A Phase 2 interventional study of LJPC-401 and Placebo in Hereditary Hemochromatosis, sponsored by La Jolla Pharmaceutical Company. Completed at 31 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-06-09.
Sponsored by La Jolla Pharmaceutical Company · Phase 2, Interventional, and Treatment
Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →
Among 16 studies in Hemochromatosis with a site in United States — none open to participants now, 2 under way, none of them phase 3. Hemochromatosis · United States.
Among the 16 studies in Hemochromatosis with a site in United States, this one is the only one that started in 2017, is one of 4 in phase 2, and was the 13th registered.
55 studies on the registry are indexed under Hemochromatosis; 4 are open to participants now.
Browse Hemochromatosis studies →None of the 16 studies in Hemochromatosis with a site in United States is open now. The open studies under Hemochromatosis, anywhere →
Of the 4 Hemochromatosis studies recruiting now, 2 set no upper age limit and 2 stop at 75 or younger; 1 takes children under 18; 2 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
16 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 5 · Phase 3 1
First in 1985; this study’s year is marked. 1 started in 2017.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 70 is above the median of 61 across 30 interventional studies indexed under Hemochromatosis.
Median enrollment 77 across the 8 interventional studies, 67 across the 8 observational. Of the 14 that completed or were terminated, 5 (36%) have results posted.
This tab as a report to print or save as PDF → With an account.
4 of 16 have an industry lead sponsor. The 10 most frequent:
La Jolla Pharmaceutical Company is the lead sponsor of 18 studies on the registry; none are open to participants now.
La Jolla Pharmaceutical Company has 1 study filed under Hemochromatosis — fewer than ten, so there is no comparison to draw.
1 of the studies in Hemochromatosis with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT00007150National Institutes of Health Clinical Center (CC)Treatment of Hemochromatosis | 2026-12-31 |
Add it to your calendar (.ics) → With an account.
Among the 16 studies in Hemochromatosis with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
Drug: LJPC-401
0.9% Sodium Chloride Injection, USP, or equivalent
Drug: Placebo
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.
Also known as: synthetic human hepcidin
0.9% Sodium Chloride Injection, USP, or equivalent
Also known as: Normal saline
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
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
Time frame: 16 Weeks
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
Change in serum ferritin as measured by blood laboratory tests
Time frame: 16 Weeks
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
Time frame: 20 Weeks
| Milestone | LJPC-401 | Placebo |
|---|---|---|
| Started | 34 | 36 |
| Safety population | 34 | 35 |
| Analysis population | 21 | 28 |
| Completed | 30 | 33 |
| Not completed | 4 | 3 |
| Withdrew: Adverse event | 2 | 1 |
| Withdrew: Withdrawal by subject | 2 | 1 |
| Withdrew: Physician decision | 0 | 1 |
Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.
| Percent Change | LJPC-401 | Placebo |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | -32.8 ± 20.53 | -2.5 ± 18.50 |
| Phlebotomies | LJPC-401 | Placebo |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Number of Phlebotomies | 0.52 ± 0.81 | 2.96 ± 1.90 |
Change in serum ferritin as measured by blood laboratory tests
| Percent Change | LJPC-401 | Placebo |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on Blood Iron Levels | -18.0 ± 25.81 | -18.5 ± 21.98 |
| Events | LJPC-401 | Placebo |
|---|---|---|
| Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events | 317 | 101 |
Collected over 20 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| LJPC-401 | 0/34 (0%) | 2/34 (5.9%) | 33/34 (97.1%) |
| Placebo | 0/35 (0%) | 2/35 (5.7%) | 30/35 (85.7%) |
| Event | LJPC-401 | Placebo |
|---|---|---|
| HyponatraemiaMetabolism and nutrition disorders | 1/34 | 0/35 |
| Arteriospasm coronaryCardiac disorders | 1/34 | 0/35 |
| HypertensionVascular disorders | 1/34 | 0/35 |
| SeizureNervous system disorders | 0/34 | 1/35 |
| Acute myocardial infarctionCardiac disorders | 0/34 | 1/35 |
| Hernia painGeneral disorders | 0/34 | 1/35 |
| Event | LJPC-401 | Placebo |
|---|---|---|
| Injection site erythemaGeneral disorders | 17/34 | 0/35 |
| Injection site pruritusGeneral disorders | 14/34 | 1/35 |
| Injection site painGeneral disorders | 8/34 | 0/35 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 6/34 | 4/35 |
| Injection site bruisingGeneral disorders | 6/34 | 0/35 |
| DiarrhoeaGastrointestinal disorders | 5/34 | 4/35 |
| FatigueGeneral disorders | 2/34 | 5/35 |
| NauseaGastrointestinal disorders | 4/34 | 1/35 |
| PruritisSkin and subcutaneous tissue disorders | 4/34 | 1/35 |
| Back painMusculoskeletal and connective tissue disorders | 4/34 | 2/35 |
Safety Population
| Age, Categorical(Participants) | LJPC-401 | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 23 | 20 | 43 |
| >=65 years | 11 | 15 | 26 |
| Sex: Female, Male(Participants) | LJPC-401 | Placebo | Total |
|---|---|---|---|
| Female | 9 | 18 | 27 |
| Male | 25 | 17 | 42 |
| Ethnicity (NIH/OMB)(Participants) | LJPC-401 | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 2 |
| Not Hispanic or Latino | 33 | 34 | 67 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | LJPC-401 | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 34 | 35 | 69 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | LJPC-401 | Placebo | Total |
|---|---|---|---|
| United States | 13 | 22 | 35 |
| United Kingdom | 5 | 4 | 9 |
| Australia | 15 | 7 | 22 |
| France | 1 | 2 | 3 |
| HH Genotype(Participants) | LJPC-401 | Placebo | Total |
|---|---|---|---|
| HFE | 1 | 2 | 3 |
| Not Available | 5 | 5 | 10 |
| Other | 28 | 28 | 56 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Nov 2017 to primary completion in Oct 2019, 22 months.
Results were first posted in Jun 2022, 2 years 7 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2022. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
La Jolla Pharmaceutical Company