Oral function in Patients with Spinal Muscular Atrophy
Organizational Data
- DRKS-ID:
- DRKS00015842
- Recruitment Status:
- Recruiting complete, study continuing
- Date of registration in DRKS:
- 2019-07-30
- Last update in DRKS:
- 2023-11-28
- Registration type:
- Prospective
Acronym/abbreviation of the study
No Entry
URL of the study
No Entry
Brief summary in lay language
We measure maximum bite force / endurance and maximum tongue pressure / endurance in patients with spinal muscular atrophy. A fifth outcome parameter is the maximum mouth opening measured by a ruler. The patients receive a therapy with Nusinersen or Risdiplam or no therapy. We hope to evaluate the effect of the therapy on the bulbar function based on the measurements.
Brief summary in scientific language
Spinal muscular atrophy (SMA) is characterized by progressive spinal and bulbar muscle weakness and atrophy caused by the degeneration of alpha-motoneurons. The recent approval of the antisense oligonucleotide nusinersen highlights the need for reliable clinical tools to evaluate motor function in patients with SMA. This study’s aim is to test if oral function measurements are suitable to quantify changes of altered bulbar function during nusinersen therapy. We want to include patients at an advanced state of SMA. In patients in an advanced stage, the maximum bite force and tongue pressure could be a quantitative measure of the bulbar function and thus an alternative to conventional motor scales, which require residual mobility of the extremities and of the head. The aim of this study is to perform isometric strength measurements in SMA patients to show possible changes in oral function indicating changes in bulbar function. Additionally endurance measurements and the measurement od maximum mouth opening are planned. A piezoelectric force sensor registers the maximum bite force in the posterior region recorded by the I-Scan software. Relative bite force changes in different treatment phases are analyzed. Tongue pressure is measured with a hendheld device IOPI. Maximum mouth opening is measured with a ruler.
Health condition or problem studied
- ICD10:
- G12.1 - Other inherited spinal muscular atrophy
- Healthy volunteers:
- No Entry
Interventions, Observational Groups
- Arm 1:
- We measure oral function (maximum bite force and endurance, maximum tongue pressure and endurance, maximum mouth opening) in patients with spinal muscular atrophy that are treated with Nusinersen (Biogen) or Risdiplam (Roche) at different time points.
- Arm 2:
- We measure oral function in naive patients with spinal muscular at different time points.
Endpoints
- Primary outcome:
- Relative changes in oral function. Measured with piezoelectric transducers and an IOPI hendheld device. Measurements are repeated over the course of one year.
- Secondary outcome:
- Relative change of oral function: maximum mouth opening, measured by a ruler.
Study Design
- Purpose:
- Other
- Retrospective/prospective:
- Prospective
- Study type:
- Non-interventional
- Longitudinal/cross-sectional:
- Longitudinal study
- Study type non-interventional:
- No Entry
Recruitment
- Recruitment Status:
- Recruiting complete, study continuing
- Reason if recruiting stopped or withdrawn:
- No Entry
Recruitment Locations
- Recruitment countries:
-
- Germany
- Number of study centers:
- Multicenter study
- Recruitment location(s):
-
- University medical center Poliklinik für Kieferorthopädie, Klinik und Poliklinik für Neurologie Köln
- University medical center Klinik für Neurologie Essen
Recruitment period and number of participants
- Planned study start date:
- 2019-10-01
- Actual study start date:
- 2020-07-24
- Planned study completion date:
- 2024-12-31
- Actual Study Completion Date:
- No Entry
- Target Sample Size:
- 50
- Final Sample Size:
- 58
Inclusion Criteria
- Sex:
- All
- Minimum Age:
- 18 Years
- Maximum Age:
- 50 Years
- Additional Inclusion Criteria:
- Patients with spinal muscular atrophy type I, II or III.
Exclusion Criteria
Maxmimum mouth opening < 8mm
Addresses
Primary Sponsor
- Address:
- Poliklinik für Kieferothopädie, Uniklinik KölnKerpener Str. 3250931 KölnGermany
- Telephone:
- No Entry
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
- Investigator Sponsored/Initiated Trial (IST/IIT):
- Yes
Contact for Scientific Queries
- Address:
- Poliklinik für Kieferorthopädie, Uniklinik KölnDr. Teresa KruseKerpener Str. 3250931 KölnGermany
- Telephone:
- 0049 221 478 4712
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Contact for Public Queries
- Address:
- Poliklinik für Kieferorthopädie, Uniklinik KölnDr. Teresa KruseKerpener Str. 3250931 KölnGermany
- Telephone:
- 0049 221 478 4712
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Principal Investigator
- Address:
- Poliklinik für Kieferorthopädie, Uniklinik KölnDr. Teresa KruseKerpener Str. 3250931 KölnGermany
- Telephone:
- 0049 221 478 4712
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Sources of Monetary or Material Support
Commercial (pharmaceutical industry, medical engineering industry, etc.)
- Address:
- Biogen GmbH81677 MünchenGermany
- Telephone:
- No Entry
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Institutional budget, no external funding (budget of sponsor/PI)
- Address:
- Poliklinik für Kieferorthopädie, Uniklinik KölnKerpener Str. 3250931 KölnGermany
- Telephone:
- 0049 221 478 4712
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Ethics Committee
Address Ethics Committee
- Address:
- Ethikkommission der Medizinischen Fakultät der Universität zu KölnGleueler Str. 26950935 KölnGermany
- Telephone:
- +49-221-478 82900
- Fax:
- +40-221-478 82905
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Vote of leading Ethics Committee
- Vote of leading Ethics Committee
- Date of ethics committee application:
- 2019-03-05
- Ethics committee number:
- 19-1137_1
- Vote of the Ethics Committee:
- Approved
- Date of the vote:
- 2019-06-13
Further identification numbers
- Other primary registry ID:
- No Entry
- EudraCT Number:
- No Entry
IPD - Individual Participant Data
- Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
- Yes
- IPD Sharing Plan:
- Only in the context of scientific publications (e.g. study protocol).
Study protocol and other study documents
- Study protocols:
- Kruse, Teresa, et al. "The maximum bite force for treatment evaluation in severely affected adult SMA patients—Protocol for a longitudinal study." Frontiers in Neurology 11 (2020): 139.
- Study abstract:
- No Entry
- Other study documents:
- No Entry
- Background literature:
- No Entry
- Related DRKS studies:
- No Entry
Publication of study results
- Planned publication:
- No Entry
- Publikationen/Studienergebnisse:
- Kruse T, Heller R, Wirth B, Glöggler J, Wurster CD, Ludolph AC, Braumann B. Maximum bite force in patients with spinal muscular atrophy during the first year of nusinersen therapy - A pilot study. Acta Myol. 2020 Jun 1;39(2):83-89. doi: 10.36185/2532-1900-010. PMID: 32904902; PMCID: PMC7460731.
- Kruse T, Shamai S, Leflerovà D, Wirth B, Heller R, Schloss N, Lehmann HC, Brakemeier S, Hagenacker T, Braumann B, Wunderlich G. Objective measurement of oral function in adults with spinal muscular atrophy. Orphanet J Rare Dis. 2023 May 3;18(1):103. doi: 10.1186/s13023-023-02688-4. PMID: 37138365; PMCID: PMC10155305.
- Date of first publication of study results:
- 2020-06-01
- DRKS entry published for the first time with results:
- No Entry
Basic reporting
- Basic Reporting / Results tables:
- No Entry
- Brief summary of results:
- No Entry