SBMAKDANCT07443449
International SBMA Project (KDA)
An observational study of SBMA and Spinal and Bulbar Muscular Atrophy (SBMA), run by Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta at 1 site in Italy.
Trial summary
This project aims at: connecting the existing registries and databases across different countries by comparing the information collected, the administered scales, the biochemical investigations performed; performing a common analysis of fully anonymized data collected in the existing registries and databases by… Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- SBMA, Spinal and Bulbar Muscular Atrophy (SBMA)
- How many people
- 700 participants
- Where
- 1 siteItaly
- Study dates
- 29 Jul 2024to Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: 700 SBMA affected individuals
Who may be able to join
- Individuals affected by SBMA for whom clinical data have been historically collected by the referral centres
Who cannot join
- na
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Connecting SBMA National Registries/Databases: a Retrospective Study
This project aims at: connecting the existing registries and databases across different countries by comparing the information collected, the administered scales, the biochemical investigations performed; performing a common analysis of fully anonymized data collected in the existing registries and databases by researchers adhering to the study to obtain cross-sectional and longitudinal data. Centres from the following countries have accepted the invitation to join Italy in this project: UK, US, Japan, France, Germany, Korea, Finland, Greece, Canada, Turkey.
Read the detailed description
Several groups have collected important data over the years on different national series of SBMA affected subjects in national databases and registries. Such data on hundreds of patients represent an invaluable collection of information that can be pooled together to gain broad knowledge of the disease. Both baseline and follow up data have been collected, thus potentially allowing either a cross-sectional analysis and a longitudinal study on disease progression. Therefore, we propose a retrospective study collating together data from the different populations into a very large international series. Such study would allow a better understanding of the clinical and laboratory characteristics of the disease, a comparison of the disease across different countries, and an analysis of the evolution of the disease according to the different outcome measures along the years. In the preliminary study preparation, we have collected the adhesion of centres from UK, US, Japan, France, Korea, Germany, Finland, Greece, Canada and Turkey. We have also compared the information that every centre collects. We have subsequently prepared a list of items (clinical history, milestones, symptoms and signs, laboratory data) to be filled by each centre with the help of the coordinating centre in Milan and sent back to Milan for centralized analysis. Anonymized data will be shared by the participating centres with the Milan coordinating centre. In the data analyses, we will focus on SBMA clinical and laboratory characteristics, disease course over time, responsiveness of outcome measures, differences and similarities between populations. Based on the updated adhesions to the current project, we will be able to collect and analyze data on about 700 SBMA patients overall, in a both cross-sectional and longitudinal retrospective study. Such project, with the largest SBMA cohort ever analyzed, will result in an increased knowledge on the characteristics and phenotype of the disease. It will improve clinical trial readiness and will be useful for future clinical trials.
Collaborators for this project are the following: Azienda Ospedale-Università di Padova (Dr Sorarù); Centro clinico NEMO Adulti, Roma (Dr Amelia Conte/Dr Mario Sabatelli); MRC Centre for Neuromuscular Disease UCL Institute of Neurology, London (Dr Pietro Fratta); Department of Neurology, Kyungpook National University Chilgok Hospital Daegu, Republic of Korea (Dr Jin-Sung Park); Hôpital Pitié-Salpêtrière, Paris, France (Dr Pierre-François Pradat, Giorgia Querin); Inherited Neuromuscolar Diseases Unit, Bethesda MD, USA (Christopher Grunseich, Dr Kenneth Fischbeck); Nagoya University Graduate School of Medicine, Department of Neurology, Nagoya Japan (Dr. Masahisa Katsuno); Department of Neurodegenerative Diseases and Gerontopsychiatry, University of Bonn, Germany (Dr Patrick Weydt); Neuromuscular Research Center, Tampere University, Finland (Dr Manu Jokela and Johanna Palmio); Neurogenetics Unit, Department of Neurology, National and Kapodistrian University of Athens, Greece (Dr Georgios Koutsis); Hotchkiss Brain Institute, Department of Clinical Neurosciences, University of Calgary, Alberta, Canada (Dr Gerald Pfeffer); Department of Neurology, Istanbul Faculty of Medicine, Turkey (Dr Yesim Parman and Arman Cakar).
- Conditions
- SBMA, Spinal and Bulbar Muscular Atrophy (SBMA)
- Keywords
- SBMA, Kennedy Disease, International Registries, Retrospective study, Study Design
- Medical subject headings
- Bulbo-Spinal Atrophy, X-Linked
What is being measured
The primary outcome is the main question the study is designed to answer.
Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS)
Change in total score of the Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS), a disease-specific functional scale assessing bulbar, spinal, trunk, and respiratory function. The SBMAFRS total score ranges from 0 to 56 points. Higher scores indicate better functional status.
Measured: 3 years
Six-Minute Walk Test (6MWT) Distance
Change in distance walked during the Six-Minute Walk Test (6MWT). The 6MWT measures the total distance (in meters) that a participant is able to walk on a flat surface in six minutes. Greater distance indicates better ambulatory and endurance capacity.
Measured: 3 years
Age at Onset of First SBMA Symptoms
Age (in years) at onset of first reported SBMA-related symptom (hand tremor/cramps/muscular weakness, dysarthria, dysphagia, handrail requirement, use of support/wheelchair, development of pneumonia)
Measured: assessed once
Secondary outcomes (3)
Creatine Phosphokinase (CPK) Serum Levels
Change in serum creatine phosphokinase (CPK) levels
Measured: 3 years
Sensory Signs and Symptoms
Presence and severity of sensory signs and symptoms assessed by clinical neurological examination (reduced vibration sense, paresthesia). Recorded as present/absent and, when applicable, graded according to standardized neurological assessment.
Measured: 3 years
Presence of Gynecomastia
Presence or absence of gynecomastia as assessed by clinical examination
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2026
- Study start29 Jul 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (27)Papers the sponsor has linked to this study.
- Other trials for SBMAStudies 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
- Other
- Time perspective
- Retrospective
- Enrolment
- 700 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Silvia Fenu, MD, Principal investigator, Fondazione IRCCS Istituto Neurologico Carlo Besta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07443449
- Sponsor's protocol ID
- Linking National SBMA Database
- Acronym
- KDA
- Status last verified
- Feb 2026
- First submitted
- 6 Feb 2026
- Last updated
- 2 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Querin G, DaRe E, Martinelli I, Bello L, Bertolin C, Pareyson D, Mariotti C, Pegoraro E, Soraru G. Validation of the Italian version of the SBMA Functional Rating Scale as outcome measure. Neurol Sci. 2016 Nov;37(11):1815-1821. doi: 10.1007/s10072-016-2666-y. Epub 2016 Jul 21.
PubMed 27444956Steinmetz K, Rudic B, Borggrefe M, Muller K, Siebert R, Rottbauer W, Ludolph A, Buckert D, Rosenbohm A. J wave syndromes in patients with spinal and bulbar muscular atrophy. J Neurol. 2022 Jul;269(7):3690-3699. doi: 10.1007/s00415-022-10992-5. Epub 2022 Feb 7.
PubMed 35132468Araki A, Katsuno M, Suzuki K, Banno H, Suga N, Hashizume A, Mano T, Hijikata Y, Nakatsuji H, Watanabe H, Yamamoto M, Makiyama T, Ohno S, Fukuyama M, Morimoto S, Horie M, Sobue G. Brugada syndrome in spinal and bulbar muscular atrophy. Neurology. 2014 May 20;82(20):1813-21. doi: 10.1212/WNL.0000000000000434. Epub 2014 Apr 23.
PubMed 24759840Koutsis G, Kladi A, Breza M, Karadima G, Panas M. Spinobulbar muscular atrophy (Kennedy's disease): A rare diagnosis in the Greek population. J Neurol Sci. 2015 Dec 15;359(1-2):450-1. doi: 10.1016/j.jns.2015.10.021. Epub 2015 Oct 21. No abstract available.
PubMed 26481168Cho HJ, Shin JH, Park YE, Sohn E, Nam TS, Kang MG, Park JM, Park D, Park JS. Characteristics of spinal and bulbar muscular atrophy in South Korea: a cross-sectional study of 157 patients. Brain. 2023 Mar 1;146(3):1083-1092. doi: 10.1093/brain/awac198.
PubMed 35639850Atsuta N, Watanabe H, Ito M, Banno H, Suzuki K, Katsuno M, Tanaka F, Tamakoshi A, Sobue G. Natural history of spinal and bulbar muscular atrophy (SBMA): a study of 223 Japanese patients. Brain. 2006 Jun;129(Pt 6):1446-55. doi: 10.1093/brain/awl096. Epub 2006 Apr 18.
PubMed 16621916Fratta P, Nirmalananthan N, Masset L, Skorupinska I, Collins T, Cortese A, Pemble S, Malaspina A, Fisher EM, Greensmith L, Hanna MG. Correlation of clinical and molecular features in spinal bulbar muscular atrophy. Neurology. 2014 Jun 10;82(23):2077-84. doi: 10.1212/WNL.0000000000000507. Epub 2014 May 9.
PubMed 24814851Rosen RC, Cappelleri JC, Smith MD, Lipsky J, Pena BM. Development and evaluation of an abridged, 5-item version of the International Index of Erectile Function (IIEF-5) as a diagnostic tool for erectile dysfunction. Int J Impot Res. 1999 Dec;11(6):319-26. doi: 10.1038/sj.ijir.3900472.
PubMed 10637462Plante M, Corcos J, Gregoire I, Belanger MF, Brock G, Rossingol M. The international prostate symptom score: physician versus self-administration in the quantification of symptomatology. Urology. 1996 Mar;47(3):326-8. doi: 10.1016/s0090-4295(99)80447-6.
PubMed 8633396Harris-Love MO, Fernandez-Rhodes L, Joe G, Shrader JA, Kokkinis A, La Pean Kirschner A, Auh S, Chen C, Li L, Levy E, Davenport TE, Di Prospero NA, Fischbeck KH. Assessing function and endurance in adults with spinal and bulbar muscular atrophy: validity of the adult myopathy assessment tool. Rehabil Res Pract. 2014;2014:873872. doi: 10.1155/2014/873872. Epub 2014 May 5.
PubMed 24876969Hashizume A, Katsuno M, Suzuki K, Banno H, Suga N, Mano T, Araki A, Hijikata Y, Grunseich C, Kokkinis A, Hirakawa A, Watanabe H, Yamamoto M, Fischbeck KH, Sobue G. A functional scale for spinal and bulbar muscular atrophy: Cross-sectional and longitudinal study. Neuromuscul Disord. 2015 Jul;25(7):554-62. doi: 10.1016/j.nmd.2015.03.008. Epub 2015 Mar 20.
PubMed 25913211Takeuchi Y, Katsuno M, Banno H, Suzuki K, Kawashima M, Atsuta N, Ito M, Watanabe H, Tanaka F, Sobue G. Walking capacity evaluated by the 6-minute walk test in spinal and bulbar muscular atrophy. Muscle Nerve. 2008 Aug;38(2):964-71. doi: 10.1002/mus.21077.
PubMed 18642379Pennuto M, Greensmith L, Pradat PF, Soraru G; European SBMA Consortium. 210th ENMC International Workshop: Research and clinical management of patients with spinal and bulbar muscular atrophy, 27-29 March, 2015, Naarden, The Netherlands. Neuromuscul Disord. 2015 Oct;25(10):802-12. doi: 10.1016/j.nmd.2015.06.462. Epub 2015 Jun 19. No abstract available.
PubMed 26206601Ambrosini A, Calabrese D, Avato FM, Catania F, Cavaletti G, Pera MC, Toscano A, Vita G, Monaco L, Pareyson D. The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage. Orphanet J Rare Dis. 2018 Oct 4;13(1):176. doi: 10.1186/s13023-018-0918-z.
PubMed 30286784Querin G, Bertolin C, Da Re E, Volpe M, Zara G, Pegoraro E, Caretta N, Foresta C, Silvano M, Corrado D, Iafrate M, Angelini L, Sartori L, Pennuto M, Gaiani A, Bello L, Semplicini C, Pareyson D, Silani V, Ermani M, Ferlin A, Soraru G; Italian Study Group on Kennedy's disease. Non-neural phenotype of spinal and bulbar muscular atrophy: results from a large cohort of Italian patients. J Neurol Neurosurg Psychiatry. 2016 Aug;87(8):810-6. doi: 10.1136/jnnp-2015-311305. Epub 2015 Oct 26.
PubMed 26503015
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Connecting SBMA National Registries/Databases: a Retrospective Study
- ClinicalTrials.gov ID
- NCT07443449
- Acronym / sponsor ID
- KDA / Linking National SBMA Database
- Conditions
- SBMA, Spinal and Bulbar Muscular Atrophy (SBMA)
- Keywords
- SBMA, Kennedy Disease, International Registries, Retrospective study, Study Design
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 700 participants (actual)
- Lead sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo BestaUniversity, hospital or other
- Study start
- 29 Jul 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 2 Mar 2026 (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 project aims at: connecting the existing registries and databases across different countries by comparing the information collected, the administered scales, the biochemical investigations performed; performing a common analysis of fully anonymized data collected in the existing registries and databases by researchers adhering to the study to obtain cross-sectional and longitudinal data. Centres from the following countries have accepted the invitation to join Italy in this project: UK, US, Japan, France, Germany, Korea, Finland, Greece, Canada, Turkey.
Detailed description
Several groups have collected important data over the years on different national series of SBMA affected subjects in national databases and registries. Such data on hundreds of patients represent an invaluable collection of information that can be pooled together to gain broad knowledge of the disease. Both baseline and follow up data have been collected, thus potentially allowing either a cross-sectional analysis and a longitudinal study on disease progression. Therefore, we propose a retrospective study collating together data from the different populations into a very large international series. Such study would allow a better understanding of the clinical and laboratory characteristics of the disease, a comparison of the disease across different countries, and an analysis of the evolution of the disease according to the different outcome measures along the years. In the preliminary study preparation, we have collected the adhesion of centres from UK, US, Japan, France, Korea, Germany, Finland, Greece, Canada and Turkey. We have also compared the information that every centre collects. We have subsequently prepared a list of items (clinical history, milestones, symptoms and signs, laboratory data) to be filled by each centre with the help of the coordinating centre in Milan and sent back to Milan for centralized analysis. Anonymized data will be shared by the participating centres with the Milan coordinating centre. In the data analyses, we will focus on SBMA clinical and laboratory characteristics, disease course over time, responsiveness of outcome measures, differences and similarities between populations. Based on the updated adhesions to the current project, we will be able to collect and analyze data on about 700 SBMA patients overall, in a both cross-sectional and longitudinal retrospective study. Such project, with the largest SBMA cohort ever analyzed, will result in an increased knowledge on the characteristics and phenotype of the disease. It will improve clinical trial readiness and will be useful for future clinical trials.
Collaborators for this project are the following: Azienda Ospedale-Università di Padova (Dr Sorarù); Centro clinico NEMO Adulti, Roma (Dr Amelia Conte/Dr Mario Sabatelli); MRC Centre for Neuromuscular Disease UCL Institute of Neurology, London (Dr Pietro Fratta); Department of Neurology, Kyungpook National University Chilgok Hospital Daegu, Republic of Korea (Dr Jin-Sung Park); Hôpital Pitié-Salpêtrière, Paris, France (Dr Pierre-François Pradat, Giorgia Querin); Inherited Neuromuscolar Diseases Unit, Bethesda MD, USA (Christopher Grunseich, Dr Kenneth Fischbeck); Nagoya University Graduate School of Medicine, Department of Neurology, Nagoya Japan (Dr. Masahisa Katsuno); Department of Neurodegenerative Diseases and Gerontopsychiatry, University of Bonn, Germany (Dr Patrick Weydt); Neuromuscular Research Center, Tampere University, Finland (Dr Manu Jokela and Johanna Palmio); Neurogenetics Unit, Department of Neurology, National and Kapodistrian University of Athens, Greece (Dr Georgios Koutsis); Hotchkiss Brain Institute, Department of Clinical Neurosciences, University of Calgary, Alberta, Canada (Dr Gerald Pfeffer); Department of Neurology, Istanbul Faculty of Medicine, Turkey (Dr Yesim Parman and Arman Cakar).
Study design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 700 SBMA affected individuals
Key inclusion criteria (1 listed)
- Individuals affected by SBMA for whom clinical data have been historically collected by the referral centres
Key exclusion criteria (1 listed)
- na
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS)
Time frame: 3 years
Change in total score of the Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS), a disease-specific functional scale assessing bulbar, spinal, trunk, and respiratory function. The SBMAFRS total score ranges from 0 to 56 points. Higher scores indicate better functional status.
Six-Minute Walk Test (6MWT) Distance
Time frame: 3 years
Change in distance walked during the Six-Minute Walk Test (6MWT). The 6MWT measures the total distance (in meters) that a participant is able to walk on a flat surface in six minutes. Greater distance indicates better ambulatory and endurance capacity.
Age at Onset of First SBMA Symptoms
Time frame: assessed once
Age (in years) at onset of first reported SBMA-related symptom (hand tremor/cramps/muscular weakness, dysarthria, dysphagia, handrail requirement, use of support/wheelchair, development of pneumonia)
3 secondary outcomes
Creatine Phosphokinase (CPK) Serum Levels
Time frame: 3 years
Sensory Signs and Symptoms
Time frame: 3 years
Presence of Gynecomastia
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
- Background: PubMed 27444956Querin G, DaRe E, Martinelli I, Bello L, Bertolin C, Pareyson D, Mariotti C, Pegoraro E, Soraru G. Validation of the Italian version of the SBMA Functional Rating Scale as outcome measure. Neurol Sci. 2016 Nov;37(11):1815-1821. doi: 10.1007/s10072-016-2666-y. Epub 2016 Jul 21.
- Background: PubMed 35132468Steinmetz K, Rudic B, Borggrefe M, Muller K, Siebert R, Rottbauer W, Ludolph A, Buckert D, Rosenbohm A. J wave syndromes in patients with spinal and bulbar muscular atrophy. J Neurol. 2022 Jul;269(7):3690-3699. doi: 10.1007/s00415-022-10992-5. Epub 2022 Feb 7.
- Background: PubMed 24759840Araki A, Katsuno M, Suzuki K, Banno H, Suga N, Hashizume A, Mano T, Hijikata Y, Nakatsuji H, Watanabe H, Yamamoto M, Makiyama T, Ohno S, Fukuyama M, Morimoto S, Horie M, Sobue G. Brugada syndrome in spinal and bulbar muscular atrophy. Neurology. 2014 May 20;82(20):1813-21. doi: 10.1212/WNL.0000000000000434. Epub 2014 Apr 23.
- Background: PubMed 26481168Koutsis G, Kladi A, Breza M, Karadima G, Panas M. Spinobulbar muscular atrophy (Kennedy's disease): A rare diagnosis in the Greek population. J Neurol Sci. 2015 Dec 15;359(1-2):450-1. doi: 10.1016/j.jns.2015.10.021. Epub 2015 Oct 21. No abstract available.
- Background: PubMed 35639850Cho HJ, Shin JH, Park YE, Sohn E, Nam TS, Kang MG, Park JM, Park D, Park JS. Characteristics of spinal and bulbar muscular atrophy in South Korea: a cross-sectional study of 157 patients. Brain. 2023 Mar 1;146(3):1083-1092. doi: 10.1093/brain/awac198.
- Background: PubMed 16621916Atsuta N, Watanabe H, Ito M, Banno H, Suzuki K, Katsuno M, Tanaka F, Tamakoshi A, Sobue G. Natural history of spinal and bulbar muscular atrophy (SBMA): a study of 223 Japanese patients. Brain. 2006 Jun;129(Pt 6):1446-55. doi: 10.1093/brain/awl096. Epub 2006 Apr 18.
- Background: PubMed 24814851Fratta P, Nirmalananthan N, Masset L, Skorupinska I, Collins T, Cortese A, Pemble S, Malaspina A, Fisher EM, Greensmith L, Hanna MG. Correlation of clinical and molecular features in spinal bulbar muscular atrophy. Neurology. 2014 Jun 10;82(23):2077-84. doi: 10.1212/WNL.0000000000000507. Epub 2014 May 9.
- Background: PubMed 10637462Rosen RC, Cappelleri JC, Smith MD, Lipsky J, Pena BM. Development and evaluation of an abridged, 5-item version of the International Index of Erectile Function (IIEF-5) as a diagnostic tool for erectile dysfunction. Int J Impot Res. 1999 Dec;11(6):319-26. doi: 10.1038/sj.ijir.3900472.
- Background: PubMed 8633396Plante M, Corcos J, Gregoire I, Belanger MF, Brock G, Rossingol M. The international prostate symptom score: physician versus self-administration in the quantification of symptomatology. Urology. 1996 Mar;47(3):326-8. doi: 10.1016/s0090-4295(99)80447-6.
- Background: PubMed 24876969Harris-Love MO, Fernandez-Rhodes L, Joe G, Shrader JA, Kokkinis A, La Pean Kirschner A, Auh S, Chen C, Li L, Levy E, Davenport TE, Di Prospero NA, Fischbeck KH. Assessing function and endurance in adults with spinal and bulbar muscular atrophy: validity of the adult myopathy assessment tool. Rehabil Res Pract. 2014;2014:873872. doi: 10.1155/2014/873872. Epub 2014 May 5.
- Background: PubMed 25913211Hashizume A, Katsuno M, Suzuki K, Banno H, Suga N, Mano T, Araki A, Hijikata Y, Grunseich C, Kokkinis A, Hirakawa A, Watanabe H, Yamamoto M, Fischbeck KH, Sobue G. A functional scale for spinal and bulbar muscular atrophy: Cross-sectional and longitudinal study. Neuromuscul Disord. 2015 Jul;25(7):554-62. doi: 10.1016/j.nmd.2015.03.008. Epub 2015 Mar 20.
- Background: PubMed 18642379Takeuchi Y, Katsuno M, Banno H, Suzuki K, Kawashima M, Atsuta N, Ito M, Watanabe H, Tanaka F, Sobue G. Walking capacity evaluated by the 6-minute walk test in spinal and bulbar muscular atrophy. Muscle Nerve. 2008 Aug;38(2):964-71. doi: 10.1002/mus.21077.
- Individual participant dataSharing planned: No
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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.
Silvia Fenu, MD principal investigator
Fondazione IRCCS Istituto Neurologico Carlo Besta
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of SBMA and Spinal and Bulbar Muscular Atrophy (SBMA), run by Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta at 1 site in Italy.
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 1 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 2 Mar 2026. 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 Italy. 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?
This study is still under way. Results are not available in the source record yet.
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?
Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) (time frame: 3 years). 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 NCT07443449, which also lists 27 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 2 Mar 2026.