Neuromyelitis OpticaPhase 3NCT02003144
An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
A Phase 3 study testing eculizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 69 sites in 20 countries.
Trial summary
The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 119 participants
- Where
- 69 sitesin 20 countries
- Study dates
- 12 Jan 2015to 12 Jul 2021
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient completed the ECU-NMO-301 trial
- Patient has given written informed consent
Who cannot join
- Patients who have withdrawn from the ECU-NMO-301 trial as a result of an AE related to trial drug
- Female patients who are pregnant, breastfeeding, or intend to conceive during the course of the trial
Trial locations and contacts
69 sites in 20 countries.
69 sites in 20 countries
United States19 sites
Johns Hopkins University Medical Center
Baltimore, Maryland, 21287
Ohio Health Reserach Institute
Columbus, Ohio, 43214
Multiple Sclerosis Treatment Center of Dallas
Dallas, Texas, 75246
Allied Physicians Inc. of Fort Wayne
Fort Wayne, Indiana, 46805
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Miami McKnight Brain Institute
Miami, Florida, 33136
Multiple Sclerosis Comprehensive Care Center NYU Langone Medical Center
New York, New York, 10016
Mount Sinai School of Medicine
New York, New York, 10029
Baptist Health Lexington
Nicholasville, Kentucky, 40503
The Research Center of Southern California
Oceanside, California, 92056
Neurological Services of Orlando
Orlando, Florida, 32806
University of Pennsylvania School of Medicine
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15232
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
University of Utah
Salt Lake City, Utah, 84108
The University of Texas Health Science
San Antonio, Texas, 78229
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
Washington University School of Medicine
St Louis, Missouri, 63110
Georgetown University Hospital
Washington D.C., District of Columbia, 20007
Japan7 sites
Chiba University Hospital
Chiba, Chiba, 260-8677
Kyushu University Hospital
Fukuoka, 812-8582
Kyoto Min-iren Chuo Hospital
Kyoto, Kyoto, 604-8453
Hyogo College of Medicine Hospital
Nishinomiya-shi, HyogoKen
Tohoku University Hospital
Sendai, Miyagi, 980-8574
National Center Hospital, NCNP
Tokyo
Yamaguchi University Hospital
Ube-shi, Yamaguchi, 755-8505
South Korea5 sites
National Cancer Center
Goyang-si, Gyeonggi-do, 410-769
Seoul University National Hospital
Seoul, 110744
Samsung Medical Center
Seoul, 135-710
Korea University Anam Hospital
Seoul, 136-705
Severance Hospital, Yonsei University
Seoul, 136-705
Turkey (Türkiye)5 sites
Hacettepe University Medical Faculty
Ankara, 06100
Trakya University Medical Faculty
Edirne
Dokuz Eylul University Medicine Faculty
Izmir, 35340
Kocaeli University Medical Faculty
Kocaeli
Ondokuz Mayis Univ. Med. Fac.
Samsun
Argentina4 sites
Hospital General de Agudos Juan Antonio Fernandez
Ciudad Autonoma, Buenos Aires, C1425AGP
Hospital General de Agudos Dr. J. M. Ramos Mejia
Ciudad Autonoma, Buenos Aires,, C1221ADC
Hospital Universitario Austral
Pilar, Buenos Aires, B1629ODT
Fundacion Rosarina de Neuro Rehabilitacion
Rosario, Santa Fe Province, S2000BZL
Germany4 sites
University Hospital Heinrich Heine University
Düsseldorf, North Rhine-Westphalia, 40225
University Hospital Heidelberg
Heidelberg, Baden-Wurttemberg, 69120
Neurologische Klinik und Poliklinik
Munich, Bavaria, 81675
Universitaetsmedizin Rostock
Rostock, 18147
Italy4 sites
Policlinico di Catania
Catania, 95123
Azienda Ospedaliera Universitaria
Naples, 80131
Azienda Ospedaliera di Padova
Padova, 35128
Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
Rome, 00178
Russia4 sites
Republican Clinical Hospital for Rehabilitation of Healthcare Ministry of Republic of Tatarstan
Kazan', 420021
SBEI "Krasnoyarsk SMU n.a. Prof. V.F. Voyno-Yasenetsky"
Krasnoyarsk, 660037
Federal State Budget Institution of Healthcare - Siberian District Medical Center of FMBA of Russia
Novosibirsk, 630068
SEIHPE "Rostov SMU of MoH of RF"
Rostov-on-Don
Spain3 sites
Hospital de Cruces
Barakaldo, Bizkaia, 48903
Hospital Universitario Reina Sofia
Córdoba, 14404
Hospital Universitario Clinico San Carlos
Madrid, 28040
Australia2 sites
Brain and Mind Research Institute
Camperdown, New South Wales
St. Vincent's Hospital
Fitzroy, Victoria, 3065
Czechia2 sites
VFN v Praze
Prague
Krajska zdravotni, a.s. - Nemocnice
Teplice, 415 01
Malaysia2 sites
Hospital Kuala Lumpur
Kuala Lumpur, 50586
Hospital Umum Sarawak
Kuching, Sarawak, 93586
Colombia1 site
Fundacion Cardiovascular de Colombia
Floridablanca, Santander Department
About this study
Official title: A Phase III, Open-label, Extension Trial of ECU-NMO-301 to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.
Read the detailed description
This study is an open label extension study to confirm the long term safety and efficacy of eculizumab in subjects with relapsing NMO who have completed the initial double-blind, randomized, placebo-controlled trial ECU-NMO-301.
- Being tested
- eculizumab
- Keywords
- Long-term safety study, Extension trial, Eculizumab, Neuromyelitis Optica Spectrum Disorder, Devic's disease, Transverse Myelitis, Optic Neuritis, Relapse, NMO-IgG, CNS Autoimmune Disorders, Demyelinating Disorders
- Medical subject headings
- Neuromyelitis Optica, Myelitis, Transverse, Optic Neuritis, Recurrence, Demyelinating Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eculizumabBiological
Also called Soliris
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | Eculizumab intravenous infusion every two weeks. Receives: Biological: eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Measured: Baseline up to end of study (up to 6.5 years)
Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality
The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent.
Measured: Baseline up to end of study (up to 6.5 years)
Number of Participants With An On-trial Relapse as Determined by The Treating Physician
An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician.
Measured: Baseline up to end of study (up to 6.5 years)
On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician
The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period.
Measured: Baseline up to end of study (up to 6.5 years)
Secondary outcomes (5)
Change From Baseline in Expanded Disability Status Scale (EDSS) Score
Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Measured: Baseline, Weeks 52, 104 and 156
Change From Baseline in Modified Rankin Scale (mRS) Score
Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Measured: Baseline, Weeks 52, 104 and 156
Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function
The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Measured: Baseline, Weeks 52, 104 and 156
Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score
The EQ-5D is a generic, standardized participant self-administered health status instrument. EQ-5D general health status can also be measured by a visual analog scale (EQ-5D VAS). EQ-5D-VAS recorded the participant's self-rated health on a vertical visual analog scale (VAS) that allowed the participants to indicate their health state that ranged from 0 (worst imaginable) to 100 (best imaginable). Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302.
Measured: Baseline, Weeks 52, 104 and 156
Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function
The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302.
Measured: Baseline, Weeks 52, 104 and 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2013 (estimated)
- Study start12 Jan 2015
- Primary completion12 Jul 2021
- Study completion12 Jul 2021
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 Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 119 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02003144
- Sponsor's protocol ID
- ECU-NMO-302
- Other IDs
- 2013-001151-12
- Status last verified
- Jul 2022
- First submitted
- 18 Nov 2013
- Last updated
- 23 Aug 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Pittock SJ, Lennon VA, McKeon A, Mandrekar J, Weinshenker BG, Lucchinetti CF, O'Toole O, Wingerchuk DM. Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study. Lancet Neurol. 2013 Jun;12(6):554-62. doi: 10.1016/S1474-4422(13)70076-0. Epub 2013 Apr 26.
PubMed 23623397Pittock SJ, Fujihara K, Palace J, Berthele A, Kim HJ, Oreja-Guevara C, Nakashima I, Levy M, Shang S, Yountz M, Miller L, Armstrong R, Wingerchuk DM; PREVENT Study Group. Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension. Mult Scler. 2022 Mar;28(3):480-486. doi: 10.1177/13524585211038291. Epub 2021 Sep 9.
PubMed 34498507Kim HJ, Nakashima I, Viswanathan S, Wang KC, Shang S, Miller L, Yountz M, Wingerchuk DM, Pittock SJ, Levy M, Berthele A, Totolyan N, Palace J, Barnett MH, Fujihara K; PREVENT Study Group. Eculizumab in Asian patients with anti-aquaporin-IgG-positive neuromyelitis optica spectrum disorder: A subgroup analysis from the randomized phase 3 PREVENT trial and its open-label extension. Mult Scler Relat Disord. 2021 May;50:102849. doi: 10.1016/j.msard.2021.102849. Epub 2021 Feb 20.
PubMed 33676197
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Open-label, Extension Trial of ECU-NMO-301 to Evaluate the Safety and Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)
- ClinicalTrials.gov ID
- NCT02003144
- Acronym / sponsor ID
- ECU-NMO-302
- Keywords
- Long-term safety study, Extension trial, Eculizumab, Neuromyelitis Optica Spectrum Disorder, Devic's disease, Transverse Myelitis, Optic Neuritis, Relapse
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Jan 2015 (actual)
- Primary completion
- 12 Jul 2021 (actual)
- Study completion
- 12 Jul 2021 (actual)
- First posted
- 6 Dec 2013 (estimated)
- Results
- Posted 23 Aug 2022
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 determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.
Detailed description
This study is an open label extension study to confirm the long term safety and efficacy of eculizumab in subjects with relapsing NMO who have completed the initial double-blind, randomized, placebo-controlled trial ECU-NMO-301.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Biological: eculizumabEculizumab intravenous infusion every two weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patient completed the ECU-NMO-301 trial
- Patient has given written informed consent
Key exclusion criteria (2 listed)
- Patients who have withdrawn from the ECU-NMO-301 trial as a result of an AE related to trial drug
- Female patients who are pregnant, breastfeeding, or intend to conceive during the course of the trial
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 |
|---|---|---|
| eculizumabAlso: Soliris | Biological | Eculizumab |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Time frame: Baseline up to end of study (up to 6.5 years)
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Treatment-emergent adverse events (TEAEs) were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Number of Participants With At Least 1 Post Baseline Columbia-Suicide Severity Rating Scale (C-SSRS) Assessment (Suicide-Related Thoughts or Behaviours) Abnormality
Time frame: Baseline up to end of study (up to 6.5 years)
The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent.
Number of Participants With An On-trial Relapse as Determined by The Treating Physician
Time frame: Baseline up to end of study (up to 6.5 years)
An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician.
On-Trial Annualized Relapse Rate (ARR) as Determined by The Treating Physician
Time frame: Baseline up to end of study (up to 6.5 years)
The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period.
5 secondary outcomes
Change From Baseline in Expanded Disability Status Scale (EDSS) Score
Time frame: Baseline, Weeks 52, 104 and 156
Change From Baseline in Modified Rankin Scale (mRS) Score
Time frame: Baseline, Weeks 52, 104 and 156
Change From Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function
Time frame: Baseline, Weeks 52, 104 and 156
Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Visual Analog Scale (EQ-5D VAS) Health Status Score
Time frame: Baseline, Weeks 52, 104 and 156
Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function
Time frame: Baseline, Weeks 52, 104 and 156
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23623397Pittock SJ, Lennon VA, McKeon A, Mandrekar J, Weinshenker BG, Lucchinetti CF, O'Toole O, Wingerchuk DM. Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study. Lancet Neurol. 2013 Jun;12(6):554-62. doi: 10.1016/S1474-4422(13)70076-0. Epub 2013 Apr 26.
- Derived from this study: PubMed 34498507Pittock SJ, Fujihara K, Palace J, Berthele A, Kim HJ, Oreja-Guevara C, Nakashima I, Levy M, Shang S, Yountz M, Miller L, Armstrong R, Wingerchuk DM; PREVENT Study Group. Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension. Mult Scler. 2022 Mar;28(3):480-486. doi: 10.1177/13524585211038291. Epub 2021 Sep 9.
- Derived from this study: PubMed 33676197Kim HJ, Nakashima I, Viswanathan S, Wang KC, Shang S, Miller L, Yountz M, Wingerchuk DM, Pittock SJ, Levy M, Berthele A, Totolyan N, Palace J, Barnett MH, Fujihara K; PREVENT Study Group. Eculizumab in Asian patients with anti-aquaporin-IgG-positive neuromyelitis optica spectrum disorder: A subgroup analysis from the randomized phase 3 PREVENT trial and its open-label extension. Mult Scler Relat Disord. 2021 May;50:102849. doi: 10.1016/j.msard.2021.102849. Epub 2021 Feb 20.
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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.
69 sites in 20 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing eculizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Alexion Pharmaceuticals, Inc. at 69 sites in 20 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 2 inclusion and 2 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 23 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 69 sites in United States, Japan, South Korea, Turkey (Türkiye), Argentina and 15 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 23 Aug 2022). 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?
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events (time frame: Baseline up to end of study (up to 6.5 years)). The study lists 4 primary outcomes in all. 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 NCT02003144, 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 23 Aug 2022.