Hereditary HemochromatosisPhase 2NCT03395704
A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
A Phase 2 study testing LJPC-401 in people with Hereditary Hemochromatosis, run by La Jolla Pharmaceutical Company at 31 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemochromatosis
- What is being tested?
- LJPC-401
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 31 sitesin 4 countries
- Study dates
- 29 Nov 2017to 28 Oct 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with clinical diagnosis of hereditary hemochromatosis
- Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis
- Patients with serum ferritin and TSAT levels above treatment guidelines
- 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
- 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
- Patient must be willing and able to provide written informed consent
Who cannot join
- Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug
- Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug
- Pregnant or lactating women
- Patients taking an immunosuppressive agent without prior Sponsor approval
- Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug
- Patients who are unwilling or unable to comply with the study protocol requirements
- Patients with type 1 or poorly controlled type 2 diabetes
- 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
Trial locations and contacts
31 sites in 4 countries.
31 sites in 4 countries
United States18 sites
Investigative Site
Dallas, Texas, 75246
Investigative Site
East Setauket, New York, 11733
Investigative Site
Fort Worth, Texas, 76104
Investigative Site
Houston, Texas, 77058
Investigative Site
Indianapolis, Indiana, 46202
Investigative Site
Jackson, Mississippi, 39216
Investigative Site
Jacksonville, Florida, 32204
Investigative Site
Los Angeles, California, 90036
Investigative Site
Manhasset, New York, 11030
Investigative Site
New York, New York, 10029
Investigative Site
North Little Rock, Arkansas, 72117
Investigative Site
Palo Alto, California, 94305
Investigative Site
Rialto, California, 92377
Investigative Site
San Antonio, Texas, 78215
Investigational Site
San Diego, California, 91942
Investigative Site
San Francisco, California, 94115
Investigative Site
Seattle, Washington, 98104
Investigative Site
Wyoming, Michigan, 49519
Australia6 sites
Investigative Site
Brisbane, Queensland, 4120
Investigative Site
Herston, Queensland, 4029
Investigative Site
Liverpool, New South Wales, 2170
Investigative Site
Melbourne, Victoria, 3004
Investigative Site
Murdoch, Western Australia, 6150
Investigative Site
Westmead, New South Wales, 2145
France4 sites
Investigative Site
Bondy, 93140
Investigative Site
Orléans, 45000
Investigative Site
Pessac, 33604
Investigative Site
Rennes, 35033
United Kingdom3 sites
Investigative Site
Bradford, England, BD9 6RJ
Investigative Site
Newcastle upon Tyne, England, NE7 7DN
Investigative Site
Portsmouth, England, PO6 3LY
About this study
Official title: A Phase 2, Multi-Center, Randomized, Placebo Controlled, Single-Blind Study With LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
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.
- Conditions
- Hereditary Hemochromatosis
- Being tested
- LJPC-401, Placebo
- Medical subject headings
- Hemochromatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LJPC-401Drug
Also called synthetic human hepcidin
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.
PlaceboDrug
Also called Normal saline
0.9% Sodium Chloride Injection, USP, or equivalent
| Study group | Role | What they receive |
|---|---|---|
| LJPC-401 | Active comparator | LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial Receives: Drug: LJPC-401 |
| Placebo | Placebo comparator | 0.9% Sodium Chloride Injection, USP, or equivalent Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
Measured: 16 Weeks
Secondary outcomes (3)
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Measured: 16 Weeks
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Change in serum ferritin as measured by blood laboratory tests
Measured: 16 Weeks
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Measured: 20 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2018
- Study start29 Nov 2017
- Primary completion28 Oct 2019
- Study completion28 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hereditary HemochromatosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- La Jolla Pharmaceutical Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PRA Health Sciences
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03395704
- Sponsor's protocol ID
- LJ401-HH01
- Status last verified
- May 2022
- First submitted
- 12 Dec 2017
- Last updated
- 9 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multi-Center, Randomized, Placebo Controlled, Single-Blind Study With LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
- ClinicalTrials.gov ID
- NCT03395704
- Acronym / sponsor ID
- LJ401-HH01
- Conditions
- Hereditary Hemochromatosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- La Jolla Pharmaceutical CompanyIndustry (pharma, biotech, devices)
- Collaborators
- PRA Health Sciences
- Study start
- 29 Nov 2017 (actual)
- Primary completion
- 28 Oct 2019 (actual)
- Study completion
- 28 Oct 2019 (actual)
- First posted
- 10 Jan 2018 (actual)
- Results
- Posted 9 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LJPC-401 | Active comparator | Drug: LJPC-401LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial |
| Placebo | Placebo comparator | Drug: Placebo0.9% Sodium Chloride Injection, USP, or equivalent |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients with clinical diagnosis of hereditary hemochromatosis
- Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis
- Patients with serum ferritin and TSAT levels above treatment guidelines
- 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
- 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
Key exclusion criteria (8 listed)
- Patients receiving iron chelation therapy within 7 days prior to the first dose of study drug
- Patients initiating phlebotomy treatments less than 3 months prior to the first dose of study drug
- Pregnant or lactating women
- Patients taking an immunosuppressive agent without prior Sponsor approval
- Patients participating in an unapproved investigational drug or investigational therapeutic device within 30 days of study drug
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LJPC-401Also: synthetic human hepcidinLJPC-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. | Drug | LJPC-401 |
| PlaceboAlso: Normal saline0.9% Sodium Chloride Injection, USP, or equivalent | Drug | Placebo |
Primary outcome
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Time frame: 16 Weeks
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
3 secondary outcomes
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Time frame: 16 Weeks
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Time frame: 16 Weeks
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Time frame: 20 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
31 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LJPC-401 in people with Hereditary Hemochromatosis, run by La Jolla Pharmaceutical Company at 31 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, Australia, France, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of LJPC-401 Versus Placebo on Blood Iron Levels (time frame: 16 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jun 2022.