Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT02534909
Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 2 study testing LFG316 and LNP023 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 7 sites in 3 countries.
Trial summary
The purpose of this study was to determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with paroxysmal nocturnal hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- LFG316 and LNP023
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 7 sitesin 3 countries
- Study dates
- 9 Sep 2015to 24 May 2022
- 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
- Written informed consent obtained before any assessment were performed.
- Male and female patients >= 18 years old with a diagnosis of PNH prior to screening. Based on local requirements (applicable in Czech Republic) only patients between the age of 18 to 65 (inclusive) with a diagnosis of PNH prior to screening were eligible for inclusion in this study.
- A documented PNH clone size of >= 10% by RBCs and/or granulocytes, measured by GPI deficiency on flow cytometry.
- Serum LDH levels at least 1.5-fold above the ULN at screening.
- Patients receiving treatment with corticosteroids and/or other immunosuppressive regimens could continue treatment throughout the study, if indicated for treatment of autoimmune disease (e.g., aplastic anemia). It was strongly recommended, at the investigator's discretion, that patients receive appropriate prophylactic antibiotics (e.g., ciprofloxacin, penicillin, erythromycin) while on treatment with any type of concomitant immunosuppressive agent other than LFG316 (including corticosteroids).
- Negative pregnancy test for women of child-bearing potential at screening.
Show all 13Show fewer
- Previous vaccination against Neisseria meningitidis types A, C, Y and W-135 at least 2 weeks prior to first dosing, and vaccination against meningitidis type B if available and acceptable by local regulations, at least 2 weeks prior to first dosing.
- Able to communicate well with the investigator, to understand and comply with the requirements of the study.
- Patients included in this study after protocol amendment 6 were also to fulfill the following:
- To be carriers of the C5 gene minor variants as defined by nucleic acid changes that lead to amino acid exchanges in position p.Arg885.
- Additional inclusion criteria for period 4
- Patients who participated in period 3 of the current study who wanted to join the long-term extension study with LNP023 (CLNP023C12001B).
- Previous vaccination for the prevention of Streptococcus pneumoniae and Haemophilus influenzae at least 2 weeks prior to first dosing with LNP023 if locally available. If LNP023 treatment had to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment was required.
Who cannot join
- Known or suspected hereditary complement deficiency.
- History of hematopoietic stem cell transplantation.
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5-times the half-life prior to screening. Note: clinical trials solely involving over-the-counter vitamins, off label use of drugs within published standard of care guidelines, supplements, or diet did not exclude an otherwise eligible patient.
- Female patients who were pregnant, breastfeeding, or intended to conceive during the course of the study.
- History of recurrent meningitis, history of meningococcal meningitis despite vaccination.
- Presence or suspicion (based on judgment of the investigator) of severe active bacterial infection within 2 weeks prior to first dose of LFG316, or severe recurrent bacterial infections.
Show all 17Show fewer
- A positive HIV test result.
- Under active therapy with other agents interfering with the complement system (e.g., eculizumab) Wash-out time was at least 5 half-lives, approximately 8 weeks for eculizumab.
- Severe concurrent co-morbidities, e.g., patients with severe kidney disease (dialysis), advanced cardiac disease (NYHA class IV), severe pulmonary arterial hypertension (WHO class IV), unstable thrombotic event not amenable to active treatment as judged by the investigator.
- Either one of the following laboratory abnormalities at screening:
- Neutrophils < 0.5 × 10^9/L
- Platelets < 30 × 10^9/L
- Co-morbidities that were likely caused by underlying autoimmune diseases other than PNH, e.g., kidney disease in the context of lupus nephritis, ANCA-associated vasculitis.
- Any medical condition deemed likely to interfere with the patient's participation in the study, or likely to cause serious adverse events during the study.
- History of hypersensitivity to a drug of the same class (human IgG1 monoclonal antibody) or any other excipient of the formulation.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they were using highly effective methods of contraception during dosing and for 50 days after the last dose of LFG316.
- Prohibited medication as specified in the study protocol.
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
Japan5 sites
Novartis Investigative Site
Fukushima, Fukushima, 960 1295
Novartis Investigative Site
Isehara, Kanagawa, 259-1193
Novartis Investigative Site
Niigata, 951 8520
Novartis Investigative Site
Shinjuku Ku, Tokyo, 160-0023
Novartis Investigative Site
Suita, Osaka, 565 0871
Czechia1 site
Novartis Investigative Site
Brno-Bohunice, Czech Republic, 625 00
About this study
Official title: An Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study was to determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Read the detailed description
The study consisted of a 60-day screening period to assess eligibility and conduct vaccinations if required (for all patients not previously vaccinated at least 2 weeks prior to first dosing or if prior vaccination cannot be confirmed) and 4 treatment periods as follows. During the treatment period 1 (Days 1 to 29), all patients received infusions of LFG316 every 14 days. Following assessment of efficacy (hemolytic activity by serum LDH) at the end of treatment period 1, patients entered the optional 48-week treatment period 2 and continued LFG316 infusions every 14 days. At the end of treatment period 2, LFG316-responsive patients (assessed based on the investigator's judgment) were allowed to enter an additional extension period of up to 260 weeks (treatment period 3) in which they continued to receive LFG316 every 14 days. Period 4, which allowed patients to switch to LNP023, lasted approximately 21 weeks. During the first 4 weeks, patients continued to receive LFG316 in addition to oral administration of LNP023. After 4 weeks, patients discontinued LFG316 and continued with LNP023 monotherapy for approximately 16 weeks (+/- 28 days). Patients who participated in period 4 could join the long-term extension study CLNP023C12001B (NCT04747613) as soon as their eligibility was confirmed and study CLNP023C12001B was open to receive patients. There was no LNP023 treatment gap between the studies.
As per strategic decision, further development of LFG316 was terminated in favor of LNP023, Novartis offered patients enrolled in study CLFG316X2201 a conversion from LFG316 to LNP023, aiming to provide uninterrupted treatment for these PNH patients. The CLFG316X2201 trial was not a terminated study.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- LFG316, LNP023
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria (PNH), LFG316, LNP023
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LFG316Biological
LFG316 20 mg/kg was administered to all patients enrolled in the study:
- Treatment Periods 1 to 3: LFG316 20 mg/kg as i.v. infusion every 2 weeks
- Treatment Period 4: LFG316 20 mg/kg as i.v. infusion every 2 weeks for 4 weeks (total 2 infusions).
LNP023Drug
Treatment Period 4: LNP023 200 mg b.i.d. for approximately 20 weeks. Four capsules (each 50 mg) were administered each time study medication was taken.
| Study group | Role | What they receive |
|---|---|---|
| LFG316 then LNP023 | Experimental | During treatment periods 1 to 3, the regimen included LFG316 at a dosage of 20 mg/kg administered as an intravenous (i.v.) infusion biweekly.
In treatment period 4, the patients were given:
* LFG316 at a dosage of 20 mg/kg as an i.v. infusion every two weeks for four weeks, amounting to two infusions in total.
* LNP023 at a dose of 200 mg administered orally twice daily (b.i.d.) for approximately 20 weeks, with four 50 mg capsules taken at each administration. Receives: Biological: LFG316; Drug: LNP023 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Reduction in Serum Lactate Dehydrogenase (LDH) Levels Within the First 4 Weeks of LFG316 Treatment as Measured by Response Rate
The primary efficacy variable for assessing the effect of LFG316 over the first 4 weeks of treatment was response rate where a patient was considered a responder if the percentage reduction from baseline in serum lactate dehydrogenase (LDH) was at least 60% at any time up to and including week 4 for that patient.
Measured: Overall (Up to Week 4), Period 1 Day 8, Period 1 Day 15, Period 1 Day 22, Period 1 Day 29
Percentage Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels Over the Entire Treatment Period
Lactate dehydrogenase (LDH) levels were measured in serum samples and the percentage change from baseline was calculated. For serum LDH, baseline was the average of all pre-dose measurements.
Measured: Baseline, Period 1 Day 29 (end of Treatment Period 1), Period 2 Day 365 (end of Treatment Period 2), Period 3 Day 1429 (end of Treatment Period 3), Period 4 Day 141 (end of Treatment Period 4)
Secondary outcomes (4)
Area Under the Concentration-time Curve (AUC) From Time Zero to the Last Measurable Serum Concentration Sampling Time (0-tlast) for LFG316
Venous whole blood samples were collected for activity-based pharmacokinetics characterization of LFG316. Pharmacokinetic parameters were determined using non-compartmental methods based on LFG316 concentrations in serum. AUC (0-tlast) was summarized using descriptive statistics.
Measured: Pre-infusion and 2 hours after the end of infusion on Period 1 Day 1.
Maximum Observed Serum Concentration (Cmax) for LFG316
Venous whole blood samples were collected for activity-based pharmacokinetics characterization of LFG316. Pharmacokinetic parameters were determined using non-compartmental methods based on LFG316 concentrations in serum. Cmax was summarized using descriptive statistics.
Measured: Pre-infusion and 2 hours after the end of infusion on Period 1 Day 1.
Time to Reach Maximum Serum Concentration (Tmax) for LFG316
Venous whole blood samples were collected for activity-based pharmacokinetics characterization of LFG316. Pharmacokinetic parameters were determined using non-compartmental methods based on LFG316 concentrations in serum. Tmax was summarized using descriptive statistics. Actual sampling times were used for the calculation of PK parameters.
Measured: Pre-infusion and 2 hours after the end of infusion on Period 1 Day 1.
LFG316 Serum Concentration
The concentration of total LFG316 in serum was determined using Liquid chromatography/mass spectroscopy (LC/MS assay) and summarized using descriptive statistics.
Measured: Period 1 Day 1 (predose (trough), post-dose), Period 2 Day 337 (predose), Period 3 Day 1289 (predose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2015 (estimated)
- Study start9 Sep 2015
- Primary completion24 May 2022
- Study completion24 May 2022
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 Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Model description
- All patients were on active treatment. In treatment periods 1 to 3, patients received the following: • LFG316 20 mg/kg as i.v. infusion every 2 weeks Patients participating in treatment period 4 received the following: * LFG316 20 mg/kg as i.v. infusion every 2 weeks for 4 weeks (total 2 infusions) * LNP023 200 mg b.i.d. for approximately 20 weeks. Four capsules (each 50 mg) were administered each time study medication was taken. Down-titration was permitted only in case of LNP023 treatment discontinuation: * LNP023 30 mg taken in the evening (once daily) for 7 days (3 capsules of 10 mg each time study medication was taken). * Followed by LNP023 10 mg taken in the evening (once daily) for 7 days (1 capsule of 10 mg each time study medication was taken). * LFG316 was administered via i.v. infusion by the investigator or designated study staff over approximately 2 hours in Period 1 and between 40 minutes and 2 hours from Period 2 onwards.
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02534909
- Sponsor's protocol ID
- CLFG316X2201
- Other IDs
- 2014-005338-74, 2014
- Status last verified
- May 2025
- First submitted
- 7 Aug 2015
- Last updated
- 16 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT02534909
- Acronym / sponsor ID
- CLFG316X2201
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria (PNH), LFG316, LNP023
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 9 Sep 2015 (actual)
- Primary completion
- 24 May 2022 (actual)
- Study completion
- 24 May 2022 (actual)
- First posted
- 28 Aug 2015 (estimated)
- Results
- Posted 1 Nov 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Detailed description
The study consisted of a 60-day screening period to assess eligibility and conduct vaccinations if required (for all patients not previously vaccinated at least 2 weeks prior to first dosing or if prior vaccination cannot be confirmed) and 4 treatment periods as follows. During the treatment period 1 (Days 1 to 29), all patients received infusions of LFG316 every 14 days. Following assessment of efficacy (hemolytic activity by serum LDH) at the end of treatment period 1, patients entered the optional 48-week treatment period 2 and continued LFG316 infusions every 14 days. At the end of treatment period 2, LFG316-responsive patients (assessed based on the investigator's judgment) were allowed to enter an additional extension period of up to 260 weeks (treatment period 3) in which they continued to receive LFG316 every 14 days. Period 4, which allowed patients to switch to LNP023, lasted approximately 21 weeks. During the first 4 weeks, patients continued to receive LFG316 in addition to oral administration of LNP023. After 4 weeks, patients discontinued LFG316 and continued with LNP023 monotherapy for approximately 16 weeks (+/- 28 days). Patients who participated in period 4 could join the long-term extension study CLNP023C12001B (NCT04747613) as soon as their eligibility was confirmed and study CLNP023C12001B was open to receive patients. There was no LNP023 treatment gap between the studies.
As per strategic decision, further development of LFG316 was terminated in favor of LNP023, Novartis offered patients enrolled in study CLFG316X2201 a conversion from LFG316 to LNP023, aiming to provide uninterrupted treatment for these PNH patients. The CLFG316X2201 trial was not a terminated study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All patients were on active treatment. In treatment periods 1 to 3, patients received the following: • LFG316 20 mg/kg as i.v. infusion every 2 weeks Patients participating in treatment period 4 received the following: * LFG316 20 mg/kg as i.v. infusion every 2 weeks for 4 weeks (total 2 infusions) * LNP023 200 mg b.i.d. for approximately 20 weeks. Four capsules (each 50 mg) were administered each time study medication was taken. Down-titration was permitted only in case of LNP023 treatment discontinuation: * LNP023 30 mg taken in the evening (once daily) for 7 days (3 capsules of 10 mg each time study medication was taken). * Followed by LNP023 10 mg taken in the evening (once daily) for 7 days (1 capsule of 10 mg each time study medication was taken). * LFG316 was administered via i.v. infusion by the investigator or designated study staff over approximately 2 hours in Period 1 and between 40 minutes and 2 hours from Period 2 onwards.
| Arm | Type | Receives |
|---|---|---|
| LFG316 then LNP023 | Experimental | Biological: LFG316, Drug: LNP023During treatment periods 1 to 3, the regimen included LFG316 at a dosage of 20 mg/kg administered as an intravenous (i.v.) infusion biweekly. In treatment period 4, the patients were given: * LFG316 at a dosage of 20 mg/kg as an i.v. infusion every two weeks for four weeks, amounting to two infusions in total. * LNP023 at a dose of 200 mg administered orally twice daily (b.i.d.) for approximately 20 weeks, with four 50 mg capsules taken at each administration. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Written informed consent obtained before any assessment were performed.
- Male and female patients >= 18 years old with a diagnosis of PNH prior to screening. Based on local requirements (applicable in Czech Republic) only patients between the age of 18 to 65 (inclusive) with a diagnosis of PNH prior to screening were eligible for inclusion in this study.
- A documented PNH clone size of >= 10% by RBCs and/or granulocytes, measured by GPI deficiency on flow cytometry.
- Serum LDH levels at least 1.5-fold above the ULN at screening.
- Patients receiving treatment with corticosteroids and/or other immunosuppressive regimens could continue treatment throughout the study, if indicated for treatment of autoimmune disease (e.g., aplastic anemia). It was strongly recommended, at the investigator's discretion, that patients receive appropriate prophylactic antibiotics (e.g., ciprofloxacin, penicillin, erythromycin) while on treatment with any type of concomitant immunosuppressive agent other than LFG316 (including corticosteroids).
Key exclusion criteria (17 listed)
- Known or suspected hereditary complement deficiency.
- History of hematopoietic stem cell transplantation.
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5-times the half-life prior to screening. Note: clinical trials solely involving over-the-counter vitamins, off label use of drugs within published standard of care guidelines, supplements, or diet did not exclude an otherwise eligible patient.
- Female patients who were pregnant, breastfeeding, or intended to conceive during the course of the study.
- History of recurrent meningitis, history of meningococcal meningitis despite vaccination.
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 |
|---|---|---|
| LFG316LFG316 20 mg/kg was administered to all patients enrolled in the study: * Treatment Periods 1 to 3: LFG316 20 mg/kg as i.v. infusion every 2 weeks * Treatment Period 4: LFG316 20 mg/kg as i.v. infusion every 2 weeks for 4 weeks (total 2 infusions). | Biological | LFG316 then LNP023 |
| LNP023Treatment Period 4: LNP023 200 mg b.i.d. for approximately 20 weeks. Four capsules (each 50 mg) were administered each time study medication was taken. | Drug | LFG316 then LNP023 |
Primary outcome
Percentage of Participants With Reduction in Serum Lactate Dehydrogenase (LDH) Levels Within the First 4 Weeks of LFG316 Treatment as Measured by Response Rate
Time frame: Overall (Up to Week 4), Period 1 Day 8, Period 1 Day 15, Period 1 Day 22, Period 1 Day 29
The primary efficacy variable for assessing the effect of LFG316 over the first 4 weeks of treatment was response rate where a patient was considered a responder if the percentage reduction from baseline in serum lactate dehydrogenase (LDH) was at least 60% at any time up to and including week 4 for that patient.
Percentage Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels Over the Entire Treatment Period
Time frame: Baseline, Period 1 Day 29 (end of Treatment Period 1), Period 2 Day 365 (end of Treatment Period 2), Period 3 Day 1429 (end of Treatment Period 3), Period 4 Day 141 (end of Treatment Period 4)
Lactate dehydrogenase (LDH) levels were measured in serum samples and the percentage change from baseline was calculated. For serum LDH, baseline was the average of all pre-dose measurements.
4 secondary outcomes
Area Under the Concentration-time Curve (AUC) From Time Zero to the Last Measurable Serum Concentration Sampling Time (0-tlast) for LFG316
Time frame: Pre-infusion and 2 hours after the end of infusion on Period 1 Day 1.
Maximum Observed Serum Concentration (Cmax) for LFG316
Time frame: Pre-infusion and 2 hours after the end of infusion on Period 1 Day 1.
Time to Reach Maximum Serum Concentration (Tmax) for LFG316
Time frame: Pre-infusion and 2 hours after the end of infusion on Period 1 Day 1.
LFG316 Serum Concentration
Time frame: Period 1 Day 1 (predose (trough), post-dose), Period 2 Day 337 (predose), Period 3 Day 1289 (predose)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LFG316 and LNP023 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 7 sites in 3 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 13 inclusion and 17 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 16 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Japan, Czechia, Lithuania. 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?
Yes. Results are posted in the registry (first posted 1 Nov 2024). 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?
Percentage of Participants With Reduction in Serum Lactate Dehydrogenase (LDH) Levels Within the First 4 Weeks of LFG316 Treatment as Measured by Response Rate (time frame: Overall (Up to Week 4), Period 1 Day 8, Period 1 Day 15, Period 1 Day 22, Period 1 Day 29). The study lists 2 primary outcomes in all. 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 16 May 2025.