We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03395704
Recruitment Status : Completed
First Posted : January 10, 2018
Results First Posted : June 9, 2022
Last Update Posted : June 9, 2022
Sponsor:
Collaborator:
PRA Health Sciences
Information provided by (Responsible Party):
La Jolla Pharmaceutical Company

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Single (Participant);   Primary Purpose: Treatment
Condition Hereditary Hemochromatosis
Interventions Drug: LJPC-401
Drug: Placebo
Enrollment 70
Recruitment Details  
Pre-assignment Details  
Arm/Group Title LJPC-401 Placebo
Hide Arm/Group Description

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

Period Title: Overall Study
Started 34 36
Safety Population [1] 34 35
Analysis Population [2] 21 28
Completed 30 33
Not Completed 4 3
Reason Not Completed
Adverse Event             2             1
Withdrawal by Subject             2             1
Physician Decision             0             1
[1]
The Safety Population included all patients who received at least one dose of study drug.
[2]
The Analysis Population included enrolled patients under global protocol Version 4.0 or later, as randomized, who received study drug, and who completed 4 months (Week 16) on study or who had at least 1 phlebotomy after the SOC therapeutic phlebotomy on Day 1 (predose) and before Week 16.
Arm/Group Title LJPC-401 Placebo Total
Hide Arm/Group Description

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

Total of all reporting groups
Overall Number of Baseline Participants 34 35 69
Hide Baseline Analysis Population Description
Safety Population
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 35 participants 69 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
23
  67.6%
20
  57.1%
43
  62.3%
>=65 years
11
  32.4%
15
  42.9%
26
  37.7%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 35 participants 69 participants
Female
9
  26.5%
18
  51.4%
27
  39.1%
Male
25
  73.5%
17
  48.6%
42
  60.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 35 participants 69 participants
Hispanic or Latino
1
   2.9%
1
   2.9%
2
   2.9%
Not Hispanic or Latino
33
  97.1%
34
  97.1%
67
  97.1%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 35 participants 69 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
34
 100.0%
35
 100.0%
69
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 34 participants 35 participants 69 participants
United States 13 22 35
United Kingdom 5 4 9
Australia 15 7 22
France 1 2 3
HH Genotype  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 35 participants 69 participants
HFE
1
   2.9%
2
   5.7%
3
   4.3%
Not Available
5
  14.7%
5
  14.3%
10
  14.5%
Other
28
  82.4%
28
  80.0%
56
  81.2%
1.Primary Outcome
Title Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Hide Description Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Time Frame 16 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Population
Arm/Group Title LJPC-401 Placebo
Hide Arm/Group Description:

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

Overall Number of Participants Analyzed 21 28
Mean (Standard Deviation)
Unit of Measure: Percent Change
-32.8  (20.53) -2.5  (18.50)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection LJPC-401, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method General Linear Model
Comments [Not Specified]
2.Secondary Outcome
Title Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Hide Description [Not Specified]
Time Frame 16 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title LJPC-401 Placebo
Hide Arm/Group Description:

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

Overall Number of Participants Analyzed 21 28
Mean (Standard Deviation)
Unit of Measure: Phlebotomies
0.52  (0.81) 2.96  (1.90)
3.Secondary Outcome
Title Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Hide Description Change in serum ferritin as measured by blood laboratory tests
Time Frame 16 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title LJPC-401 Placebo
Hide Arm/Group Description:

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

Overall Number of Participants Analyzed 21 28
Mean (Standard Deviation)
Unit of Measure: Percent Change
-18.0  (25.81) -18.5  (21.98)
4.Secondary Outcome
Title Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Hide Description [Not Specified]
Time Frame 20 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title LJPC-401 Placebo
Hide Arm/Group Description:

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

Overall Number of Participants Analyzed 34 35
Measure Type: Number
Unit of Measure: Events
317 101
Time Frame 20 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title LJPC-401 Placebo
Hide Arm/Group Description

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.

0.9% Sodium Chloride Injection, USP, or equivalent

Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent

All-Cause Mortality
LJPC-401 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/34 (0.00%)      0/35 (0.00%)    
Hide Serious Adverse Events
LJPC-401 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/34 (5.88%)      2/35 (5.71%)    
Cardiac disorders     
Acute myocardial infarction   0/34 (0.00%)  0 1/35 (2.86%)  1
Arteriospasm coronary   1/34 (2.94%)  1 0/35 (0.00%)  0
General disorders     
Hernia pain   0/34 (0.00%)  0 1/35 (2.86%)  1
Metabolism and nutrition disorders     
Hyponatraemia   1/34 (2.94%)  1 0/35 (0.00%)  0
Nervous system disorders     
Seizure   0/34 (0.00%)  0 1/35 (2.86%)  1
Vascular disorders     
Hypertension   1/34 (2.94%)  1 0/35 (0.00%)  0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
LJPC-401 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   33/34 (97.06%)      30/35 (85.71%)    
Gastrointestinal disorders     
Abdominal pain upper   0/34 (0.00%)  0 2/35 (5.71%)  2
Diarrhoea   5/34 (14.71%)  10 4/35 (11.43%)  5
Nausea   4/34 (11.76%)  7 1/35 (2.86%)  1
General disorders     
Fatigue   2/34 (5.88%)  4 5/35 (14.29%)  5
Influenza like illness   0/34 (0.00%)  0 2/35 (5.71%)  2
Injection site bruising   6/34 (17.65%)  7 0/35 (0.00%)  0
Injection site erythema   17/34 (50.00%)  56 0/35 (0.00%)  0
Injection site haemorrhage   0/34 (0.00%)  0 2/35 (5.71%)  2
Injection site induration   2/34 (5.88%)  7 0/35 (0.00%)  0
Injection site mass   3/34 (8.82%)  7 0/35 (0.00%)  0
Injection site nodule   3/34 (8.82%)  3 0/35 (0.00%)  0
Injection site pain   8/34 (23.53%)  40 0/35 (0.00%)  0
Injection site pruritus   14/34 (41.18%)  50 1/35 (2.86%)  1
Injection site rash   2/34 (5.88%)  2 0/35 (0.00%)  0
Injection site reaction   4/34 (11.76%)  29 0/35 (0.00%)  0
Injection site swelling   2/34 (5.88%)  6 0/35 (0.00%)  0
Infections and infestations     
Nasopharyngitis   3/34 (8.82%)  3 1/35 (2.86%)  1
Rhinitis   1/34 (2.94%)  1 3/35 (8.57%)  4
Upper respiratory tract infection   3/34 (8.82%)  3 3/35 (8.57%)  3
Urinary tract infection   0/34 (0.00%)  0 3/35 (8.57%)  4
Injury, poisoning and procedural complications     
Contusion   0/34 (0.00%)  0 2/35 (5.71%)  2
Metabolism and nutrition disorders     
Type 2 diabetes mellitus   0/34 (0.00%)  0 2/35 (5.71%)  2
Musculoskeletal and connective tissue disorders     
Arthralgia   6/34 (17.65%)  8 4/35 (11.43%)  6
Back pain   4/34 (11.76%)  4 2/35 (5.71%)  2
Muscle spasms   2/34 (5.88%)  2 1/35 (2.86%)  1
Pain in extremity   2/34 (5.88%)  2 1/35 (2.86%)  1
Nervous system disorders     
Dizziness   2/34 (5.88%)  2 0/35 (0.00%)  0
Headache   2/34 (5.88%)  2 3/35 (8.57%)  3
Presyncope   0/34 (0.00%)  0 2/35 (5.71%)  3
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain   2/34 (5.88%)  2 1/35 (2.86%)  1
Skin and subcutaneous tissue disorders     
Pruritis   4/34 (11.76%)  4 1/35 (2.86%)  1
Rash   2/34 (5.88%)  3 1/35 (2.86%)  1
Vascular disorders     
Hypertension   3/34 (8.82%)  3 2/35 (5.71%)  2
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Disclosure was restricted for all data, unless published by the company.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Stew Kroll, MA
Organization: La Jolla Pharmaceutical Company
Phone: 650-576-9679
EMail: skroll@ljpc.com
Layout table for additonal information
Responsible Party: La Jolla Pharmaceutical Company
ClinicalTrials.gov Identifier: NCT03395704    
Other Study ID Numbers: LJ401-HH01
First Submitted: December 12, 2017
First Posted: January 10, 2018
Results First Submitted: December 15, 2021
Results First Posted: June 9, 2022
Last Update Posted: June 9, 2022