Hereditary Hemochromatosis
Conditions
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.
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.
DRUGPlacebo
0.9% Sodium Chloride Injection, USP, or equivalent
Sponsors
PRA Health Sciences
La Jolla Pharmaceutical Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | 16 Weeks | Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Number of Phlebotomies | 16 Weeks | — |
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | 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 | 20 Weeks | — |
Countries
Australia, France, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| LJPC-401 LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
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. | 34 |
| Placebo 0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent | 35 |
| Total | 69 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 2 | 1 |
| Overall Study | Physician Decision | 0 | 1 |
| Overall Study | Withdrawal by Subject | 2 | 1 |
Baseline characteristics
| Characteristic | LJPC-401 | Total | Placebo |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 11 Participants | 26 Participants | 15 Participants |
| Age, Categorical Between 18 and 65 years | 23 Participants | 43 Participants | 20 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 2 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 33 Participants | 67 Participants | 34 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| HH Genotype HFE | 1 Participants | 3 Participants | 2 Participants |
| HH Genotype Not Available | 5 Participants | 10 Participants | 5 Participants |
| HH Genotype Other | 28 Participants | 56 Participants | 28 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 34 Participants | 69 Participants | 35 Participants |
| Region of Enrollment Australia | 15 participants | 22 participants | 7 participants |
| Region of Enrollment France | 1 participants | 3 participants | 2 participants |
| Region of Enrollment United Kingdom | 5 participants | 9 participants | 4 participants |
| Region of Enrollment United States | 13 participants | 35 participants | 22 participants |
| Sex: Female, Male Female | 9 Participants | 27 Participants | 18 Participants |
| Sex: Female, Male Male | 25 Participants | 42 Participants | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 34 | 0 / 35 |
| other Total, other adverse events | 33 / 34 | 30 / 35 |
| serious Total, serious adverse events | 2 / 34 | 2 / 35 |
Outcome results
Primary
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
Population: Efficacy Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| LJPC-401 | Effect of LJPC-401 Versus Placebo on Blood Iron Levels | -32.8 Percent Change | Standard Deviation 20.53 |
| Placebo | Effect of LJPC-401 Versus Placebo on Blood Iron Levels | -2.5 Percent Change | Standard Deviation 18.5 |
p-value: <0.0001General Linear Model
Secondary
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Change in serum ferritin as measured by blood laboratory tests
Time frame: 16 Weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| LJPC-401 | Effect of LJPC-401 Versus Placebo on Blood Iron Levels | -18.0 Percent Change | Standard Deviation 25.81 |
| Placebo | Effect of LJPC-401 Versus Placebo on Blood Iron Levels | -18.5 Percent Change | Standard Deviation 21.98 |
Secondary
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Time frame: 16 Weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| LJPC-401 | Effect of LJPC-401 Versus Placebo on Number of Phlebotomies | 0.52 Phlebotomies | Standard Deviation 0.81 |
| Placebo | Effect of LJPC-401 Versus Placebo on Number of Phlebotomies | 2.96 Phlebotomies | Standard Deviation 1.9 |
Secondary
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Time frame: 20 Weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LJPC-401 | Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events | 317 Events |
| Placebo | Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events | 101 Events |