Chronic Troublesome SialorrheaPhase 3SIPEXINCT02270736
Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability
A Phase 3 study testing NT 201 in people with Chronic Troublesome Sialorrhea and Cerebral Palsy, run by Merz Pharmaceuticals GmbH at 31 sites in 6 countries.
Trial summary
The objective of this study is to investigate the efficacy and safety of NT 201 compared with placebo for the treatment of chronic troublesome sialorrhea associated with neurological disorders (e.g. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Chronic Troublesome Sialorrhea, Cerebral Palsy, Stroke
- What is being tested?
- NT 201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 256 participants
- Where
- 31 sitesin 6 countries
- Study dates
- 9 Feb 2015to 7 May 2019
- 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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female child/adolescent age 2-17 years.
- Any neurological disorder (e.g. cerebral palsy or traumatic brain injury) and/or intellectual disability associated with chronic troublesome sialorrhea for at least 3 months up to the screening. In subjects with intellectual disability (ID) without neurological disorders, a diagnosis of ID by a specialist, e.g. pediatrician or by a center for developmental medicine is required for inclusion.
- Severe drooling (modified Teacher´s Drooling Scale [mTDS] ≥ 6; clothing occasionally becomes damp) as rated by the investigator.
- Parental consent and the subject's oral or written assent as the subject is able to provide.
Who cannot join
- Chronic troublesome sialorrhea not related to neurological disorders and/or intellectual disability.
- Body weight < 12 kg.
- Pharmacological treatment for sialorrhea or concomitant medication known to influence sialorrhea strongly (e.g. anticholinergics with exception of locally applied or short acting drugs used under general anesthesia) within 45 days before baseline and during the entire study period.
- Any previous known or suspected hypersensitivity to Botulinum toxin.
- Aspiration pneumonia within 6 month before screening.
- Any previous treatment with Botulinum toxin for any body region during the year before screening or within the screening period
Show all 10Show fewer
- Prior, concomitant or planned surgery or irradiation to head and neck to control sialorrhea (including salivary gland surgery or salivary gland irradiation) within one year before screening or planned for any part of the entire study period.
- Concurrent diseases, including hematological, hepatic, renal, gastrointestinal, endocrine, pulmonary, musculoskeletal, or psychiatric diseases or conditions, which in the judgment of the investigator would put the subject at risk while in the study, could influence the results of the study, or negatively impact the subject's ability to participate in the study.
- Extremely poor dental and/or oral condition that might preclude safe study participation by the judgment of the investigator.
- Nursing mother or pregnant female subject.
Trial locations and contacts
31 sites in 6 countries.
31 sites in 6 countries
Russia8 sites
Merz Investigational Site #0070016
Kazan', 420012
Merz Investigational Site # 0070288
Kemerovo, 650066
Merz Investigational Site #0070290
Khabarovsk, 680038
Merz Investigational # 0070017
Saint Petersburg, 194100
Merz Investigational Site #0070013
Smolensk, 214018
Merz Investigational Site # 070019
Stavropol, 355029
Merz Investigational Site #0070300
Tomsk, 634052
Merz Investigational Site #0070301
Yekaterinburg, 620149
Ukraine8 sites
Merz Investigational Site #3800001
Dnipropetrovsk, 49027
Merz Investigational Site #3800012
Ivano-Frankivsk, 76014
Merz Investigational Site #3800005
Kharkiv, 61068
Merz Investigational Site #3800007
Kharkiv, 61153
Merz Investigational Site #3800013
Kherson, 73010
Merz Investigational site #3800003
Odesa, 65012
Merz Investigational Site #3800009
Ternopil, 46020
Merz Investigational Site #3800011
Zaporizhzhya, 69063
Hungary6 sites
Merz Investigational Site #0360017
Balassagyarmat, 2660
Merz Investigational Site #0360013
Budapest, 1083
Merz Investigational Site # 0360014
Budapest, 1125
Merz Investigational Site # 0360015
Budapest, 1125
Merz Investigational Site #0360018
Budapest, 1146
Merz Investigational Site #0360016
Szombathely, 9700
Poland5 sites
Merz Investigational Site #0480092
Bialystok, 15-274
Merz Investigational Site #0480090
Gdansk, 80-952
Merz Investigational Site #0480076
Katowice, 40-954
Merz Investigational Site #0480059
Krakow, 30-359
Merz Investigational Site #0480060
Wiązowna, 05-462
Georgia3 sites
Merz Investigational Site #9950003
K'obulet'i, 6200
Merz Investigational Site #9950001
Tbilisi, 0159
Merz Investigational Site #9950002
Tbilisi, 0159
About this study
Official title: Prospective, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study With an Open-label Extension Period to Investigate the Efficacy and Safety of NT 201 in the Treatment of Children and Adolescents (2-17 Years) With Chronic Troublesome Sialorrhea Associated With Neurological Disorders, and/or Intellectual Disability
The objective of this study is to investigate the efficacy and safety of NT 201 compared with placebo for the treatment of chronic troublesome sialorrhea associated with neurological disorders (e.g. cerebral palsy, traumatic brain injury) and/or intellectual disability in children and adolescents naïve to Botulinum neurotoxin treatment and aged 2-17 years.
- Conditions
- Chronic Troublesome Sialorrhea, Cerebral Palsy, Stroke, Traumatic Brain Injury, Intellectual Disability
- Being tested
- NT 201 Placebo, NT 201
- Medical subject headings
- Cerebral Palsy, Stroke, Brain Injuries, Traumatic, Intellectual Disability
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NT 201 PlaceboDrug
NT 201 placebo matching injection.
NT 201Drug
Also called IncobotulinumtoxinA, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
NT 201 injection.
| Study group | Role | What they receive |
|---|---|---|
| Double-blind MP: Placebo (Age 6 to 17 Years) | Experimental | Participants will receive placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes will be matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm. Receives: Drug: NT 201 Placebo |
| Double-blind, MP: NT 201 (Age 6 to 17 Years) | Experimental | Participants will receive NT 201 (up to 2.5 Units per kilogram [U/kg] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Receives: Drug: NT 201 |
| Open-label, MP: NT 201 (Age 2 to 5 Years) | Experimental | Participants will receive NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Receives: Drug: NT 201 |
| OLEX: NT 201 (Age 6 to 17 Years) | Experimental | Participants will receive NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm will consist of participants who will participate in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)". Receives: Drug: NT 201 |
| OLEX: NT 201 (Age 2 to 5 Years) | Experimental | Participants will receive NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). Receives: Drug: NT 201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4
This endpoint was planned to be analyzed in double-blind, MP, 6 to 17 years participants only. uSFR was assessed by weighing of absorbent swabs with safety threads soaked with saliva over 5 minutes and the procedure was repeated after 30 minutes. Salivary flow rate was equal to weight increase of swabs/time of collection. The average of the 2 results for flow rate was calculated. The reduction of measured weight over the study relates to improvement of sialorrhea.
Measured: Baseline and Week 4
Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. The GICS was used to measure the carer's/parent's impression of change due to treatment. The response option was a common 7-point Likert scale, with the following values: +3 (very much improved); +2 (much improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (much worse); -3 (very much worse).
Measured: Week 4
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
Measured: Baseline up to Week 64
Secondary outcomes (6)
Change From Baseline in uSFR at Weeks 8 and 12
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. uSFR was assessed by weighing of absorbent swabs with safety threads soaked with saliva over 5 minutes and then procedure was repeated after 30 minutes. Salivary flow rate was equal to weight increase of swabs/time of collection. The average of the 2 results for flow rate was calculated. The reduction of measured weight over the study relates to improvement of sialorrhea.
Measured: Baseline and Weeks 8 and 12
GICS at Weeks 8 and 12
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. The GICS was used to measure the carer's/parent's impression of change due to treatment. The response option was a common 7-point Likert scale with the following values: +3 (very much improved); +2 (much improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (much worse); -3 (very much worse).
Measured: Weeks 8 and 12
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle
Measured: Baseline up to Week 64
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle
Measured: Baseline up to Week 64
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle
Measured: Baseline up to Week 64
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle
Measured: Baseline up to Week 64
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2014 (estimated)
- Study start9 Feb 2015
- Primary completion23 Feb 2018
- Study completion7 May 2019
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 (2)Papers the sponsor has linked to this study.
- Other trials for Chronic Troublesome SialorrheaStudies 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 256 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merz Pharmaceuticals GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Merz Medical Expert, Study director, Merz Pharmaceuticals GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02270736
- Sponsor's protocol ID
- MRZ60201_3091_1
- Other IDs
- 2013-004532-30
- Acronym
- SIPEXI
- Status last verified
- Jul 2019
- First submitted
- 17 Oct 2014
- Last updated
- 10 Aug 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Berweck S, Bonikowski M, Kim H, Althaus M, Flatau-Baque B, Mueller D, Banach MD. Placebo-Controlled Clinical Trial of IncobotulinumtoxinA for Sialorrhea in Children: SIPEXI. Neurology. 2021 Oct 4;97(14):e1425-e1436. doi: 10.1212/WNL.0000000000012573.
PubMed 34341153Berweck S, Banach M, Gaebler-Spira D, Chambers HG, Schroeder AS, Geister TL, Althaus M, Hanschmann A, Vacchelli M, Bonfert MV, Heinen F, Dabrowski E. Safety Profile and Lack of Immunogenicity of IncobotulinumtoxinA in Pediatric Spasticity and Sialorrhea: A Pooled Analysis. Toxins (Basel). 2022 Aug 25;14(9):585. doi: 10.3390/toxins14090585.
PubMed 36136523
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study With an Open-label Extension Period to Investigate the Efficacy and Safety of NT 201 in the Treatment of Children and Adolescents (2-17 Years) With Chronic Troublesome Sialorrhea Associated With Neurological Disorders, and/or Intellectual Disability
- ClinicalTrials.gov ID
- NCT02270736
- Acronym / sponsor ID
- SIPEXI / MRZ60201_3091_1
- Conditions
- Chronic Troublesome Sialorrhea, Cerebral Palsy, Stroke, Traumatic Brain Injury, Intellectual Disability
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 256 participants (actual)
- Lead sponsor
- Merz Pharmaceuticals GmbHIndustry (pharma, biotech, devices)
- Study start
- 9 Feb 2015 (actual)
- Primary completion
- 23 Feb 2018 (actual)
- Study completion
- 7 May 2019 (actual)
- First posted
- 21 Oct 2014 (estimated)
- Results
- Posted 20 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to investigate the efficacy and safety of NT 201 compared with placebo for the treatment of chronic troublesome sialorrhea associated with neurological disorders (e.g. cerebral palsy, traumatic brain injury) and/or intellectual disability in children and adolescents naïve to Botulinum neurotoxin treatment and aged 2-17 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Double-blind MP: Placebo (Age 6 to 17 Years) | Experimental | Drug: NT 201 PlaceboParticipants will receive placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes will be matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm. |
| Double-blind, MP: NT 201 (Age 6 to 17 Years) | Experimental | Drug: NT 201Participants will receive NT 201 (up to 2.5 Units per kilogram [U/kg] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. |
| Open-label, MP: NT 201 (Age 2 to 5 Years) | Experimental | Drug: NT 201Participants will receive NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. |
| OLEX: NT 201 (Age 6 to 17 Years) | Experimental | Drug: NT 201Participants will receive NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm will consist of participants who will participate in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)". |
| OLEX: NT 201 (Age 2 to 5 Years) | Experimental | Drug: NT 201Participants will receive NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Male or female child/adolescent age 2-17 years.
- Any neurological disorder (e.g. cerebral palsy or traumatic brain injury) and/or intellectual disability associated with chronic troublesome sialorrhea for at least 3 months up to the screening. In subjects with intellectual disability (ID) without neurological disorders, a diagnosis of ID by a specialist, e.g. pediatrician or by a center for developmental medicine is required for inclusion.
- Severe drooling (modified Teacher´s Drooling Scale [mTDS] ≥ 6; clothing occasionally becomes damp) as rated by the investigator.
- Parental consent and the subject's oral or written assent as the subject is able to provide.
Key exclusion criteria (10 listed)
- Chronic troublesome sialorrhea not related to neurological disorders and/or intellectual disability.
- Body weight < 12 kg.
- Pharmacological treatment for sialorrhea or concomitant medication known to influence sialorrhea strongly (e.g. anticholinergics with exception of locally applied or short acting drugs used under general anesthesia) within 45 days before baseline and during the entire study period.
- Any previous known or suspected hypersensitivity to Botulinum toxin.
- Aspiration pneumonia within 6 month before screening.
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 |
|---|---|---|
| NT 201 PlaceboNT 201 placebo matching injection. | Drug | Double-blind MP: Placebo (Age 6 to 17 Years) |
| NT 201Also: IncobotulinumtoxinA, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsNT 201 injection. | Drug | Double-blind, MP: NT 201 (Age 6 to 17 Years), OLEX: NT 201 (Age 2 to 5 Years), OLEX: NT 201 (Age 6 to 17 Years), Open-label, MP: NT 201 (Age 2 to 5 Years) |
Primary outcome
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4
Time frame: Baseline and Week 4
This endpoint was planned to be analyzed in double-blind, MP, 6 to 17 years participants only. uSFR was assessed by weighing of absorbent swabs with safety threads soaked with saliva over 5 minutes and the procedure was repeated after 30 minutes. Salivary flow rate was equal to weight increase of swabs/time of collection. The average of the 2 results for flow rate was calculated. The reduction of measured weight over the study relates to improvement of sialorrhea.
Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)
Time frame: Week 4
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. The GICS was used to measure the carer's/parent's impression of change due to treatment. The response option was a common 7-point Likert scale, with the following values: +3 (very much improved); +2 (much improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (much worse); -3 (very much worse).
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
Time frame: Baseline up to Week 64
6 secondary outcomes
Change From Baseline in uSFR at Weeks 8 and 12
Time frame: Baseline and Weeks 8 and 12
GICS at Weeks 8 and 12
Time frame: Weeks 8 and 12
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 34341153Berweck S, Bonikowski M, Kim H, Althaus M, Flatau-Baque B, Mueller D, Banach MD. Placebo-Controlled Clinical Trial of IncobotulinumtoxinA for Sialorrhea in Children: SIPEXI. Neurology. 2021 Oct 4;97(14):e1425-e1436. doi: 10.1212/WNL.0000000000012573.
- Derived from this study: PubMed 36136523Berweck S, Banach M, Gaebler-Spira D, Chambers HG, Schroeder AS, Geister TL, Althaus M, Hanschmann A, Vacchelli M, Bonfert MV, Heinen F, Dabrowski E. Safety Profile and Lack of Immunogenicity of IncobotulinumtoxinA in Pediatric Spasticity and Sialorrhea: A Pooled Analysis. Toxins (Basel). 2022 Aug 25;14(9):585. doi: 10.3390/toxins14090585.
- Individual participant dataSharing planned: No
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.
Merz Medical Expert study director
Merz Pharmaceuticals GmbH
31 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NT 201 in people with Chronic Troublesome Sialorrhea and Cerebral Palsy, run by Merz Pharmaceuticals GmbH at 31 sites in 6 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 4 inclusion and 10 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 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in Russia, Ukraine, Hungary, Poland, Georgia and 1 more country. 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 Jan 2021). 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?
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4 (time frame: Baseline and Week 4). The study lists 3 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 NCT02270736, which also lists 2 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 10 Aug 2021.