Beta-ThalassemiaPhase 2NCT03381833
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
A Phase 2 study testing LJPC-401 in people with Beta-Thalassemia, run by La Jolla Pharmaceutical Company at 22 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- LJPC-401
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 84 participants
- Where
- 22 sitesin 8 countries
- Study dates
- 30 Nov 2017to 14 Jan 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients ≥ 14 years of age with transfusion-dependent beta thalassemia.
- Patients must have increased iron levels in the heart as measured by magnetic resonance imaging (MRI). Two separate cardiac T2*MRI from 6 to 35 msec; <= 15% difference between the two.
- Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose prior to study and expected to remain stable during study.
- Female patients of childbearing potential must not be pregnant, must have negative pregnancy tests, and must use an effective birth control method during the study.
- Male patients must be either surgically sterile or use an effective birth control method during the study.
- Patient must be willing and able to provide written informed consent. Parent of legal guardian to patients younger than age of majority must be willing and able to provide informed consent.
Who cannot join
- Any significant medical condition or lab abnormality that would prevent the patient from participating in the study.
- Pregnant or lactating women.
- Patients taking an immunosuppressive agent (except topical over-the-counter steroids, inhaled steroid medications, and non-steroidal anti-inflammatory drugs) or have a planned surgery (except dental surgery or simple dermatologic procedures).
- Patients participating in an unapproved investigational clinical trial within 30 days of this study.
- Patients with a disease, disability or condition 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.
- Patients who are unwilling or unable to comply with the study requirements.
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- Patients with known hepatitis B or hepatitis C, or being treated for a positive viral load or are noncompliant with hepatitis medications.
- Known and active human immunodeficiency virus (HIV) infection.
- Patients with Child Pugh class C cirrhosis or liver failure.
- Patients with severe congestive heart failure (NYHA Class 4).
- Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
- History of allergic reaction to hepcidin or excipients.
- Contraindication to MRI scanning.
Trial locations and contacts
22 sites in 8 countries.
22 sites in 8 countries
Italy5 sites
Investigative Site
Cagliari, 09121
Investigative Site
Modena, 41124
Investigative Site
Naples, 80138
Investigative Site
Orbassano, 10043
Investigative Site
Verona, 37134
Turkey (Türkiye)5 sites
Investigative Site
Adana, 01330
Investigative Site
Ankara, 06100
Investigative Site
Antalya, 07059
Investigative Site
Antalya, 07160
Investigative Site
Izmir, 35100
Greece3 sites
Investigative Site
Goudi, Attica, 115 27
Investigative Site
Patra, Peloponnese, 26504
Investigative Site
Thessaloniki, Macedonia, 54642
United States2 sites
Investigative Site
New York, New York, 10021
Investigative Site
Oakland, California, 94609
Australia2 sites
Investigative Site
Camperdown, New South Wales, 2050
Investigative Site
Liverpool, New South Wales, 2170
Thailand2 sites
Investigative Site
Bangkok Noi, 10700
Investigative Site
Chiang Mai, 50200
United Kingdom2 sites
Investigative Site
London, England, E1 1BB
Investigative Site
London, N19 5NF
About this study
Official title: A Multi-Center, Randomized, Open-Label, Parallel-Group Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
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.
- Conditions
- Beta-Thalassemia
- Being tested
- LJPC-401
- Medical subject headings
- beta-Thalassemia
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
subcutaneous injection, up to 20 mg per week from week 26 to 52
LJPC-401Drug
Also called synthetic human hepcidin
subcutaneous injection, up to 20 mg per week from week 1 to 52
| Study group | Role | What they receive |
|---|---|---|
| Group A - Delayed therapy | Active comparator | standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks Receives: Drug: LJPC-401 |
| Group B - Immediate therapy | Active comparator | standard chelation therapy plus LJPC-401 for 52 weeks Receives: Drug: LJPC-401 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of LJPC-401 on cardiac iron
Change in cardiac T2* magnetic resonance imaging (MRI)
Measured: 26 Weeks
Secondary outcomes (12)
Effect of LJPC-401 on cardiac iron
Change in cardiac T2*MRI
Measured: 52 Weeks
Effect of LJPC-401 on hepatic iron
Change in hepatic T2*MRI
Measured: 52 Weeks
Effect of LJPC-401 on serum iron
Mean change in serum iron
Measured: 56 Weeks
Effect of LJPC-401 on hemoglobin
Mean change in hemoglobin
Measured: 52 Weeks
Effect of LJPC-401 on volume of blood transfused
Change in transfused blood volume
Measured: 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
Measured: 56 Weeks
Effect of LJPC-401 on the incidence of treatment-emergent adverse events
Measured: 56 Weeks
Effect of LJPC-401 on vital signs-heart rate
Measured: 56 Weeks
Effect of LJPC-401 on vital signs-body temperature
Measured: 56 Weeks
Effect of LJPC-401 on vital signs-respiratory rate
Measured: 56 Weeks
Effect of LJPC-401 on vital signs-blood pressure
Measured: 56 Weeks
Effect of LJPC-401 on body weight
Change in body weight (kilograms)
Measured: 56 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2017
- Study start30 Nov 2017
- Primary completion3 Dec 2019
- Study completion14 Jan 2020
Why it stopped: Terminated early for lack of efficacy as determined by interim endpoint analyses
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies 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
- None (open label)
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- La Jolla Pharmaceutical Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03381833
- Sponsor's protocol ID
- LJ401-BT01
- Status last verified
- Jul 2021
- First submitted
- 12 Dec 2017
- Last updated
- 29 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Randomized, Open-Label, Parallel-Group Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
- ClinicalTrials.gov ID
- NCT03381833
- Acronym / sponsor ID
- LJ401-BT01
- Conditions
- Beta-Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated early for lack of efficacy as determined by interim endpoint analyses
- Enrolment
- 84 participants (actual)
- Lead sponsor
- La Jolla Pharmaceutical CompanyIndustry (pharma, biotech, devices)
- Study start
- 30 Nov 2017 (actual)
- Primary completion
- 3 Dec 2019 (actual)
- Study completion
- 14 Jan 2020 (actual)
- First posted
- 22 Dec 2017 (actual)
- Results
- Not posted in the source record
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, 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A - Delayed therapy | Active comparator | Drug: LJPC-401standard chelation therapy alone for 26 weeks followed by standard chelation therapy plus LJPC-401 for 26 weeks |
| Group B - Immediate therapy | Active comparator | Drug: LJPC-401standard chelation therapy plus LJPC-401 for 52 weeks |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patients ≥ 14 years of age with transfusion-dependent beta thalassemia.
- Patients must have increased iron levels in the heart as measured by magnetic resonance imaging (MRI). Two separate cardiac T2*MRI from 6 to 35 msec; <= 15% difference between the two.
- Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose prior to study and expected to remain stable during study.
- Female patients of childbearing potential must not be pregnant, must have negative pregnancy tests, and must use an effective birth control method during the study.
- Male patients must be either surgically sterile or use an effective birth control method during the study.
Key exclusion criteria (13 listed)
- Any significant medical condition or lab abnormality that would prevent the patient from participating in the study.
- Pregnant or lactating women.
- Patients taking an immunosuppressive agent (except topical over-the-counter steroids, inhaled steroid medications, and non-steroidal anti-inflammatory drugs) or have a planned surgery (except dental surgery or simple dermatologic procedures).
- Patients participating in an unapproved investigational clinical trial within 30 days of this study.
- Patients with a disease, disability or condition 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.
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 hepcidinsubcutaneous injection, up to 20 mg per week from week 26 to 52 | Drug | Group A - Delayed therapy |
| LJPC-401Also: synthetic human hepcidinsubcutaneous injection, up to 20 mg per week from week 1 to 52 | Drug | Group B - Immediate therapy |
Primary outcome
Effect of LJPC-401 on cardiac iron
Time frame: 26 Weeks
Change in cardiac T2* magnetic resonance imaging (MRI)
12 secondary outcomes
Effect of LJPC-401 on cardiac iron
Time frame: 52 Weeks
Effect of LJPC-401 on hepatic iron
Time frame: 52 Weeks
Effect of LJPC-401 on serum iron
Time frame: 56 Weeks
Effect of LJPC-401 on hemoglobin
Time frame: 52 Weeks
Effect of LJPC-401 on volume of blood transfused
Time frame: 56 Weeks
Effect of LJPC-401 on its potential to elicit an immune response
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
Time frame: 56 Weeks
Results and safety
The study was stopped early (reason given: Terminated early for lack of efficacy as determined by interim endpoint analyses). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2021.
- Individual participant dataSharing planned: No
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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.
22 sites in 8 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 Beta-Thalassemia, run by La Jolla Pharmaceutical Company at 22 sites in 8 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 13 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: stopped early, as of the sponsor's last update on 29 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in Italy, Turkey (Türkiye), Greece, United States, Australia and 3 more countries. 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. 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?
The study was stopped early (reason given: Terminated early for lack of efficacy as determined by interim endpoint analyses). Results are not available in the source record.
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 on cardiac iron (time frame: 26 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 29 Jul 2021.