phenotypeS in Non Ambulant Duchenne Muscular Dystrophy : Characterizing Phenothypes in Non Ambulant Duchenne Muscular Dystrophy
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function, to retrospectively review similar information on the data collected in the last decade and to establish the effect of steroids after loss of ambulation on different aspects of function.We also aim to use this integrated approach to identify patterns of severity and progression, the most appropriate outcome measures and endpoints in each group and possible genotype/phenotype correlations..
| Medienart: |
|
|---|
Klinische Studie| Erscheinungsjahr: |
2024 |
|---|---|
| Erschienen: |
2024 |
| Enthalten in: |
ClinicalTrials.gov - (2024) vom: 16. Apr. Zur Gesamtaufnahme - year:2024 |
|---|
| Sprache: |
Englisch |
|---|
| Links: |
Volltext [kostenfrei] |
|---|
| Themen: |
610 |
|---|
| Anmerkungen: |
Source: Link to the current ClinicalTrials.gov record., First posted: April 16, 2024, Last downloaded: ClinicalTrials.gov processed this data on April 24, 2024, Last updated: April 24, 2024 |
|---|
| Study ID: |
NCT06366815 |
|---|---|
| Veröffentlichungen zur Studie: |
|
| fisyears: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
CTG009680659 |
|---|
| LEADER | 01000caa a22002652 4500 | ||
|---|---|---|---|
| 001 | CTG009680659 | ||
| 003 | DE-627 | ||
| 005 | 20240424010604.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 240417s2024 xx |||||o 00| ||eng c | ||
| 035 | |a (DE-627)CTG009680659 | ||
| 035 | |a (UBBS_Klinische_Studien)NCT06366815 | ||
| 035 | |a (UBBS_Klinische_Studien)4619 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 245 | 1 | 0 | |a phenotypeS in Non Ambulant Duchenne Muscular Dystrophy |b Characterizing Phenothypes in Non Ambulant Duchenne Muscular Dystrophy |
| 264 | 1 | |c 2024 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Source: Link to the current ClinicalTrials.gov record., First posted: April 16, 2024, Last downloaded: ClinicalTrials.gov processed this data on April 24, 2024, Last updated: April 24, 2024 | ||
| 520 | |a The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function, to retrospectively review similar information on the data collected in the last decade and to establish the effect of steroids after loss of ambulation on different aspects of function.We also aim to use this integrated approach to identify patterns of severity and progression, the most appropriate outcome measures and endpoints in each group and possible genotype/phenotype correlations. | ||
| 650 | 2 | |a Muscular Dystrophies | |
| 650 | 2 | |a Muscular Dystrophy, Duchenne | |
| 650 | 4 | |a Study Type: Observational | |
| 650 | 4 | |a Recruitment Status: Recruiting | |
| 650 | 4 | |a 610 | |
| 773 | 0 | 8 | |i Enthalten in |t ClinicalTrials.gov |g (2024) vom: 16. Apr. |
| 773 | 1 | 8 | |g year:2024 |g day:16 |g month:04 |
| 856 | 4 | 0 | |u https://clinicaltrials.gov/show/NCT06366815 |x 0 |z kostenfrei |3 Volltext |
| 912 | |a GBV_CTG | ||
| 951 | |a AR | ||
| 952 | |j 2024 |b 16 |c 04 | ||