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)LJPC-401PlaceboTotal
United States132235
United Kingdom549
Australia15722
France123
HH Genotype
HH Genotype(Participants)LJPC-401PlaceboTotal
HFE123
Not Available5510
Other282856
07

Study locations

31 sites
  • Investigative Site
    North Little Rock, Arkansas 72117, United States
  • Investigative Site
    Los Angeles, California 90036, United States
  • Investigative Site
    Palo Alto, California 94305, United States
  • Investigative Site
    Rialto, California 92377, United States
  • Investigational Site
    San Diego, California 91942, United States
  • Investigative Site
    San Francisco, California 94115, United States
  • Investigative Site
    Jacksonville, Florida 32204, United States
  • Investigative Site
    Indianapolis, Indiana 46202, United States
  • Investigative Site
    Wyoming, Michigan 49519, United States
  • Investigative Site
    Jackson, Mississippi 39216, United States
  • Investigative Site
    East Setauket, New York 11733, United States
  • Investigative Site
    Manhasset, New York 11030, United States
  • Investigative Site
    New York, New York 10029, United States
  • Investigative Site
    Dallas, Texas 75246, United States
  • Investigative Site
    Fort Worth, Texas 76104, United States
  • Investigative Site
    Houston, Texas 77058, United States
  • Investigative Site
    San Antonio, Texas 78215, United States
  • Investigative Site
    Seattle, Washington 98104, United States
  • Investigative Site
    Liverpool, New South Wales 2170, Australia
  • Investigative Site
    Westmead, New South Wales 2145, Australia
  • Investigative Site
    Brisbane, Queensland 4120, Australia
  • Investigative Site
    Herston, Queensland 4029, Australia
  • Investigative Site
    Melbourne, Victoria 3004, Australia
  • Investigative Site
    Murdoch, Western Australia 6150, Australia
  • Investigative Site
    Bondy, 93140, France
  • Investigative Site
    Orléans, 45000, France
  • Investigative Site
    Pessac, 33604, France
  • Investigative Site
    Rennes Cedex 9, 35033, France
  • Investigative Site
    Bradford, England BD9 6RJ, United Kingdom
  • Investigative Site
    Newcastle Upon Tyne, England NE7 7DN, United Kingdom
  • Investigative Site
    Portsmouth, England PO6 3LY, United Kingdom
08

References and documents

Study documents

  • Study protocol · Dec 14, 2018
  • Statistical analysis plan · Mar 22, 2019

Documents are hosted by the registry — open the source record to download them.

09

Registry details

Key details

Study ID
NCT03395704
Lead sponsor
La Jolla Pharmaceutical Company
Collaborators
PRA Health Sciences
Responsible party
Sponsor
First posted
Jan 10, 2018
Start date
Nov 29, 2017
Primary completion
Oct 28, 2019
Completion
Oct 28, 2019
Results posted
Jun 9, 2022
Last update
Jun 9, 2022

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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