Mitochondrial DiseasesPhase 2Falcon-OLENCT07514338
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
A Phase 2 study testing Napazimone in people with Mitochondrial Diseases, run by Pharming Technologies B.V. at 23 sites in 9 countries.
Trial summary
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mitochondrial Diseases
- What is being tested?
- Napazimone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 140 participants
- Where
- 23 sitesin 9 countries
- Study dates
- 12 Jun 2026to 31 Mar 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Completed the FALCON study (age 18 years or older), and in the opinion of the investigator and sponsor has been compliant with the study requirements
- Willingness and ability to attend study appointments within the specified time windows
- Willingness and ability to complete electronic patient-reported outcomes
- Concomitant medications likely to remain stable throughout participation in the study where clinically possible
- Willingness to suspend treatment with idebenone during the study
Who cannot join
- The subject is, in the investigator's opinion, unlikely to comply with the protocol, e.g., due to cognitive impairment, or is unsuitable for any reason.
- Any medical, psychiatric, laboratory or other condition that may negatively affect the benefit-risk considerations of study participation or interfere with the interpretation of study results and, in the judgment of the investigator and/or the medical monitor, would make the subject inappropriate for entry into this study.
- Subjects not enrolling directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) study will be required to fulfill the additional exclusion criteria below during the screening visit:
- General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator.
Trial locations and contacts
23 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
23 sites in 9 countries
France7 sites, 7 recruiting
- Recruiting
Centre Hospitalier Universitaire d'Angers
Angers, 49933
Marco Spinazzi, MD
Marco Spinazzi
- Recruiting
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux
Aurelien Trimouille, MD
Aurelien Trimouille, MD
- Recruiting
Hopital Roger Salengro, CHRU de Lille
Lille
Celine Tard
Celine Tard, MD
- Recruiting
Centre Hospitalier Universitaire de Nantes
Nantes, 44093
Yann Pereon, MD
Yann Pereon
- Recruiting
Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2
Nice
Sabrina Sacconi, MD
Sabrina Sacconi, MD
- Recruiting
CHU de NICE - Hôpital Archet 2
Nice
Véronique Paquis-Flucklinger, MD
- Recruiting
Hopitaux Universitaires de Strasbourg
Strasbourg, 67091
Aleksandra Nadaj-Pakleza
Aleksandra Nadaj-Pakleza
Belgium6 sites, 6 recruiting
- Recruiting
Hospital Erasme
Brussels, 1070
Gauthier Remiche
- Recruiting
Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel
Brussels
Gauthier Remiche, MD
Christiane Kitoko
- Recruiting
University Hospital
Ghent, 9000
Hemelsoet, PI
- Recruiting
Universitair Ziekenhuis Gent
Ghent
Fien Demeulemeester
Dimitri Hemelsoet, MD
- Recruiting
University Hospital
Leuven, 3000
Claeys
- Recruiting
Universitair Ziekenhuis Leuven Gasthuisberg Campus
Leuven
Kristl Claeys, MD
Kristl Claeys, MD
Italy3 sites, 3 recruiting
- Recruiting
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Messina, 98125
Olimpia Musumeci, MD Professor
Selene Drago
- Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Costanza Lamperti, MD
Valeria Nicoletta
- Recruiting
Azienda Ospedaliero Universitaria Pisana
Pisa, 56126
Michelangelo Mancuso, MD
Riani Pierangela
Spain2 sites, 2 recruiting
- Recruiting
Hospital General Universitario de Catalunya
Barcelona
Susana Andrade Olivié
Josep Gamez Carbonell, MD
- Recruiting
Hospital Universitario 12 de Octubre
Madrid
Montserrat Morales, MD
Montserrat Morales, MD
United States1 site, 1 recruiting
- Recruiting
Akron Children's Hospital
Akron, Ohio, 44308
Bruce Cohen
Czechia1 site, 1 recruiting
- Recruiting
Charles University and General University Hospital
Prague, 128 08
Hana Krejcova, MD
Hana Krejcova
Denmark1 site, 1 recruiting
- Recruiting
Copenhagen Neuromuscular Center, Rigshospitalet
Copenhagen
Nicolai Rasmus Preisler, MD
Nicolai Preisler, MD
Germany1 site, 1 recruiting
- Recruiting
Universitaetsklinikum Halle
Halle, 6120
Alexander Mensch, MD
Netherlands1 site, 1 recruiting
- Recruiting
Radboud University Medical Center
Nijmegen, 6525
Christiaan Saris, MD
About this study
Official title: An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of KL1333 (Napazimone) in Patients With Primary Mitochondrial Disease
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD.
Read the detailed description
This is a 12-month open-label extension (OLE) study to evaluate the safety, tolerability, and efficacy of KL1333 in subjects previously treated with KL1333 or placebo in Study KL1333-2020-104A (hereafter referred to as FALCON).
Subjects can be enrolled in this extension study either directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) or later in time. Subjects who previously received KL1333 in FALCON will receive retreatment whereas subjects previously treated with placebo will be treatment naïve to KL1333.
The study consists of a screening visit (if the subject rolls over directly from the FALCON study completion visit [FALCON Week 48] or the safety follow up visit [FALCON Week 53], the screening visit is coincident with the visit), a 48-week treatment course with KL1333 up to 100 mg/day, a completion visit (end of treatment; EoT) and approximately 5 weeks of follow up, including the end of study (EoS) visit. The treatment period may extend beyond 48 weeks until the study drug is commercially or otherwise available, in which case the EoT visit will occur later than Week 48. Periodic safety monitoring visits (phone visits every 4 weeks after Week 48 and clinic visits every 24 weeks after Week 48) will continue for subjects who receive KL1333 in the optional extended treatment period until the study drug is commercially or otherwise available.
- Conditions
- Mitochondrial Diseases
- Being tested
- Napazimone
- Medical subject headings
- Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NapazimoneDrug
Also called KL1333
Product: KL1333 (international nonproprietary name: napazimone) Dose: Each subject will be up-titrated to his/her maximum well tolerated dose. The starting dose will be 25 mg KL1333 twice daily (BID; total daily dose of 50 mg). If KL1333 is considered to be well tolerated after 4 weeks of treatment, the dose will be increased to 50 mg KL1333 BID (total daily dose of 100 mg). The dose may be lowered from 50 mg BID to 25 mg BID at the investigator's discretion throughout the study in case of tolerability issues.
Frequency: Twice daily Route: Oral
| Study group | Role | What they receive |
|---|---|---|
| Open label extension | Experimental | Subjects will receive medication twice a day for 48 weeks minimum Receives: Drug: Napazimone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Number of adverse events will be monitored throughout the study for all subjects
Measured: Through study at least for 48 weeks
Physical examination
The following parameters and body systems will be examined and any abnormalities described: height and weight; general appearance; skin; head, ears, eyes, nose, and throat; lungs; heart; lower extremity examination; abdomen; neurologic and lymph nodes. Any clinically significant changes from baseline should be recorded as AEs.
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Vital signs
Body temperature, systolic and diastolic cuff blood pressure, pulse rate and pulse oximetry will be measured and any clinically significant changes from baseline should be recorded as AEs.
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Electrocardiogram
Changes from baseline of ECG parameters will be evaluated.
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Safety laboratory - blood chemistry
Monitoring of the clinically significant laboratory results for sodium, potassium, chloride, bicarbonate/carbon dioxide;, blood urea nitrogen, serum creatinine, glucose, albumin, total protein, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, total bilirubin, direct bilirubin, indirect bilirubin, calcium, gamma-glutamyl transferase, creatine kinase
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Safety laboratory - urinalysis
Monitoring of the clinically significant laboratory results for specific gravity, pH, semi-quantitative "dipstick" evaluation of glucose, protein, bilirubin, ketones, leukocytes, blood microscopy and/or culture to be performed if clinically indicated or if urinalysis results positive.
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Safety laboratory - hematology
Monitoring of the clinically significant laboratory results for Hemoglobin, hematocrit, white blood cell with differentials (monocytes, eosinophils, basophils, neutrophils, lymphocytes) as an absolute value, red blood cell count, platelet count, C-reactive protein
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Occurence of metabolic decompensation and lactic acidosis or image-verified stroke-like episodes consequent to GI AE and AESIs
These events will be monitored throughout the study.
Measured: Through study at least for 48 weeks
Columbia Suicide Severity Rating Scale (C-SSRS)
C-SSRS assesses suicidal ideation and behavior risk through a series of questions to assess for suicidal ideation and behavior, the severity and immediacy of the risk, and the level of support the subject may need. C-SSRS Severity of Ideation scores of 4 or 5 are considered SAEs.
Measured: At Baseline Week 0
Secondary outcomes (8)
Patient-reported mitochondrial fatigue
Patient-Reported Outcomes Measurement Information System® Fatigue PMD short form. The PROMIS® Fatigue PMD short form consists of 9 items. Each item has numerical rating scale (NRS) response options consisting of never (1), rarely (2), sometimes (3), often (4), and always (5).
Measured: Through study at least for 48 weeks
Functional outcome
30 seconds sit-to-stand test
Measured: At Baseline Week 0, Week 4, Week 24 and Week 48
Patient-reported lower extremity function
Neuro-QOL Lower Extremity Function (Mobility) - short form. It is a reliable and validated brief 8-item survey of one's ability to carry out various activities involving the lower limb/trunk region and increasing degrees of bodily movement. Each item has numeric rating scale (NRS) response options consisting of Without any difficulty (5), With a little difficulty (4), With some difficulty (3), With much difficulty (2), and unable to do (1).
Measured: At Baseline Week 0, Week 4, Week 12, Week 24, Week 36 and Week 48
Other patient-reported outcome - Patient Global Impression (multiple)
The Patient Global Impression of Severity is rated on a 4-point NRS, assessed on a scale ranging from 1 (none) to 4 (severe) for severity. The Patient Global Impression of Change is rated on a 5-point numeric rating scale (NRS), with the change from baseline assessed on a scale ranging from 2 (much better) to -2 (much worse), with 0 indicating no change from baseline.
Measured: At Baseline Week 0, Week 4, Week 12, Week 24, Week 36 and Week 48
Other patient-reported outcomes - 5-level EuroQol-5 Dimension
The EQ-5D-5L includes items addressing mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The scale yields a single score on a 0 to 100 scale.
Measured: At Baseline Week 0, Week 4, Week 12, Week 24, Week 36 and Week 48
Global impression of severity of PMD disease expression
Clinician Global Impression of PMD - severity and change
Measured: At Baseline Week 0, Week 24 and Week 48
Assessments of mitochondrial disease progression
Newcastle Mitochondrial Disease Adult Scale, Subscales I-III
Measured: At Baseline Week 0, Week 24 and Week 48
Mitochondrial diabetes, subgroup analysis
Glycated hemoglobin (HbA1c, in subjects with diabetes)
Measured: At Baseline Week 0, Week 24 and Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2026
- Study start12 Jun 2026
- Primary completion31 Mar 2029 (estimated)
- Study completion31 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial DiseasesStudies 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
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label Extension
- Masking
- None (open label)
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharming Technologies B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07514338
- Sponsor's protocol ID
- KL1333 2025-104B
- Other IDs
- 2025-524367-20-00, 2025
- Acronym
- Falcon-OLE
- Status last verified
- Jul 2026
- First submitted
- 24 Feb 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of KL1333 (Napazimone) in Patients With Primary Mitochondrial Disease
- ClinicalTrials.gov ID
- NCT07514338
- Acronym / sponsor ID
- Falcon-OLE / KL1333 2025-104B
- Conditions
- Mitochondrial Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Pharming Technologies B.V.Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- 12 Jun 2026 (actual)
- Primary completion
- 31 Mar 2029 (estimated)
- Study completion
- 31 Mar 2029 (estimated)
- First posted
- 7 Apr 2026 (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.
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD.
Detailed description
This is a 12-month open-label extension (OLE) study to evaluate the safety, tolerability, and efficacy of KL1333 in subjects previously treated with KL1333 or placebo in Study KL1333-2020-104A (hereafter referred to as FALCON).
Subjects can be enrolled in this extension study either directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) or later in time. Subjects who previously received KL1333 in FALCON will receive retreatment whereas subjects previously treated with placebo will be treatment naïve to KL1333.
The study consists of a screening visit (if the subject rolls over directly from the FALCON study completion visit [FALCON Week 48] or the safety follow up visit [FALCON Week 53], the screening visit is coincident with the visit), a 48-week treatment course with KL1333 up to 100 mg/day, a completion visit (end of treatment; EoT) and approximately 5 weeks of follow up, including the end of study (EoS) visit. The treatment period may extend beyond 48 weeks until the study drug is commercially or otherwise available, in which case the EoT visit will occur later than Week 48. Periodic safety monitoring visits (phone visits every 4 weeks after Week 48 and clinic visits every 24 weeks after Week 48) will continue for subjects who receive KL1333 in the optional extended treatment period until the study drug is commercially or otherwise available.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label Extension
| Arm | Type | Receives |
|---|---|---|
| Open label extension | Experimental | Drug: NapazimoneSubjects will receive medication twice a day for 48 weeks minimum |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Completed the FALCON study (age 18 years or older), and in the opinion of the investigator and sponsor has been compliant with the study requirements
- Willingness and ability to attend study appointments within the specified time windows
- Willingness and ability to complete electronic patient-reported outcomes
- Concomitant medications likely to remain stable throughout participation in the study where clinically possible
- Willingness to suspend treatment with idebenone during the study
Key exclusion criteria (4 listed)
- The subject is, in the investigator's opinion, unlikely to comply with the protocol, e.g., due to cognitive impairment, or is unsuitable for any reason.
- Any medical, psychiatric, laboratory or other condition that may negatively affect the benefit-risk considerations of study participation or interfere with the interpretation of study results and, in the judgment of the investigator and/or the medical monitor, would make the subject inappropriate for entry into this study.
- Subjects not enrolling directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) study will be required to fulfill the additional exclusion criteria below during the screening visit:
- General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator.
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 |
|---|---|---|
| NapazimoneAlso: KL1333Product: KL1333 (international nonproprietary name: napazimone) Dose: Each subject will be up-titrated to his/her maximum well tolerated dose. The starting dose will be 25 mg KL1333 twice daily (BID; total daily dose of 50 mg). If KL1333 is considered to be well tolerated after 4 weeks of treatment, the dose will be increased to 50 mg KL1333 BID (total daily dose of 100 mg). The dose may be lowered from 50 mg BID to 25 mg BID at the investigator's discretion throughout the study in case of tolerability issues. Frequency: Twice daily Route: Oral | Drug | Open label extension |
Primary outcome
Adverse events
Time frame: Through study at least for 48 weeks
Number of adverse events will be monitored throughout the study for all subjects
Physical examination
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
The following parameters and body systems will be examined and any abnormalities described: height and weight; general appearance; skin; head, ears, eyes, nose, and throat; lungs; heart; lower extremity examination; abdomen; neurologic and lymph nodes. Any clinically significant changes from baseline should be recorded as AEs.
Vital signs
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
Body temperature, systolic and diastolic cuff blood pressure, pulse rate and pulse oximetry will be measured and any clinically significant changes from baseline should be recorded as AEs.
Electrocardiogram
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
Changes from baseline of ECG parameters will be evaluated.
Safety laboratory - blood chemistry
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
Monitoring of the clinically significant laboratory results for sodium, potassium, chloride, bicarbonate/carbon dioxide;, blood urea nitrogen, serum creatinine, glucose, albumin, total protein, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, total bilirubin, direct bilirubin, indirect bilirubin, calcium, gamma-glutamyl transferase, creatine kinase
Safety laboratory - urinalysis
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
Monitoring of the clinically significant laboratory results for specific gravity, pH, semi-quantitative "dipstick" evaluation of glucose, protein, bilirubin, ketones, leukocytes, blood microscopy and/or culture to be performed if clinically indicated or if urinalysis results positive.
Safety laboratory - hematology
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
Monitoring of the clinically significant laboratory results for Hemoglobin, hematocrit, white blood cell with differentials (monocytes, eosinophils, basophils, neutrophils, lymphocytes) as an absolute value, red blood cell count, platelet count, C-reactive protein
Occurence of metabolic decompensation and lactic acidosis or image-verified stroke-like episodes consequent to GI AE and AESIs
Time frame: Through study at least for 48 weeks
These events will be monitored throughout the study.
Columbia Suicide Severity Rating Scale (C-SSRS)
Time frame: At Baseline Week 0
C-SSRS assesses suicidal ideation and behavior risk through a series of questions to assess for suicidal ideation and behavior, the severity and immediacy of the risk, and the level of support the subject may need. C-SSRS Severity of Ideation scores of 4 or 5 are considered SAEs.
8 secondary outcomes
Patient-reported mitochondrial fatigue
Time frame: Through study at least for 48 weeks
Functional outcome
Time frame: At Baseline Week 0, Week 4, Week 24 and Week 48
Patient-reported lower extremity function
Time frame: At Baseline Week 0, Week 4, Week 12, Week 24, Week 36 and Week 48
Other patient-reported outcome - Patient Global Impression (multiple)
Time frame: At Baseline Week 0, Week 4, Week 12, Week 24, Week 36 and Week 48
Other patient-reported outcomes - 5-level EuroQol-5 Dimension
Time frame: At Baseline Week 0, Week 4, Week 12, Week 24, Week 36 and Week 48
Global impression of severity of PMD disease expression
Time frame: At Baseline Week 0, Week 24 and Week 48
Assessments of mitochondrial disease progression
Time frame: At Baseline Week 0, Week 24 and Week 48
Mitochondrial diabetes, subgroup analysis
Time frame: At Baseline Week 0, Week 24 and Week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
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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.
23 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Napazimone in people with Mitochondrial Diseases, run by Pharming Technologies B.V. at 23 sites in 9 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 5 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in France, Belgium, Italy, Spain, United States and 4 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Audrey Simon, Sr. CPM (+31 71 524 7448, a.simon@pharming.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: Through study at least for 48 weeks). The study lists 9 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 Sep 2026.