Amyotrophic Lateral SclerosisPhase 3NCT03836716
Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
A Phase 3 study testing Arimoclomol in people with Amyotrophic Lateral Sclerosis, run by ZevraDenmark at 23 sites in 11 countries.
Trial summary
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Arimoclomol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 23 sitesin 11 countries
- Study dates
- 19 Sep 2019to 2 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
- Subject is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures, or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures
- Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment period)
- Subject completed ORARIALS-01 while on treatment, where on treatment is defined as having taken the last dose of IMP within 2 weeks of the End of Trial visit (whether at week 76 or prior)
Who cannot join
- Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
- Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
- Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.
- Any of the following medically significant conditions:
- Clinically significant renal or hepatic disease OR clinical laboratory assessment (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase and/or alanine aminotransferase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5 times the ULN).
- Any new condition or worsening of existing condition which, in the opinion of the investigator, would put the subject at undue risk.
- Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP
Trial locations and contacts
23 sites in 11 countries.
23 sites in 11 countries
United States7 sites
University of Virginia Health System
Charlottesville, Virginia, 22908
University of Miami
Miami, Florida, 33136
Hospital for Special Surgery
New York, New York, 10021
UC Irvine Health ALS and Neuromuscular Center
Orange, California, 92868
University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center
Philadelphia, Pennsylvania, 19107
St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
Phoenix, Arizona, 85013
Providence Brain & Spine Institute
Portland, Oregon, 97213
Germany3 sites
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen
Berlin, 13353
Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie
Hanover, 30625
Universitaetsklinikum Ulm - Klinik fuer Neurologie
Ulm, 89081
France2 sites
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac
Montpellier, 34295
Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422
Paris, 75013
Italy2 sites
Instituti Clinica Scientifici Maugeri
Milan, 20138
Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino
Torino, 10126
Poland2 sites
Centrum Medyczne NeuroProtect
Warsaw, 01-684
Citi Clinic
Warsaw, 02-473
Spain2 sites
Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11
Barcelona, 08035
Hospital Carlos III - Hospital Universitario La Paz, ALS Unit
Madrid, 28046
United Kingdom1 site
Leonard Wolfson Experimental Neurology Centre
London, WC1N 3BG
About this study
Official title: Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.
Read the detailed description
The planned duration of the open-label trial was 152 weeks, but the trial was terminated early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28 weeks (range approximately 2 to 71 weeks).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Arimoclomol
- Keywords
- Arimoclomol, ALS
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ArimoclomolDrug
2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily
| Study group | Role | What they receive |
|---|---|---|
| Arimoclomol | Experimental | 248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily Receives: Drug: Arimoclomol |
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 (TEAEs) Over the Open-label Treatment Period
Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks.
Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration >14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals.
Measured: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1)
Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2)
Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes
Standard hematology parameter.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit
Standard hematology parameter.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin
Standard hematology parameter.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1)
Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2)
Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3)
Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein
Standard clinical chemistry parameters.
Measured: Week 76
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C
Standard clinical chemistry parameter.
Measured: Week 76
Mean and Change From Baseline in Vital Signs - Blood Pressure
Standard vital signs. Systolic and diastolic blood pressure.
Measured: Week 76
Mean and Change From Baseline in Vital Signs - Pulse Rate
Standard vital signs measurement.
Measured: Week 76
Mean and Change From Baseline in Vital Signs - Respiratory Rate
Standard vital signs measurement.
Measured: Week 76
Mean and Change From Baseline in Vital Signs - Temperature
Standard vital signs measurement.
Measured: Week 76
Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period
Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks.
Measured: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period
The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate.
Measured: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
Secondary outcomes (2)
Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76
The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence.
Measured: Week 76
Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial)
Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:
- If male: Predicted SVC = 0.061 x height (cm) - 0.028 x age (years) - 4.65
- If female: Predicted SVC = 0.0466 x height (cm) - 0.024 x age (years) - 3.28
Measured: 76 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2019
- Study start19 Sep 2019
- Primary completion2 Jul 2021
- Study completion2 Jul 2021
Why it stopped: As a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. The planned duration was 152 weeks. After termination, the actual mean duration of treatment was approx. 28 weeks (range approx. 2 to 71 weeks).
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 Amyotrophic Lateral SclerosisStudies 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
- Not applicable
- Intervention model
- Single group
- Model description
- Non-randomized open label
- Masking
- None (open label)
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- ZevraDenmark
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Benatar, MD PhD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03836716
- Sponsor's protocol ID
- ORARIALS-02
- Status last verified
- Aug 2023
- First submitted
- 7 Feb 2019
- Last updated
- 24 Aug 2023
- Results posted
- Yes, on ClinicalTrials.gov
- 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 24 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial
- ClinicalTrials.gov ID
- NCT03836716
- Acronym / sponsor ID
- ORARIALS-02
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Arimoclomol, ALS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: As a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. The planned duration was 152 weeks. After termination, the actual mean duration of treatment was approx. 28 weeks (range approx. 2 to 71 weeks).
- Enrolment
- 120 participants (actual)
- Lead sponsor
- ZevraDenmarkIndustry (pharma, biotech, devices)
- Study start
- 19 Sep 2019 (actual)
- Primary completion
- 2 Jul 2021 (actual)
- Study completion
- 2 Jul 2021 (actual)
- First posted
- 11 Feb 2019 (actual)
- Results
- Posted 24 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.
Detailed description
The planned duration of the open-label trial was 152 weeks, but the trial was terminated early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28 weeks (range approximately 2 to 71 weeks).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Non-randomized open label
| Arm | Type | Receives |
|---|---|---|
| Arimoclomol | Experimental | Drug: Arimoclomol248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Subject is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures, or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures
- Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment period)
- Subject completed ORARIALS-01 while on treatment, where on treatment is defined as having taken the last dose of IMP within 2 weeks of the End of Trial visit (whether at week 76 or prior)
Key exclusion criteria (7 listed)
- Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
- Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
- Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.
- Any of the following medically significant conditions:
- Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP
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 |
|---|---|---|
| Arimoclomol2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily | Drug | Arimoclomol |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period
Time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks. Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration >14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1)
Time frame: Week 76
Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2)
Time frame: Week 76
Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes
Time frame: Week 76
Standard hematology parameter.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit
Time frame: Week 76
Standard hematology parameter.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin
Time frame: Week 76
Standard hematology parameter.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1)
Time frame: Week 76
Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2)
Time frame: Week 76
Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3)
Time frame: Week 76
Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein
Time frame: Week 76
Standard clinical chemistry parameters.
Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C
Time frame: Week 76
Standard clinical chemistry parameter.
Mean and Change From Baseline in Vital Signs - Blood Pressure
Time frame: Week 76
Standard vital signs. Systolic and diastolic blood pressure.
Mean and Change From Baseline in Vital Signs - Pulse Rate
Time frame: Week 76
Standard vital signs measurement.
Mean and Change From Baseline in Vital Signs - Respiratory Rate
Time frame: Week 76
Standard vital signs measurement.
Mean and Change From Baseline in Vital Signs - Temperature
Time frame: Week 76
Standard vital signs measurement.
Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period
Time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks.
Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period
Time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate.
2 secondary outcomes
Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76
Time frame: Week 76
Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial)
Time frame: 76 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Michael Benatar, MD PhD principal investigator
University of Miami
23 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Arimoclomol in people with Amyotrophic Lateral Sclerosis, run by ZevraDenmark at 23 sites in 11 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 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Aug 2023. 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 United States, Germany, France, Italy, Poland and 6 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 24 Aug 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?
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period (time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks). The study lists 17 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 24 Aug 2023.