A Phase 2 interventional study of LJPC-401 and LJPC-401 in Beta-Thalassemia, sponsored by La Jolla Pharmaceutical Company. Terminated at 22 sites in 8 countries. Open to participants aged 14 Years and older. Per ClinicalTrials.gov, last updated 2021-07-29.
Sponsored by La Jolla Pharmaceutical Company · Phase 2, Interventional, and Treatment
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Among 49 studies in beta-Thalassemia with a site in Italy — 8 open to participants now, 17 under way, 6 of them phase 3. beta-Thalassemia · Italy.
Among the 49 studies in beta-Thalassemia with a site in Italy, this one is the 14th largest by enrollment of the 39 interventional ones that give a count, is one of 5 that started in 2017, is one of 18 in phase 2, and was the 26th registered.
294 studies on the registry are indexed under beta-Thalassemia; 69 are open to participants now.
Browse beta-Thalassemia studies →8 of the 49 studies in beta-Thalassemia with a site in Italy are open now. Recruiting first, the newest first:
Fondazione Telethon · Sites in Italy
Ages 3–35
Cyprus Institute of Neurology and Genetics · Sites in Italy, United States, Angola and 13 more countries
Ages 2+
Vertex Pharmaceuticals Incorporated · Sites in Italy, United States, Germany and 1 more country
Ages 12–35
Celgene · Sites in Italy, United States, China and 6 more countries
Ages 6–17
Celgene · Sites in Italy, United States, Australia and 19 more countries
Ages 18+
A study can be open while its sites in Italy have not opened yet or have stopped. Only those recruiting at a site in Italy →
Of the 43 beta-Thalassemia studies recruiting now, 10 (23%) set no upper age limit and 31 (72%) stop at 75 or younger; 34 take children under 18; 6 take healthy volunteers.
This study: 14 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.
49 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 21 · Phase 3 13 · Phase 4 4
First in 2000; this study’s year is marked. 5 started in 2017.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 84 is above the median of 40 across 208 interventional studies indexed under beta-Thalassemia.
Median enrollment 55 across the 39 interventional studies, 120 across the 10 observational. Of the 29 that completed or were terminated, 15 (52%) have results posted.
This tab as a report to print or save as PDF → With an account.
29 of 49 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 beta-Thalassemia — fewer than ten, so there is no comparison to draw.
5 of the studies in beta-Thalassemia with a site in Italy that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05477563Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | 2027-06-09 |
| NCT05356195Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | 2027-11-14 |
| NCT06421636Regeneron PharmaceuticalsA Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body | 2026-12-03 |
| NCT01639690San Rocco Therapeuticsß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene | 2027-07 |
| NCT05799118Cyprus Institute of Neurology and GeneticsStudy of the Role of Genetic Modifiers in Hemoglobinopathies | 2027-09-30 |
Add all 5 to your calendar (.ics) → With an account.
Among the 49 studies in beta-Thalassemia with a site in Italy, 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, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with transfusion-dependent beta thalassemia with myocardial iron overload.
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
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.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks
Drug: LJPC-401
standard chelation therapy plus LJPC-401 for 52 weeks
Drug: LJPC-401
subcutaneous injection, up to 20 mg per week from week 26 to 52
Also known as: synthetic human hepcidin
subcutaneous injection, up to 20 mg per week from week 1 to 52
Also known as: synthetic human hepcidin
Effect of LJPC-401 on cardiac iron
Change in cardiac T2\* magnetic resonance imaging (MRI)
Time frame: 26 Weeks
Effect of LJPC-401 on cardiac iron
Change in cardiac T2\*MRI
Time frame: 52 Weeks
Effect of LJPC-401 on hepatic iron
Change in hepatic T2\*MRI
Time frame: 52 Weeks
Effect of LJPC-401 on serum iron
Mean change in serum iron
Time frame: 56 Weeks
Effect of LJPC-401 on hemoglobin
Mean change in hemoglobin
Time frame: 52 Weeks
Effect of LJPC-401 on volume of blood transfused
Change in transfused blood volume
Time frame: 56 Weeks
Effect of LJPC-401 on its potential to elicit an immune response
Measured by blood laboratory tests and the presence of anti-drug antibodies
Time frame: 56 Weeks
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-heart rate
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-body temperature
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-respiratory rate
Time frame: 56 Weeks
Effect of LJPC-401 on vital signs-blood pressure
Time frame: 56 Weeks
Effect of LJPC-401 on body weight
Change in body weight (kilograms)
Time frame: 56 Weeks
Plan to share: No
No publications or documents are linked to this record.
Ran from Nov 2017 to primary completion in Dec 2019, 24 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
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La Jolla Pharmaceutical Company