- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01902940
Natural History in CCFDN and IBM Syndromes
August 31, 2015 updated by: Maggie Walter, Ludwig-Maximilians - University of Munich
Retrospective Cohort Study Assessing the Natural Course in Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and Sporadic and Hereditary Inclusion Body Myopathies (IBM)
So far, only limited data is available regarding the natural course in Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and sporadic and hereditary inclusion body myopathies (IBM).
Several criteria and outcome measures have led to contradicting results.
The investigators want to retrospectively assess the natural course of the disease in CCFDN and IBM patients according to the data recorded during clinical routine visits.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
We wanted to assess the natural course in Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and sporadic and hereditary inclusion body myopathies (IBM) over 10 years to gain new insights in both conditions.
Study Type
Observational
Enrollment (Actual)
350
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Bavaria
-
Munich, Bavaria, Germany, 80336
- Friedrich-Baur-Institut, Ludwig-Maximilians-University of Munich
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Patients with CCFDN and IBM
Description
Inclusion Criteria:
- Clinical and/or genetic diagnosis of Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and sporadic and hereditary inclusion body myopathies (IBM)
Exclusion Criteria:
- Additional neuromuscular diseases
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Retrospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Natural History
Assessment of natural history in IBM and CCFDN
|
Assessment of natural history in IBM and CCFDN
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Manual Muscle Strength assessed by Medical Research Council (MRC)
Time Frame: 6-months intervals
|
Retrospective
|
6-months intervals
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Study Chair: Maggie C Walter, MD, MA, Friedrich-Baur-Institute, Dept. of Neurology
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
June 1, 2013
Primary Completion (Actual)
September 1, 2013
Study Completion (Actual)
October 1, 2013
Study Registration Dates
First Submitted
July 3, 2013
First Submitted That Met QC Criteria
July 17, 2013
First Posted (Estimate)
July 18, 2013
Study Record Updates
Last Update Posted (Estimate)
September 1, 2015
Last Update Submitted That Met QC Criteria
August 31, 2015
Last Verified
August 1, 2015
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CCFDN-IBM-2013 (Other Identifier: Friedrich-Baur-Institute)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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