Raynaud Phenomenon Secondary to Systemic SclerosisPhase 3BRASSNCT03717961
Efficacy of Botulinum Toxin A in Adult Subjects With Raynaud Phenomenon Secondary to Systemic Sclerosis
A Phase 3 study testing BOTOX® solution in people with Raynaud Phenomenon Secondary to Systemic Sclerosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
This study aims to assess whether or not a single injection schedule of botulinum toxin A (BTX-A) in both hands improves Raynaud phenomenon (RP) secondary to systemic sclerosis (SSc) better than a placebo at 4, 12 and 24 weeks after the treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Raynaud Phenomenon Secondary to Systemic Sclerosis
- What is being tested?
- BOTOX® solution
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 91 participants
- Where
- 1 siteFrance
- Study dates
- 15 Oct 2018to 31 Jul 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Aged 18 years and older
- Diagnosed with systemic sclerosis (EULAR/ACR 2013 criteria ; or Leroy and Metsger criteria).
- Symptoms of Raynaud's phenomenon affecting both hands (not necessarily to equal extents)
- Frequency of Raynaud's attacks ≥ 5/week during cold weather
- Stable dose of phosphodiesterase inhibitors (sildenafil, tadalafil or vardenafil), endothelin antagonists, or calcium channel blockers defined as 1-month with no change in dose
- Ability to return/be available for follow-up evaluations
Show all 9Show fewer
- Ability to fill the diary
- Ability/willingness to give informed consent
- Affiliation to any French social security regime
Who cannot join
- History of myasthenia gravis or Eaton Lambert syndrome
- Inflammatory myositis <2 years or pre-existing motor neurone disease or upper limb motor neuropathy
- Reported allergy or hypersensitivity to any Botulinum toxin preparation or to lidocaine or other local anesthetic agent or to albumin or to inhaled nitrous oxide/oxygen.
- Active infection in either hand.
- Pregnant or lactating women (women of child bearing potential must undergo a urine pregnancy teste before inclusion and at treatment day).
- women of child bearing potential without medically accepted method of birth control
Show all 9Show fewer
- Patients who have previously undergone any vascular surgery on the upper extremity, including surgical sympathectomy or ever received botulinum toxin or planned to receive botulinum toxin in the next 6 months
- Cognitive impairment
- Iloprost scheduled the month following injections
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Multicenter, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Study to Assess Efficacy of Botulinum Toxin A in Adult Subjects With Raynaud Phenomenon Secondary to Systemic Sclerosis
This study aims to assess whether or not a single injection schedule of botulinum toxin A (BTX-A) in both hands improves Raynaud phenomenon (RP) secondary to systemic sclerosis (SSc) better than a placebo at 4, 12 and 24 weeks after the treatment.
This study's hypothesis is that the number of RP attacks per week from baseline to 4 weeks after treatment is significantly lower in the group treated with BTX-A than in the control group treated by the placebo. Furthermore, BTX-A in both hands is expected to improve both symptomatic (attack frequency, digital ulcer healing) and functional (pain, hand function, quality of life) symptoms of RP secondary to SSc more than placebo.
Read the detailed description
In SSc, RP is present in 95% of the patients. Despite cold avoidance, RP attacks are severe, painful, affecting the quality of life, with frequent severe ischemic symptoms such as digital ulcers. Treating patients with RP secondary to SSc is extremely challenging. Oral calcium channel blockers, the most commonly prescribed drugs, were shown to be minimally effective in reducing frequency and severity of RP attacks. There is limited evidence for the efficacy of alpha-1-adrenergic receptor antagonists, angiotensin receptor blockers, phosphodiesterase inhibitors and endothelin-1 receptor antagonists to treat RP. The intravenously administered prostacyclin analogue iloprost is considered a second-line therapy, after oral calcium channel blockers, but its administration requires a 5-days hospitalisation for progressive increase in dosage and close blood pressure monitoring. Side effects are almost constant: headache, flushing, malaise and hypotension. Between 2004 and 2015, a total of 145 patients underwent botulinum toxin-A (BTX-A) injections to treat RP in case reports, retrospective or open studies. Since 2016, only 1 randomized, controlled study was performed, including 40 patients but was underpowered, not designed for clinical outcomes, and treated the patient only in one hand. Symptomatic patients treated by BTX-A experienced, in the 3 to 6 following months, pain relief in 75-100% of the cases, as well as reduction in severity and frequency of attacks and increased ulcer healing rate, with minimal adverse effects. The most common complication was temporary hand weakness, related to the injected dose. When the total dose/hand was 50 UI or lower, the rate of hand weakness was 0 to 9%. The proposed study is the first randomized double blinded multicenter clinical trial designed to assess whether or not a single injection schedule of BTX-A in both hands improves RP secondary to SSc better than a placebo at 4, 12 and 24 weeks after the treatment.
- Being tested
- BOTOX® solution, Placebo group
- Keywords
- Raynaud Phenomenon, Systemic Sclerosis, Botulinum toxin A
- Medical subject headings
- Raynaud Disease, Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BOTOX® solutionDrug
Botox solution + Saline serum + lidocaine/prilocaine cream + Nitrous oxide/oxygen (50%/50%)
Placebo groupDrug
Placebo + lidocaine/prilocaine cream + Nitrous oxide/oxygen (50%/50%)
| Study group | Role | What they receive |
|---|---|---|
| " BTX-A" group | Experimental | * BOTOX® solution
* Saline serum
* lidocaine/prilocaine cream
* Nitrous oxide/oxygen (50%/50%)
Intervention Description :
* BOTOX 100 UNITES (Allergan, Courbevoie, France, and São Paulo, Brazil) Single injection schedule of BOTOX® in both hands, diluted in 2 ml of sterile serum saline, with a dosage of 50 UI (1 ml) by hand. Injections will be performed at the level of the distal palmar crease, targeting the neurovascular bundles in the 4 web spaces (0.25 ml/injection site)
* between 2 to 3 hours before the procedure, application of lidocaine/prilocaine cream under an occlusive dressing (polyurethane film) in each palmar crease of the patients (1/2 tube/hand), according to the recommendations of the manufacturer
* inhaled nitrous oxide/oxygen (50% / 50%) may be used, if needed Receives: Drug: BOTOX® solution |
| Placebo group | Placebo comparator | * Saline serum
* lidocaine/prilocaine cream
* Nitrous oxide/oxygen (50%/50%)
Intervention Description :
* Sterile saline solution Single injection schedule of 2 ml (1 ml by hand) of serum saline, in both hands. Injections will be performed at the level of the distal palmar crease, targeting the neurovascular bundles in the 4 web spaces (0.25 ml/injection site).
* between 2 to 3 hours before the procedure, application of lidocaine/prilocaine cream under an occlusive dressing (polyurethane film) in each palmar crease of the patients (1/2 tube/hand), according to the recommendations of the manufacturer of the lidocaine cream.
* inhaled nitrous oxide/oxygen (50% / 50%) may be used, if needed Receives: Drug: Placebo group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute change from baseline in the number of RP attacks per week at 4 weeks
Comparison between the 2 groups of the change (absolute) from baseline in the number of RP attacks per week at 4 weeks. An attack is defined as an episode of pallor or cyanosis (with or without pain, tingling or numbness). Subjects will keep a daily record of the number of RP attacks they experienced per day and the duration of each attack, from Day -14 to Day 0 (+/- 4 days) and from Day 14 to Day 28 ( +/- 4 days)
Measured: 4 weeks
Secondary outcomes (9)
Absolute change from baseline in the number of RP attacks per week at 12 and 24 weeks
Comparison between the 2 groups of the change (absolute) from baseline in the number of RP attacks per week at 12 and 24 weeks. Subjects kept a daily record of the number of RP attacks they experienced per day and the duration of each attack, over a 14-day +/- 4 days period before each assessment visit at 12 and 24 weeks
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the percentage of digital ulcers with complete healing
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the percentage of digital ulcers with complete healing
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the number of new digital ulcers.
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the number of new digital ulcers.
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in Raynaud's pain score.
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in Raynaud's pain score. Pain associated with each attack is self-reported on a 10-point scale, and averaged over 14-day +/- 4 days period before each assessment visit
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in Raynaud's Condition Score.
Comparison at 4, 12 and 24 weeks of follow-up after the treatment of the change in Raynaud's Condition Score. Raynaud Condition Score is a daily self-assessment of RP activity using a 0-10 ordinal scale and averaged over 14-day +/- 4 days period before each assessment visit. The RCS incorporates the cumulative daily frequency, duration, severity and impact of RP attacks.
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in quality of life from baseline to weeks 4, 12 and 24
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in quality of life from baseline to weeks 4, 12 and 24. QOL is assessed by the Health Assessment Questionnaire (HAQ)
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in hand function from baseline to weeks 4, 12 and 24
Comparison between the 2 groups from baseline to 4, 12 and 24 weeks of follow-up after the treatment of the change in hand function measured by Quick-DASH (Disabilities of the Arm, Shoulder and Hand)
Measured: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in hand function from baseline to weeks 4, 12 and 24
Comparison between the 2 groups from baseline to at 4, 12 and 24 weeks of follow-up after the treatment of the change in hand function measured by the Hand Function Scale of Cochin
Measured: 24 weeks
Frequency and severity of adverse drug events during treatment from baseline to weeks 4, 12 and 24.
Frequency and severity of adverse drug events during treatment from baseline to weeks 4, 12 and 24.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2018
- Study start15 Oct 2018
- Primary completion31 Jul 2020
- Study completion31 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Raynaud Phenomenon Secondary to Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Multicenter, double-blind, randomized, placebo-controlled, parallel groups, phase III
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Patricia Senet, MD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03717961
- Sponsor's protocol ID
- P170904J
- Acronym
- BRASS
- Status last verified
- Feb 2022
- First submitted
- 15 Oct 2018
- Last updated
- 10 Feb 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Senet P, Maillard H, Diot E, Lazareth I, Blaise S, Arnault JP, Pistorius MA, Boulon C, Cogrel O, Warzocha U, Riviere S, Malloizel-Delaunay J, Servettaz A, Sassolas B, Viguier M, Monfort JB, Janique S, Vicaut E; BRASS collaborators. Efficacy and Safety of Botulinum Toxin in Adults with Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study. Arthritis Rheumatol. 2023 Mar;75(3):459-467. doi: 10.1002/art.42342. Epub 2022 Dec 14.
PubMed 36066501
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Study to Assess Efficacy of Botulinum Toxin A in Adult Subjects With Raynaud Phenomenon Secondary to Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT03717961
- Acronym / sponsor ID
- BRASS / P170904J
- Keywords
- Raynaud Phenomenon, Systemic Sclerosis, Botulinum toxin A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 91 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 15 Oct 2018 (actual)
- Primary completion
- 31 Jul 2020 (actual)
- Study completion
- 31 Jul 2020 (actual)
- First posted
- 24 Oct 2018 (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.
This study aims to assess whether or not a single injection schedule of botulinum toxin A (BTX-A) in both hands improves Raynaud phenomenon (RP) secondary to systemic sclerosis (SSc) better than a placebo at 4, 12 and 24 weeks after the treatment.
This study's hypothesis is that the number of RP attacks per week from baseline to 4 weeks after treatment is significantly lower in the group treated with BTX-A than in the control group treated by the placebo. Furthermore, BTX-A in both hands is expected to improve both symptomatic (attack frequency, digital ulcer healing) and functional (pain, hand function, quality of life) symptoms of RP secondary to SSc more than placebo.
Detailed description
In SSc, RP is present in 95% of the patients. Despite cold avoidance, RP attacks are severe, painful, affecting the quality of life, with frequent severe ischemic symptoms such as digital ulcers. Treating patients with RP secondary to SSc is extremely challenging. Oral calcium channel blockers, the most commonly prescribed drugs, were shown to be minimally effective in reducing frequency and severity of RP attacks. There is limited evidence for the efficacy of alpha-1-adrenergic receptor antagonists, angiotensin receptor blockers, phosphodiesterase inhibitors and endothelin-1 receptor antagonists to treat RP. The intravenously administered prostacyclin analogue iloprost is considered a second-line therapy, after oral calcium channel blockers, but its administration requires a 5-days hospitalisation for progressive increase in dosage and close blood pressure monitoring. Side effects are almost constant: headache, flushing, malaise and hypotension. Between 2004 and 2015, a total of 145 patients underwent botulinum toxin-A (BTX-A) injections to treat RP in case reports, retrospective or open studies. Since 2016, only 1 randomized, controlled study was performed, including 40 patients but was underpowered, not designed for clinical outcomes, and treated the patient only in one hand. Symptomatic patients treated by BTX-A experienced, in the 3 to 6 following months, pain relief in 75-100% of the cases, as well as reduction in severity and frequency of attacks and increased ulcer healing rate, with minimal adverse effects. The most common complication was temporary hand weakness, related to the injected dose. When the total dose/hand was 50 UI or lower, the rate of hand weakness was 0 to 9%. The proposed study is the first randomized double blinded multicenter clinical trial designed to assess whether or not a single injection schedule of BTX-A in both hands improves RP secondary to SSc better than a placebo at 4, 12 and 24 weeks after the treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Multicenter, double-blind, randomized, placebo-controlled, parallel groups, phase III
| Arm | Type | Receives |
|---|---|---|
| " BTX-A" group | Experimental | Drug: BOTOX® solution* BOTOX® solution * Saline serum * lidocaine/prilocaine cream * Nitrous oxide/oxygen (50%/50%) Intervention Description : * BOTOX 100 UNITES (Allergan, Courbevoie, France, and São Paulo, Brazil) Single injection schedule of BOTOX® in both hands, diluted in 2 ml of sterile serum saline, with a dosage of 50 UI (1 ml) by hand. Injections will be performed at the level of the distal palmar crease, targeting the neurovascular bundles in the 4 web spaces (0.25 ml/injection site) * between 2 to 3 hours before the procedure, application of lidocaine/prilocaine cream under an occlusive dressing (polyurethane film) in each palmar crease of the patients (1/2 tube/hand), according to the recommendations of the manufacturer * inhaled nitrous oxide/oxygen (50% / 50%) may be used, if needed |
| Placebo group | Placebo comparator | Drug: Placebo group* Saline serum * lidocaine/prilocaine cream * Nitrous oxide/oxygen (50%/50%) Intervention Description : * Sterile saline solution Single injection schedule of 2 ml (1 ml by hand) of serum saline, in both hands. Injections will be performed at the level of the distal palmar crease, targeting the neurovascular bundles in the 4 web spaces (0.25 ml/injection site). * between 2 to 3 hours before the procedure, application of lidocaine/prilocaine cream under an occlusive dressing (polyurethane film) in each palmar crease of the patients (1/2 tube/hand), according to the recommendations of the manufacturer of the lidocaine cream. * inhaled nitrous oxide/oxygen (50% / 50%) may be used, if needed |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Aged 18 years and older
- Diagnosed with systemic sclerosis (EULAR/ACR 2013 criteria ; or Leroy and Metsger criteria).
- Symptoms of Raynaud's phenomenon affecting both hands (not necessarily to equal extents)
- Frequency of Raynaud's attacks ≥ 5/week during cold weather
- Stable dose of phosphodiesterase inhibitors (sildenafil, tadalafil or vardenafil), endothelin antagonists, or calcium channel blockers defined as 1-month with no change in dose
Key exclusion criteria (9 listed)
- History of myasthenia gravis or Eaton Lambert syndrome
- Inflammatory myositis <2 years or pre-existing motor neurone disease or upper limb motor neuropathy
- Reported allergy or hypersensitivity to any Botulinum toxin preparation or to lidocaine or other local anesthetic agent or to albumin or to inhaled nitrous oxide/oxygen.
- Active infection in either hand.
- Pregnant or lactating women (women of child bearing potential must undergo a urine pregnancy teste before inclusion and at treatment day).
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 |
|---|---|---|
| BOTOX® solutionBotox solution + Saline serum + lidocaine/prilocaine cream + Nitrous oxide/oxygen (50%/50%) | Drug | " BTX-A" group |
| Placebo groupPlacebo + lidocaine/prilocaine cream + Nitrous oxide/oxygen (50%/50%) | Drug | Placebo group |
Primary outcome
Absolute change from baseline in the number of RP attacks per week at 4 weeks
Time frame: 4 weeks
Comparison between the 2 groups of the change (absolute) from baseline in the number of RP attacks per week at 4 weeks. An attack is defined as an episode of pallor or cyanosis (with or without pain, tingling or numbness). Subjects will keep a daily record of the number of RP attacks they experienced per day and the duration of each attack, from Day -14 to Day 0 (+/- 4 days) and from Day 14 to Day 28 ( +/- 4 days)
9 secondary outcomes
Absolute change from baseline in the number of RP attacks per week at 12 and 24 weeks
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the percentage of digital ulcers with complete healing
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the number of new digital ulcers.
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in Raynaud's pain score.
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in Raynaud's Condition Score.
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in quality of life from baseline to weeks 4, 12 and 24
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in hand function from baseline to weeks 4, 12 and 24
Time frame: 24 weeks
Comparison between the 2 groups at 4, 12 and 24 weeks of follow-up after the treatment of the change in hand function from baseline to weeks 4, 12 and 24
Time frame: 24 weeks
Frequency and severity of adverse drug events during treatment from baseline to weeks 4, 12 and 24.
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2022.
- Derived from this study: PubMed 36066501Senet P, Maillard H, Diot E, Lazareth I, Blaise S, Arnault JP, Pistorius MA, Boulon C, Cogrel O, Warzocha U, Riviere S, Malloizel-Delaunay J, Servettaz A, Sassolas B, Viguier M, Monfort JB, Janique S, Vicaut E; BRASS collaborators. Efficacy and Safety of Botulinum Toxin in Adults with Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study. Arthritis Rheumatol. 2023 Mar;75(3):459-467. doi: 10.1002/art.42342. Epub 2022 Dec 14.
- Individual participant dataSharing planned: Undecided
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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.
Patricia Senet, MD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BOTOX® solution in people with Raynaud Phenomenon Secondary to Systemic Sclerosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 9 inclusion and 9 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 10 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Absolute change from baseline in the number of RP attacks per week at 4 weeks (time frame: 4 weeks). 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 NCT03717961, which also lists 1 linked publication.
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 10 Feb 2022.