Wilson DiseasePhase 3NCT03539952
Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease
A Phase 3 study testing Penicillamine (D1-W12), TETA 4HCL (W12-60) and Penicillamine (W12-W60) in people with Wilson Disease, run by Orphalan at 15 sites in 9 countries.
Trial summary
This is a multicenter, randomized, open-label study with an active standard-of-care comparator (penicillamine) Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Wilson Disease
- What is being tested?
- Penicillamine (D1-W12), TETA 4HCL (W12-60) and Penicillamine (W12-W60)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 77 participants
- Where
- 15 sitesin 9 countries
- Study dates
- 3 Sep 2018to 18 Jan 2022
- Phase
- Phase 3
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient is able to provide, and has provided, written informed consent
- Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including: For US sites: Authorization for Use and Release of Health Research Study Information and for EU sites: Data Protection Consent
- Male or female, aged ≥ 18 and ≤ 75 years of age at time of consent
- Patient has a diagnosis of Wilson's disease, as defined by a prior or current Leipzig score of ≥ 4
- Patient's Wilson's disease is clinically stable, in the opinion of the investigator, and being treated with penicillamine for at least 1 year (52 weeks) prior to the screening/enrolment visit
- Patient is on a stable dose and regimen of penicillamine for at least 4 months (16 weeks) prior to the screening/enrolment visit (other prescribed treatments for Wilson's disease not permitted during this study)
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- No anticipated need that patient will require additional pharmacological therapies other than study medication, including prescribed zinc therapy, for the management of copper levels during the study
- Patient must be willing to maintain stable diet throughout the study, and avoid foods with high copper content, including the Penicillamine Baseline Period
- Patient considered suitable to receive therapy with both TETA 4HCl and penicillamine administered twice a day
- Negative central laboratory tests for HIV and viral hepatitis (results will be available after start of run-in period)
- For female patients of childbearing potential, negative urine pregnancy test (at screening/enrolment visit and prior to randomization)
- For females of childbearing potential, use of a reliable form of contraceptive
- Patient is considered as able to complete study requirements and attend the study visits, in the opinion of the investigator
- Additional inclusion criteria following receipt of Screening laboratory results
- Patient is adequately controlled and tolerating penicillamine therapy as defined by fulfilment of all of the following: a. Serum non-ceruloplasmin bound copper (NCC) level between ≥ 25 and ≤ 150 μg/L* b. 24-hour urinary copper excretion of between ≥ 100 and ≤ 900 μg/24 hours* c. Alanine transaminase (ALT) < 2 times upper limit of normal* d. No other laboratory or clinical findings that would prevent continuation of maintenance therapy, in the opinion of the investigator
- Based on results from screening/enrolment visit samples for which can be taken within ± 7 days of visit. Result should be within the assay limits of quantification for the sample. The ranges in μmol of copper are 0.40 to 2.38 μmol/L for NCC and 1.59 to 14.29 for 24-hour urinary copper excretion (using division by 63 of value in μg per Walshe, 2011). In the event that one or more of the above lab values fall outside the specified range, it can be repeated, including at the Week 4 and Week 8 visits.
- Additional inclusion criteria at Week 12 visit (end of Penicillamine Baseline Period) and prior to randomization
- Patient is adequately controlled and tolerating penicillamine therapy as defined by fulfilment of all of the following criteria:
- Serum non-ceruloplasmin bound copper (NCC) level between ≥ 25 and ≤ 150 μg/L*
- 24-hour urinary copper excretion of between ≥ 100 and ≤ 900 μg/24 hours**
- Alanine transaminase (ALT) < 2 times upper limit of normal*
- No other laboratory or clinical findings that would prevent continuation of maintenance therapy, in the opinion of the investigator
- Based on lab values from Week 8 visit; ** Based on lab value from Week 4 visit as routinely not performed at Week 8 visit, however can also be based on value at Week 8 visit if a repeat (unscheduled) urinary copper excretion was performed at this visit. Result should be within the assay limits of quantification for the sample. The ranges in μmol of copper are 0.40 to 2.38 μmol/L for NCC and 1.59 to 14.29 for 24-hour urinary copper excretion (using division by 63 of value in μg per Walshe, 2011). In the event that one or more of the above lab values fall outside the specified range, it can be repeated. The repeat value(s) must be available prior to randomization at Week 12 and, if within specified range, the patient can continue to randomization. If a patient fails this additional criterion at the end of the Penicillamine Baseline Period, the patient can return to the start of the run-in period i.e. Day 1 (but only once). A negative urinary pregnancy test is also required prior to randomization for females of childbearing potential.
Who cannot join
- Patient is in 'de-coppering' phase of treatment for Wilson's disease, in the opinion of the investigator
- Patient evidence of uncontrolled liver disease, including but not limited to:
- Modified Nazer score of > 4 (result may not be available until after start of run in period since based on lab results*)
- decompensated cirrhosis
- acute hemolytic anemia
- acute hepatitis
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- hepatic malignancy
- evidence of acute liver failure
- Cause of patient's liver disease is due to another condition, in the investigator's opinion
- Patient has severe anemia defined as hemoglobin of ≤ 9 g/dL (result will be available after start of run-in period*)
- Patient has experienced a gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment visit
- Patient has renal impairment defined as creatinine clearance of ≤ 30 mL/min (result may not be available until after start of run-in period*), or patient has nephritis or nephrotic syndrome, in the opinion of the investigator
- Patient has neurological disease that prevents swallowing of study medication (e.g., requires a nasogastric feeding tube) or requires intensive in-patient medical care
- Patient is currently taking medication containing trientine for management of Wilson's disease or has taken it within 4 months (16 weeks) of screening/enrolment visit
- Patient is currently receiving prescribed zinc therapy for management of Wilson's disease or has taken it within 4 months (16 weeks) of screening/enrolment visit
- Patient is taking any of the following concomitant therapies: gold therapy, antimalarial therapy, cytotoxic drugs, oxyphenbutazone, phenyl butazone
- Patient has a known intolerance, allergy or sensitivity to penicillamine (that is uncontrolled) or to TETA 4HCl, including any component of the study medication
- For female patients of childbearing potential, planning a pregnancy during study period or currently nursing
- For female patients of childbearing potential, unable or unwilling to use a reliable form of contraceptive throughout the study
- Patient is currently participating in another therapeutic study, or has previously participated in a therapeutic study within 30 days of screening/enrolment visit (or longer, if local requirements specify this)
- Patient has any condition or in any situation which, in the investigator's opinion, puts the patient at significant risk, could confound study results, or may interfere significantly with the patient's participation in the study
Trial locations and contacts
15 sites in 9 countries.
15 sites in 9 countries
Brazil3 sites
Hospital Nossa Senhora das Graças (HNSG)
Curitiba
Nucleo de Pesquisa e Desenvolvimento de Medicamentos - Universidade Federal do Ceará - Rodolfo Teófilo
Fortaleza, 60430-275
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo
France2 sites
Hospital mother children
Bron, 69677
Centre National de Référence Wilson, Hôpital Lariboisière
Paris, 75475
Germany2 sites
Innere Medizin
Heidelberg, 69120
Poliklinik Hepatologie/Transplantationsambulanz
Munich, 81377
Italy2 sites
A.O. San Paolo Milano
Milan, 20142
DiSCOG Gastroenterology Unit
Padova, 35128
United Kingdom2 sites
University of Surrey, Department of Clinical and Experimental Medicine
Guildford, Surrey, GU2 7XH
Leeds Teaching Hospitals NHS Trust
Leeds, LS9 7TF
United States1 site
Yale University School of Medicine
New Haven, Connecticut, 06510
Belgium1 site
KU Leuven, Department of Clinical and Experimental Medicine
Leuven
About this study
Official title: CHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease
This is a multicenter, randomized, open-label study with an active standard-of-care comparator (penicillamine)
Read the detailed description
This is a multicenter, randomized, open label study with an active standard-of-care comparator.
Stable patients who are already considered to be stable on their standard-of-care penicillamine chelation therapy for at least 1 year will enroll in the study and enter a 12-week Penicillamine Baseline Period comprising of 1 month (4 weeks) run-in period followed by a 2 month (8 weeks) evaluation period. During this time all patients will continue to take their current penicillamine under study conditions. At the end of the Penicillamine Baseline Period, patients who fulfill the protocol definition of being adequately controlled and tolerating penicillamine will be randomized in a 1:1 ratio to receive either TETA 4HCl or to continue to receive penicillamine. There is then a 24-week Post-randomization Phase comprising of a 1 month (4 weeks) run-in period for both treatment arms and a 5 month (20 weeks) evaluation period.
Patients who successfully complete the 24-week Post-randomization Phase of the study will have the opportunity to enter an Extension Phase. In the first version of the clinical trial protocol, the intention was to have an 18 month (72 weeks) Extension Phase. During the first 24 weeks of the Extension Phase, subjects would continue receiving their allocated TETA 4HCl or penicillamine (i.e., up to Week 60 of the study). Thereafter all patients were receiving TETA 4HCl for a further 48 weeks (i.e., from Week 60 to Week 108). Study clinic visits occur were scheduled every 6 months in the Extension Phase.
With the final version of the protocol, the Extension Phase stopped at Week 60. Patients who already passed the Week 60 visit were allowed to end the study at the next planned visit.
As a consequece end of treatment varied Week 60 and Week 108
- Conditions
- Wilson Disease
- Being tested
- Penicillamine (D1-W12), TETA 4HCL (W12-60), Penicillamine (W12-W60), TETA 4HCL (60-<W108)
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Penicillamine (D1-W12)Drug
Also called D-penicillamine
Penicillamine during baseline period (D1-W12)
TETA 4HCL (W12-60)Drug
Also called trientine tetrahydrochloride
TETA 4HCL during post randomisation and 1st extension period (W12-W60)
Penicillamine (W12-W60)Drug
Also called D-Penicillamine
Penicillamine during rondomisation and 1st extension period period (W12-W60)
TETA 4HCL (60-<W108)Drug
Also called trientine tetrahydrochloride
TETA 4HCL during 2nd extension period (W60-<W108)
| Study group | Role | What they receive |
|---|---|---|
| Penicillamine arm | Active comparator | Patients randomized to penicillamine during the postrandomisation and 1st extension period Receives: Drug: Penicillamine (D1-W12); Drug: Penicillamine (W12-W60); Drug: TETA 4HCL (60-<W108) |
| Trientine arm | Experimental | Patients randomized to TETA 4HCl during the postrandomisation and 1st extension period Receives: Drug: Penicillamine (D1-W12); Drug: TETA 4HCL (W12-60); Drug: TETA 4HCL (60-<W108) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum NCC Concentration
The primary outcome of efficacy was serum NCC by speciation assay (μg/L), with comparative analysis of mean difference between the two groups 24 weeks after randomization. The non-inferiority margin was set at -50 μg/L.
Measured: Week 36
Secondary outcomes (2)
24-hour Urinary Copper Excretion (UCE)
24-hour urinary copper excretion (μg/ 24 hr) from urine collected by the patient over a 24-hour period.
Measured: Week 36
Clinical Global Impression of Change (CGIC) Rating Scale
The clinician will rate the change in the patient's Wilson's disease relative to the prior study clinic visit using a 7-point scale to a specific statement: 'Please rate the change in the overall severity of the patients Wilson's disease compared to the previous study clinic visit".
Available options were (1) very much improved; (2) much improved; (3) minimally improved; (4) no change; (5) minimally worse; (6), much worse; or (7) very much worse.
Measured: Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2018
- Study start3 Sep 2018
- Primary completion19 Aug 2020
- Study completion18 Jan 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.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Wilson DiseaseStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orphalan
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03539952
- Sponsor's protocol ID
- GMPO-131-002
- Status last verified
- Jun 2025
- First submitted
- 4 May 2018
- Last updated
- 2 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Schilsky ML, Czlonkowska A, Zuin M, Cassiman D, Twardowschy C, Poujois A, Gondim FAA, Denk G, Cury RG, Ott P, Moore J, Ala A, D'Inca R, Couchonnal-Bedoya E, D'Hollander K, Dubois N, Kamlin COF, Weiss KH; CHELATE trial investigators. Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial. Lancet Gastroenterol Hepatol. 2022 Dec;7(12):1092-1102. doi: 10.1016/S2468-1253(22)00270-9. Epub 2022 Sep 30.
PubMed 36183738Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury RG, Czlonkowska A, Denk G, D'Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy CA, Weiss KH, Zuin M, Kamlin COF, Schilsky ML. Evaluation of novel assays of non-ceruloplasmin copper to monitor chelation treatment in patients with Wilson disease. JHEP Rep. 2026 Jun;8(6):101822. doi: 10.1016/j.jhepr.2026.101822. Epub 2026 Mar 12.
PubMed 41831608Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury RG, Czlonkowska A, Denk G, D'Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy CA, Weiss KH, Zuin M, Kamlin COF, Schilsky ML. Non-ceruloplasmin copper and urinary copper in clinically stable Wilson disease: Alignment with recommended targets. JHEP Rep. 2024 May 6;6(8):101115. doi: 10.1016/j.jhepr.2024.101115. eCollection 2024 Aug.
PubMed 39139457
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease
- ClinicalTrials.gov ID
- NCT03539952
- Acronym / sponsor ID
- GMPO-131-002
- Conditions
- Wilson Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- OrphalanIndustry (pharma, biotech, devices)
- Study start
- 3 Sep 2018 (actual)
- Primary completion
- 19 Aug 2020 (actual)
- Study completion
- 18 Jan 2022 (actual)
- First posted
- 30 May 2018 (actual)
- Results
- Posted 20 Apr 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, randomized, open-label study with an active standard-of-care comparator (penicillamine)
Detailed description
This is a multicenter, randomized, open label study with an active standard-of-care comparator.
Stable patients who are already considered to be stable on their standard-of-care penicillamine chelation therapy for at least 1 year will enroll in the study and enter a 12-week Penicillamine Baseline Period comprising of 1 month (4 weeks) run-in period followed by a 2 month (8 weeks) evaluation period. During this time all patients will continue to take their current penicillamine under study conditions. At the end of the Penicillamine Baseline Period, patients who fulfill the protocol definition of being adequately controlled and tolerating penicillamine will be randomized in a 1:1 ratio to receive either TETA 4HCl or to continue to receive penicillamine. There is then a 24-week Post-randomization Phase comprising of a 1 month (4 weeks) run-in period for both treatment arms and a 5 month (20 weeks) evaluation period.
Patients who successfully complete the 24-week Post-randomization Phase of the study will have the opportunity to enter an Extension Phase. In the first version of the clinical trial protocol, the intention was to have an 18 month (72 weeks) Extension Phase. During the first 24 weeks of the Extension Phase, subjects would continue receiving their allocated TETA 4HCl or penicillamine (i.e., up to Week 60 of the study). Thereafter all patients were receiving TETA 4HCl for a further 48 weeks (i.e., from Week 60 to Week 108). Study clinic visits occur were scheduled every 6 months in the Extension Phase.
With the final version of the protocol, the Extension Phase stopped at Week 60. Patients who already passed the Week 60 visit were allowed to end the study at the next planned visit.
As a consequece end of treatment varied Week 60 and Week 108
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Penicillamine arm | Active comparator | Drug: Penicillamine (D1-W12), Drug: Penicillamine (W12-W60), Drug: TETA 4HCL (60-<W108)Patients randomized to penicillamine during the postrandomisation and 1st extension period |
| Trientine arm | Experimental | Drug: Penicillamine (D1-W12), Drug: TETA 4HCL (W12-60), Drug: TETA 4HCL (60-<W108)Patients randomized to TETA 4HCl during the postrandomisation and 1st extension period |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Patient is able to provide, and has provided, written informed consent
- Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including: For US sites: Authorization for Use and Release of Health Research Study Information and for EU sites: Data Protection Consent
- Male or female, aged ≥ 18 and ≤ 75 years of age at time of consent
- Patient has a diagnosis of Wilson's disease, as defined by a prior or current Leipzig score of ≥ 4
- Patient's Wilson's disease is clinically stable, in the opinion of the investigator, and being treated with penicillamine for at least 1 year (52 weeks) prior to the screening/enrolment visit
Key exclusion criteria (21 listed)
- Patient is in 'de-coppering' phase of treatment for Wilson's disease, in the opinion of the investigator
- Patient evidence of uncontrolled liver disease, including but not limited to:
- Cause of patient's liver disease is due to another condition, in the investigator's opinion
- Patient has severe anemia defined as hemoglobin of ≤ 9 g/dL (result will be available after start of run-in period*)
- Patient has experienced a gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment visit
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 |
|---|---|---|
| Penicillamine (D1-W12)Also: D-penicillaminePenicillamine during baseline period (D1-W12) | Drug | Penicillamine arm, Trientine arm |
| TETA 4HCL (W12-60)Also: trientine tetrahydrochlorideTETA 4HCL during post randomisation and 1st extension period (W12-W60) | Drug | Trientine arm |
| Penicillamine (W12-W60)Also: D-PenicillaminePenicillamine during rondomisation and 1st extension period period (W12-W60) | Drug | Penicillamine arm |
| TETA 4HCL (60-<W108)Also: trientine tetrahydrochlorideTETA 4HCL during 2nd extension period (W60-<W108) | Drug | Penicillamine arm, Trientine arm |
Primary outcome
Serum NCC Concentration
Time frame: Week 36
The primary outcome of efficacy was serum NCC by speciation assay (μg/L), with comparative analysis of mean difference between the two groups 24 weeks after randomization. The non-inferiority margin was set at -50 μg/L.
2 secondary outcomes
24-hour Urinary Copper Excretion (UCE)
Time frame: Week 36
Clinical Global Impression of Change (CGIC) Rating Scale
Time frame: Week 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 36183738Schilsky ML, Czlonkowska A, Zuin M, Cassiman D, Twardowschy C, Poujois A, Gondim FAA, Denk G, Cury RG, Ott P, Moore J, Ala A, D'Inca R, Couchonnal-Bedoya E, D'Hollander K, Dubois N, Kamlin COF, Weiss KH; CHELATE trial investigators. Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial. Lancet Gastroenterol Hepatol. 2022 Dec;7(12):1092-1102. doi: 10.1016/S2468-1253(22)00270-9. Epub 2022 Sep 30.
- Derived from this study: PubMed 41831608Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury RG, Czlonkowska A, Denk G, D'Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy CA, Weiss KH, Zuin M, Kamlin COF, Schilsky ML. Evaluation of novel assays of non-ceruloplasmin copper to monitor chelation treatment in patients with Wilson disease. JHEP Rep. 2026 Jun;8(6):101822. doi: 10.1016/j.jhepr.2026.101822. Epub 2026 Mar 12.
- Derived from this study: PubMed 39139457Ott P, Sandahl T, Ala A, Cassiman D, Couchonnal-Bedoya E, Cury RG, Czlonkowska A, Denk G, D'Inca R, de Assis Aquino Gondim F, Moore J, Poujois A, Twardowschy CA, Weiss KH, Zuin M, Kamlin COF, Schilsky ML. Non-ceruloplasmin copper and urinary copper in clinically stable Wilson disease: Alignment with recommended targets. JHEP Rep. 2024 May 6;6(8):101115. doi: 10.1016/j.jhepr.2024.101115. eCollection 2024 Aug.
- 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.
15 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Penicillamine (D1-W12), TETA 4HCL (W12-60) and Penicillamine (W12-W60) in people with Wilson Disease, run by Orphalan at 15 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 23 inclusion and 21 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 2 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Brazil, France, Germany, Italy, United Kingdom 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 20 Apr 2023). 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?
Serum NCC Concentration (time frame: Week 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03539952, which also lists 3 linked publications.
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 2 Jul 2025.