Spastic AtaxiaSPAX-PBPNCT04297891
Phenotypes, Biomarkers and Pathophysiology in Spastic Ataxias
An observational study of Spastic Ataxia, run by Dr. Rebecca Schule at 9 sites in 7 countries.
Trial summary
The aim of this study is to determine the clinical spectrum and natural progression of Spastic Ataxias (SPAX) and related disorders in a prospective multicenter natural history study, identify digital, imaging and molecular biomarkers that can assist in diagnosis and therapy development and study the genetic etiology… Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Spastic Ataxia
- What is being tested?
- Clinical rating scale to measure Ataxia disease severity and progression, Clinical rating scale to measure spastic paraplegia disease severity and progression and Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS)
- How many people
- 250 participants
- Where
- 9 sitesin 7 countries
- Study dates
- 1 Sep 2020to Jun 2025
- How long will I take part?
- 24 Months
- Phase
- Observational
- 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 Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Probands with clinically manifest and genetically confirmed spastic ataxia (genetic subtypes: ARSACS, SPG7) will be enrolled in this study. Additionally, healthy unrelated controls will be enrolled; these are necessary to contrast unspecific, gender- or age-related findings to disease specific findings.
Who may be able to join
- ARSACS cohort: genetic diagnosis of ARSACS and clinically manifest disease
- SPG7 cohort: genetic diagnosis of SPG7 and clinically manifest disease
- Unrelated healthy controls: no signs or history of neurological or psychiatric disease
- AND
- written informed consent provided
- AND
- Participants are willing and able to comply with study procedures
Who cannot join
- Missing informed consent
- For controls: evidence of a neurodegenerative disease or movement disorders; inability to give informed consent
Trial locations and contacts
9 sites in 7 countries.
9 sites in 7 countries
Canada2 sites, 2 recruiting
- Recruiting
Montreal Neurological Institute of McGill University, Department of Neurology and Neurosurgery and Human Genetics
Montreal, Quebec, H3A 2B4
Bernard Brais, MD, PhD, +1-514-398 ext. 3334, bernard.brais@mcgill.ca
Bernard Brais, MD, PhD
- Recruiting
Université de Sherbrooke
Saguenay, Quebec, G7X 7X2
Cynthia Gagnon, PhD, 1-418-695-7700, cynthia.gagnon4@usherbrooke.ca
Isabelle Côté, M.Sc., 1-418-695-7700, Isabelle.Cote7@USherbrooke.ca
Germany2 sites, 2 recruiting
- Recruiting
University Hospital Essen (AöR)
Essen, 45147
Stephan Klebe, Prof. Dr., +49-201-723-6513, Stephan.Klebe@uk-essen.de
Dagmar Timmann-braun, Prof. Dr., (+)49 (0)201 7232180, Dagmar.Timmann-braun@uk-essen.de
- Recruiting
Center for Neurology & Hertie-Institute for Clinical Brain Research, Dept. for Neurodegenerative Diseases
Tübingen, Baden-Wurttemberg, 72076
Selina Reich, M.Sc, +49 7071 29 ext. 85548, selina.reich@uni-tuebingen.de
Katrin Dillmann-Jehn, +49 7071 29 ext. 85653, katrin.dillmann@med.uni-tuebingen.de
France1 site, 1 recruiting
- Recruiting
Département d'information médicale (DIM); Département de Biostatistique, Santn 3emé Publique et Information Médicale (BIOSPIM)- Bâtiment Mazarie étage; Hôpitaux Universitaires Pitié Salpêtrière
Paris, 75013
Hortense Hurmic, (33)1 57 27 42 22, hortense.hurmic@icm-institute.org
Sophie Tezenas Du Montcel, MD, PhD, (33) 1 42 16 05 82, sophie.tezenas@aphp.fr
Italy1 site, 1 recruiting
- Recruiting
IRCCS Fondazione Stella Maris
Pisa, 56128
Filippo M. Santorelli, MD, +39 050 886275, filippo.santorelli@fsm.unipi.it
Sirio Cocozza, MD, +39 081 7462560, sirio.cocozza@unina.it
Netherlands1 site, 1 recruiting
- Recruiting
Radboud University Medical Center; Department of Neurology & Donders Institute for Brain, Cognition, and Behaviour
Nijmegen, 6525 GC
Bart van de Warrenburg, MD, PhD, 0031-24-3613396, Bart.vandeWarrenburg@radboudumc.nl
Bart van de Warrenburg, MD, PhD
Turkey (Türkiye)1 site, 1 recruiting
- Recruiting
Koç Univ. Hospital, KUTTAMNDAL
Istanbul, 34010
Nazli BAŞAK, PhD, +90-850-250825023811, nbasak@ku.edu.tr
Atay Vural, MD, PhD, +90 850 250 8250, atayvural@ku.edu.tr
United Kingdom1 site, 1 recruiting
- Recruiting
Department of Clinical Neurosciences, University of Cambridge; John Van Geest Cambridge Centre for Brain Repair
Cambridge, CB2 0PY
Rita Horvath, MD, PhD, +44 (0) 1223 331165, rh732@medschl.cam.ac.uk
Rita Horvath, MD, PhD
About this study
The aim of this study is to determine the clinical spectrum and natural progression of Spastic Ataxias (SPAX) and related disorders in a prospective multicenter natural history study, identify digital, imaging and molecular biomarkers that can assist in diagnosis and therapy development and study the genetic etiology and molecular mechanisms of these diseases.
Read the detailed description
The investigators will perform a registry-based standardized prospective Natural History Study (NHS) in SPAX and related disorders. Participants will be seen annually. At study visits a standardized clinical examination will be performed including application of clinical rating scales (selection of rating scales may vary depending on the individual phenotype and specific genotype); data will be entered into a clinical database (HSP Registry; https://www.hsp-registry.net and ARCA Registry; www.ARCA-registry.org). At all study visits, patients will be asked to donate biosamples; biomaterial collection is optional and participants can elect to participate in sampling of blood, urine, CSF, and/or a skin biopsy.
Optionally, additional examinations may be performed including imaging, quantitative movement analysis, neuropsychological examinations, analysis of patient or observer reported outcomes and OMICS analysis to characterize molecular biomarkers.
In participants without a genetic diagnosis, next generation sequencing may be performed.
Thus this study will establish a model of disease progression and mechanistic evolution in SPAX, which will allow to track and understand selective as well as overlapping dysfunction of the cerebellum and corticospinal tract. In a transatlantic natural history study we will longitudinally validate clinician- and patient-reported, digital and molecular outcomes. In addition, we will improve on existing and develop new outcome parameters that show superior sensitivity to change. These include a novel clinical SPAX composite score, a smartphone mHealth toolbox combining remote assessment of daily living by wearable sensors with app-based patient-entered outcomes (SPAX.app), and multimodal MRI radiomics with an innovative machine learning approach for multisite MRI analysis, including in particular the infratentorial space. Longitudinal validation of targeted fluid biomarker candidates will aslo be an important part.
- Conditions
- Spastic Ataxia
- Being tested
- Clinical rating scale to measure Ataxia disease severity and progression, Clinical rating scale to measure spastic paraplegia disease severity and progression, Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS), Next-Gen Sequencing (NGS)
- Keywords
- Spastic Ataxia, Biomarker, Genetic etiology, Molecular mechanisms, Natural History Study
- Medical subject headings
- Spastic Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinical rating scale to measure Ataxia disease severity and progressionOther
Also called Scale for the Assessment and rating of Ataxia (SARA)
SARA is a clinical scale developed by Schmitz-Hübsch et al which assesses a range of different impairments in cerebellar ataxia. The scale is made up of 8 items related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements and heel-shin test.
Clinical rating scale to measure spastic paraplegia disease severity and progressionOther
Also called Spastic Paraplegia Rating Scale (SPRS)
A 13-item scale to rate functional impairment occurring in pure forms of spastic paraplegia (SP). Additional symptoms constituting a complicated form of SP are recorded in an inventory.
Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS)Other
Also called DSI-ARSACS
The DSI-ARSACS is a valid measure of disease severity for the adult ARSACS population that is able to distinguish between patients with different clinical profiles.
It considers the 3 components (pyramidal, cerebellar, neuropathic) of the disease, and documents its content validity, internal consistency, and construct validity.
Next-Gen Sequencing (NGS)Diagnostic test
Whole Genome Sequencing, Whole Exome Sequencing, Transcriptomics, Proteomics, Metabolomics
| Study group | Role | What they receive |
|---|---|---|
| ARSACS | Participants with genetically confirmed ARSACS (ORPHA:98) will be recruited. Target sample size for the ARSACS cohort is 120. Receives: Other: Clinical rating scale to measure Ataxia disease severity and progression; Other: Clinical rating scale to measure spastic paraplegia disease severity and progression; Other: Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS); Diagnostic Test: Next-Gen Sequencing (NGS) | |
| SPG7 | Participants with genetically confirmed SPG7 (ORPHA:99013) will be recruited. Target sample size for the SPG7 cohort is 72. Receives: Other: Clinical rating scale to measure Ataxia disease severity and progression; Other: Clinical rating scale to measure spastic paraplegia disease severity and progression; Other: Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS); Diagnostic Test: Next-Gen Sequencing (NGS) | |
| Unrelated healthy control | Unrelated healthy controls Healthy controls may undergo the same study procedures as the ARSACS and SPG7 cohort. Target sample size for the control cohort is 50. Receives: Other: Clinical rating scale to measure Ataxia disease severity and progression; Other: Clinical rating scale to measure spastic paraplegia disease severity and progression; Other: Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS); Diagnostic Test: Next-Gen Sequencing (NGS) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of Scale for the Assessment and Rating of Ataxia (SARA) from baseline to 2-year follow-up
Severity of the ataxia component of the disease will be assessed by application of the Scale for the Assessment and Rating of Ataxia (SARA). The total score is calculated as the sum of al items, yielding a total score between 0 and 38. Hereby, higher SARA scores indicate more severe disease.
Measured: 24 months
Change of Spastic Paraplegia Rating Scale (SPRS) from baseline to 2-year follow-up
Severity of the spasticity component of the disease will be assessed by application of the Spastic Paraplegia Rating Scale. The total score is calculated as the sum of al items, yielding a total score between 0 and 52. Hereby, higher SPRS scores indicate more severe disease.
Measured: 24 months
Secondary outcomes (1)
Change of Disease severity index - Autosomal recessive spastic ataxia of Charlevoix-Saguenay(DSI-ARSACS) from baseline to 2-year follow-up
The DSI-ARSACS is a clinical rating scale consisting of 8 items reflecting the 3 main components of the disease (pyramidal, cerebellar, and neuropath systems) specifically developed to measure disease progression in ARSACs. The DSI-ARSACS total score can range from 0 to 38. Hereby, higher DSI-ARSACS scores indicate more severe disease.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2020
- Study start1 Sep 2020
- Primary completionJun 2024 (estimated)
- Study completionJun 2025 (estimated)
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 Spastic AtaxiaStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (estimated)
- Target duration
- 24 Months
Sponsor and investigators
- Lead sponsor
- Dr. Rebecca Schule
- Sponsor type
- University, hospital or other
- Collaborators
- German Center for Neurodegenerative Diseases (DZNE), German Research Foundation
- Responsible party
- Sponsor-investigator, Dr. Rebecca Schule, University Hospital Tuebingen
- Investigators
- Rebecca Schüle, PD Dr., Principal investigator, University Hospital TuebingenMatthis Synofzik, Prof., Dr., Principal investigator, University Hospital Tuebingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04297891
- Sponsor's protocol ID
- SPAX-PBP
- Other IDs
- 441409627
- Acronym
- SPAX-PBP
- Status last verified
- May 2022
- First submitted
- 27 Feb 2020
- Last updated
- 18 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Hamdan A, Traschutz A, Beichert L, Chen X, Gagnon C, van de Warrenburg BP, Santorelli FM, Basak N, Coarelli G, Horvath R, Klebe S; PROSPAX consortium; EVIDENCE-RND consortium; Schule R, Hooker AC, Synofzik M, Karlsson MO. Integrated Modeling of Digital-Motor and Clinician-Reported Outcomes Using Item Response Theory: Towards Powerful Trials for Rare Neurological Diseases. CPT Pharmacometrics Syst Pharmacol. 2025 Nov;14(11):1857-1868. doi: 10.1002/psp4.70081. Epub 2025 Jul 21.
PubMed 40685914
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phenotypes, Biomarkers and Pathophysiology in Spastic Ataxias
- ClinicalTrials.gov ID
- NCT04297891
- Acronym / sponsor ID
- SPAX-PBP / SPAX-PBP
- Conditions
- Spastic Ataxia
- Keywords
- Spastic Ataxia, Biomarker, Genetic etiology, Molecular mechanisms, Natural History Study
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Dr. Rebecca SchuleUniversity, hospital or other
- Collaborators
- German Center for Neurodegenerative Diseases (DZNE), German Research Foundation
- Study start
- 1 Sep 2020 (actual)
- Primary completion
- Jun 2024 (estimated)
- Study completion
- Jun 2025 (estimated)
- First posted
- 6 Mar 2020 (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.
The aim of this study is to determine the clinical spectrum and natural progression of Spastic Ataxias (SPAX) and related disorders in a prospective multicenter natural history study, identify digital, imaging and molecular biomarkers that can assist in diagnosis and therapy development and study the genetic etiology and molecular mechanisms of these diseases.
Detailed description
The investigators will perform a registry-based standardized prospective Natural History Study (NHS) in SPAX and related disorders. Participants will be seen annually. At study visits a standardized clinical examination will be performed including application of clinical rating scales (selection of rating scales may vary depending on the individual phenotype and specific genotype); data will be entered into a clinical database (HSP Registry; https://www.hsp-registry.net and ARCA Registry; www.ARCA-registry.org). At all study visits, patients will be asked to donate biosamples; biomaterial collection is optional and participants can elect to participate in sampling of blood, urine, CSF, and/or a skin biopsy.
Optionally, additional examinations may be performed including imaging, quantitative movement analysis, neuropsychological examinations, analysis of patient or observer reported outcomes and OMICS analysis to characterize molecular biomarkers.
In participants without a genetic diagnosis, next generation sequencing may be performed.
Thus this study will establish a model of disease progression and mechanistic evolution in SPAX, which will allow to track and understand selective as well as overlapping dysfunction of the cerebellum and corticospinal tract. In a transatlantic natural history study we will longitudinally validate clinician- and patient-reported, digital and molecular outcomes. In addition, we will improve on existing and develop new outcome parameters that show superior sensitivity to change. These include a novel clinical SPAX composite score, a smartphone mHealth toolbox combining remote assessment of daily living by wearable sensors with app-based patient-entered outcomes (SPAX.app), and multimodal MRI radiomics with an innovative machine learning approach for multisite MRI analysis, including in particular the infratentorial space. Longitudinal validation of targeted fluid biomarker candidates will aslo be an important part.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 24 Months
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ARSACS | – | Other: Clinical rating scale to measure Ataxia disease severity and progression, Other: Clinical rating scale to measure spastic paraplegia disease severity and progression, Other: Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS), Diagnostic Test: Next-Gen Sequencing (NGS)Participants with genetically confirmed ARSACS (ORPHA:98) will be recruited. Target sample size for the ARSACS cohort is 120. |
| SPG7 | – | Other: Clinical rating scale to measure Ataxia disease severity and progression, Other: Clinical rating scale to measure spastic paraplegia disease severity and progression, Other: Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS), Diagnostic Test: Next-Gen Sequencing (NGS)Participants with genetically confirmed SPG7 (ORPHA:99013) will be recruited. Target sample size for the SPG7 cohort is 72. |
| Unrelated healthy control | – | Other: Clinical rating scale to measure Ataxia disease severity and progression, Other: Clinical rating scale to measure spastic paraplegia disease severity and progression, Other: Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS), Diagnostic Test: Next-Gen Sequencing (NGS)Unrelated healthy controls Healthy controls may undergo the same study procedures as the ARSACS and SPG7 cohort. Target sample size for the control cohort is 50. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Probands with clinically manifest and genetically confirmed spastic ataxia (genetic subtypes: ARSACS, SPG7) will be enrolled in this study. Additionally, healthy unrelated controls will be enrolled; these are necessary to contrast unspecific, gender- or age-related findings to disease specific findings.
Key inclusion criteria (7 listed)
- ARSACS cohort: genetic diagnosis of ARSACS and clinically manifest disease
- SPG7 cohort: genetic diagnosis of SPG7 and clinically manifest disease
- Unrelated healthy controls: no signs or history of neurological or psychiatric disease
- AND
- written informed consent provided
Key exclusion criteria (2 listed)
- Missing informed consent
- For controls: evidence of a neurodegenerative disease or movement disorders; inability to give informed consent
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 |
|---|---|---|
| Clinical rating scale to measure Ataxia disease severity and progressionAlso: Scale for the Assessment and rating of Ataxia (SARA)SARA is a clinical scale developed by Schmitz-Hübsch et al which assesses a range of different impairments in cerebellar ataxia. The scale is made up of 8 items related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements and heel-shin test. | Other | ARSACS, SPG7, Unrelated healthy control |
| Clinical rating scale to measure spastic paraplegia disease severity and progressionAlso: Spastic Paraplegia Rating Scale (SPRS)A 13-item scale to rate functional impairment occurring in pure forms of spastic paraplegia (SP). Additional symptoms constituting a complicated form of SP are recorded in an inventory. | Other | ARSACS, SPG7, Unrelated healthy control |
| Disease-specific severity index for adults with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS)Also: DSI-ARSACSThe DSI-ARSACS is a valid measure of disease severity for the adult ARSACS population that is able to distinguish between patients with different clinical profiles. It considers the 3 components (pyramidal, cerebellar, neuropathic) of the disease, and documents its content validity, internal consistency, and construct validity. | Other | ARSACS, SPG7, Unrelated healthy control |
| Next-Gen Sequencing (NGS)Whole Genome Sequencing, Whole Exome Sequencing, Transcriptomics, Proteomics, Metabolomics | Diagnostic test | ARSACS, SPG7, Unrelated healthy control |
Primary outcome
Change of Scale for the Assessment and Rating of Ataxia (SARA) from baseline to 2-year follow-up
Time frame: 24 months
Severity of the ataxia component of the disease will be assessed by application of the Scale for the Assessment and Rating of Ataxia (SARA). The total score is calculated as the sum of al items, yielding a total score between 0 and 38. Hereby, higher SARA scores indicate more severe disease.
Change of Spastic Paraplegia Rating Scale (SPRS) from baseline to 2-year follow-up
Time frame: 24 months
Severity of the spasticity component of the disease will be assessed by application of the Spastic Paraplegia Rating Scale. The total score is calculated as the sum of al items, yielding a total score between 0 and 52. Hereby, higher SPRS scores indicate more severe disease.
1 secondary outcome
Change of Disease severity index - Autosomal recessive spastic ataxia of Charlevoix-Saguenay(DSI-ARSACS) from baseline to 2-year follow-up
Time frame: 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2022.
- Derived from this study: PubMed 40685914Hamdan A, Traschutz A, Beichert L, Chen X, Gagnon C, van de Warrenburg BP, Santorelli FM, Basak N, Coarelli G, Horvath R, Klebe S; PROSPAX consortium; EVIDENCE-RND consortium; Schule R, Hooker AC, Synofzik M, Karlsson MO. Integrated Modeling of Digital-Motor and Clinician-Reported Outcomes Using Item Response Theory: Towards Powerful Trials for Rare Neurological Diseases. CPT Pharmacometrics Syst Pharmacol. 2025 Nov;14(11):1857-1868. doi: 10.1002/psp4.70081. Epub 2025 Jul 21.
- Website of the HSP RegistryLink provided by the sponsor.
- Website of the ARCA RegistryLink provided by the sponsor.
- 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.
Rebecca Schüle, PD Dr. principal investigator
University Hospital Tuebingen
Matthis Synofzik, Prof., Dr. principal investigator
University Hospital Tuebingen
9 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spastic Ataxia, run by Dr. Rebecca Schule at 9 sites in 7 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 7 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: status not confirmed, as of the sponsor's last update on 18 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Canada, Germany, France, Italy, Netherlands and 2 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rebecca Schüle, PD Dr. (+49 7071 29 ext. 85653, rebecca.schuele-freyer@uni-tuebingen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of Scale for the Assessment and Rating of Ataxia (SARA) from baseline to 2-year follow-up (time frame: 24 months). The study lists 2 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 NCT04297891, 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 18 May 2022.