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| Name | Class |
|---|---|
| PRA Health Sciences | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LJPC-401 | Active Comparator | LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial |
|
| Placebo | Placebo Comparator | 0.9% Sodium Chloride Injection, USP, or equivalent |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LJPC-401 | Drug | 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests. | 16 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Number of Phlebotomies | 16 Weeks | |
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | Change in serum ferritin as measured by blood laboratory tests | 16 Weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site | North Little Rock | Arkansas | 72117 | United States | ||
| Investigative Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 14, 2018 | Dec 9, 2021 |
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|
| Placebo | Drug | 0.9% Sodium Chloride Injection, USP, or equivalent |
|
|
| Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events | 20 Weeks |
| 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 | 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 |
| Placebo |
0.9% Sodium Chloride Injection, USP, or equivalent Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent |
| Safety Population | The Safety Population included all patients who received at least one dose of study drug. |
|
| Analysis Population | 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. |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
Safety Population
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | Placebo | 0.9% Sodium Chloride Injection, USP, or equivalent Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| HH Genotype | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Effect of LJPC-401 Versus Placebo on Blood Iron Levels | Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests. | Efficacy Population | Posted | Mean | Standard Deviation | Percent Change | 16 Weeks |
|
|
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| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Effect of LJPC-401 Versus Placebo on Number of Phlebotomies | Posted | Mean | Standard Deviation | Phlebotomies | 16 Weeks |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Effect of LJPC-401 Versus Placebo on Blood Iron Levels | Change in serum ferritin as measured by blood laboratory tests | Posted | Mean | Standard Deviation | Percent Change | 16 Weeks |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events | Posted | Number | Events | 20 Weeks |
|
|
20 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. | 0 | 34 | 2 | 34 | 33 | 34 |
| EG001 | Placebo | 0.9% Sodium Chloride Injection, USP, or equivalent Placebo: 0.9% Sodium Chloride Injection, USP, or equivalent | 0 | 35 | 2 | 35 | 30 | 35 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hyponatraemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Acute myocardial infarction | Cardiac disorders | Systematic Assessment |
| ||
| Arteriospasm coronary | Cardiac disorders | Systematic Assessment |
| ||
| Hypertension | Vascular disorders | Systematic Assessment |
| ||
| Hernia pain | General disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Rhinitis | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection | Infections and infestations | Systematic Assessment |
| ||
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Dizziness | Nervous system disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Presyncope | Nervous system disorders | Systematic Assessment |
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| Hypertension | Vascular disorders | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Abdominal pain upper | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pruritis | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
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| Influenza like illness | General disorders | Systematic Assessment |
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| Injection site bruising | General disorders | Systematic Assessment |
| ||
| Injection site erythema | General disorders | Systematic Assessment |
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| Injection site haemorrhage | General disorders | Systematic Assessment |
| ||
| Injection site induration | General disorders | Systematic Assessment |
| ||
| Injection site mass | General disorders | Systematic Assessment |
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| Injection site nodule | General disorders | Systematic Assessment |
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| Injection site pain | General disorders | Systematic Assessment |
| ||
| Injection site pruritus | General disorders | Systematic Assessment |
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| Injection site rash | General disorders | Systematic Assessment |
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| Injection site reaction | General disorders | Systematic Assessment |
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| Injection site swelling | General disorders | Systematic Assessment |
| ||
| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
Disclosure was restricted for all data, unless published by the company.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Stew Kroll, MA | La Jolla Pharmaceutical Company | 650-576-9679 | skroll@ljpc.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 22, 2019 | Nov 2, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006432 | Hemochromatosis |
| ID | Term |
|---|---|
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D019190 | Iron Overload |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D000077330 | Saline Solution |
| ID | Term |
|---|---|
| D000077324 | Crystalloid Solutions |
| D007552 | Isotonic Solutions |
| D012996 | Solutions |
| D004364 | Pharmaceutical Preparations |
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| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| United Kingdom |
|
| Australia |
|
| France |
|
| Not Available |
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| Other |
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