Trial Outcomes & Findings for A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis (NCT NCT03395704)
NCT ID: NCT03395704
Last Updated: 2022-06-09
Results Overview
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
70 participants
Primary outcome timeframe
16 Weeks
Results posted on
2022-06-09
Participant Flow
Participant milestones
| Measure |
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.
|
Placebo
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Overall Study
STARTED
|
34
|
36
|
|
Overall Study
Safety Population
|
34
|
35
|
|
Overall Study
Analysis Population
|
21
|
28
|
|
Overall Study
COMPLETED
|
30
|
33
|
|
Overall Study
NOT COMPLETED
|
4
|
3
|
Reasons for withdrawal
| Measure |
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.
|
Placebo
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Overall Study
Adverse Event
|
2
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
1
|
|
Overall Study
Physician Decision
|
0
|
1
|
Baseline Characteristics
A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
Baseline characteristics by cohort
| Measure |
LJPC-401
n=34 Participants
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.
|
Placebo
n=35 Participants
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
Total
n=69 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
23 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
25 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
67 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
34 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
69 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=5 Participants
|
22 participants
n=7 Participants
|
35 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
5 participants
n=5 Participants
|
4 participants
n=7 Participants
|
9 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
15 participants
n=5 Participants
|
7 participants
n=7 Participants
|
22 participants
n=5 Participants
|
|
Region of Enrollment
France
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
HH Genotype
HFE
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
HH Genotype
Not Available
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
HH Genotype
Other
|
28 Participants
n=5 Participants
|
28 Participants
n=7 Participants
|
56 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 16 WeeksPopulation: Efficacy Population
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Outcome measures
| Measure |
LJPC-401
n=21 Participants
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.
|
Placebo
n=28 Participants
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
|
-32.8 Percent Change
Standard Deviation 20.53
|
-2.5 Percent Change
Standard Deviation 18.50
|
SECONDARY outcome
Timeframe: 16 WeeksOutcome measures
| Measure |
LJPC-401
n=21 Participants
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.
|
Placebo
n=28 Participants
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
|
0.52 Phlebotomies
Standard Deviation 0.81
|
2.96 Phlebotomies
Standard Deviation 1.90
|
SECONDARY outcome
Timeframe: 16 WeeksChange in serum ferritin as measured by blood laboratory tests
Outcome measures
| Measure |
LJPC-401
n=21 Participants
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.
|
Placebo
n=28 Participants
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
|
-18.0 Percent Change
Standard Deviation 25.81
|
-18.5 Percent Change
Standard Deviation 21.98
|
SECONDARY outcome
Timeframe: 20 WeeksOutcome measures
| Measure |
LJPC-401
n=34 Participants
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.
|
Placebo
n=35 Participants
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
|
317 Events
|
101 Events
|
Adverse Events
LJPC-401
Serious events: 2 serious events
Other events: 33 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 30 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
LJPC-401
n=34 participants at risk
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.
|
Placebo
n=35 participants at risk
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Metabolism and nutrition disorders
Hyponatraemia
|
2.9%
1/34 • Number of events 1 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
Nervous system disorders
Seizure
|
0.00%
0/34 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Cardiac disorders
Acute myocardial infarction
|
0.00%
0/34 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Cardiac disorders
Arteriospasm coronary
|
2.9%
1/34 • Number of events 1 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
Vascular disorders
Hypertension
|
2.9%
1/34 • Number of events 1 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Hernia pain
|
0.00%
0/34 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
Other adverse events
| Measure |
LJPC-401
n=34 participants at risk
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.
|
Placebo
n=35 participants at risk
0.9% Sodium Chloride Injection, USP, or equivalent
Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
8.8%
3/34 • Number of events 3 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Infections and infestations
Rhinitis
|
2.9%
1/34 • Number of events 1 • 20 weeks
|
8.6%
3/35 • Number of events 4 • 20 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
8.8%
3/34 • Number of events 3 • 20 weeks
|
8.6%
3/35 • Number of events 3 • 20 weeks
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/34 • 20 weeks
|
8.6%
3/35 • Number of events 4 • 20 weeks
|
|
Metabolism and nutrition disorders
Type 2 diabetes mellitus
|
0.00%
0/34 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
Nervous system disorders
Dizziness
|
5.9%
2/34 • Number of events 2 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
Nervous system disorders
Headache
|
5.9%
2/34 • Number of events 2 • 20 weeks
|
8.6%
3/35 • Number of events 3 • 20 weeks
|
|
Nervous system disorders
Presyncope
|
0.00%
0/34 • 20 weeks
|
5.7%
2/35 • Number of events 3 • 20 weeks
|
|
Vascular disorders
Hypertension
|
8.8%
3/34 • Number of events 3 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.9%
2/34 • Number of events 2 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/34 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
14.7%
5/34 • Number of events 10 • 20 weeks
|
11.4%
4/35 • Number of events 5 • 20 weeks
|
|
Gastrointestinal disorders
Nausea
|
11.8%
4/34 • Number of events 7 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
11.8%
4/34 • Number of events 4 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
17.6%
6/34 • Number of events 8 • 20 weeks
|
11.4%
4/35 • Number of events 6 • 20 weeks
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
11.8%
4/34 • Number of events 4 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.9%
2/34 • Number of events 2 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.9%
2/34 • Number of events 2 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
General disorders
Fatigue
|
5.9%
2/34 • Number of events 4 • 20 weeks
|
14.3%
5/35 • Number of events 5 • 20 weeks
|
|
General disorders
Influenza like illness
|
0.00%
0/34 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
General disorders
Injection site bruising
|
17.6%
6/34 • Number of events 7 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site erythema
|
50.0%
17/34 • Number of events 56 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site haemorrhage
|
0.00%
0/34 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
General disorders
Injection site induration
|
5.9%
2/34 • Number of events 7 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site mass
|
8.8%
3/34 • Number of events 7 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site nodule
|
8.8%
3/34 • Number of events 3 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site pain
|
23.5%
8/34 • Number of events 40 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site pruritus
|
41.2%
14/34 • Number of events 50 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
|
General disorders
Injection site rash
|
5.9%
2/34 • Number of events 2 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site reaction
|
11.8%
4/34 • Number of events 29 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
General disorders
Injection site swelling
|
5.9%
2/34 • Number of events 6 • 20 weeks
|
0.00%
0/35 • 20 weeks
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/34 • 20 weeks
|
5.7%
2/35 • Number of events 2 • 20 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.9%
2/34 • Number of events 3 • 20 weeks
|
2.9%
1/35 • Number of events 1 • 20 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Disclosure was restricted for all data, unless published by the company.
- Publication restrictions are in place
Restriction type: OTHER