Inclusion Body Myositis, SporadicNCT01902940
Natural History in CCFDN and IBM Syndromes
An observational study of Inclusion Body Myositis, Sporadic and Inclusion Body Myopathy, Autosomal-recessive, run by Ludwig-Maximilians - University of Munich at 1 site in Germany.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Inclusion Body Myositis, Sporadic, Inclusion Body Myopathy, Autosomal-recessive, Inclusion Body Myopathy, Autosomal-dominant
- What is being tested?
- Natural History
- How many people
- 350 participants
- Where
- 1 siteGermany
- Study dates
- Jun 2013to Oct 2013
- 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
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with CCFDN and IBM
Who may be able to join
- Clinical and/or genetic diagnosis of Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and sporadic and hereditary inclusion body myopathies (IBM)
Who cannot join
- Additional neuromuscular diseases
Trial locations and contacts
1 site in 1 country.
Germany1 site
Friedrich-Baur-Institut, Ludwig-Maximilians-University of Munich
Munich, Bavaria, 80336
About this study
Official title: 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.
Read the 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.
- Conditions
- Inclusion Body Myositis, Sporadic, Inclusion Body Myopathy, Autosomal-recessive, Inclusion Body Myopathy, Autosomal-dominant, Congenital Cataracts, Facial Dysmorphism, And Neuropathy
- Being tested
- Natural History
- Medical subject headings
- Myositis, Inclusion Body, Distal myopathy, Nonaka type, Inclusion body myopathy, autosomal dominant, Congenital Cataracts, Facial Dysmorphism, And Neuropathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Natural HistoryOther
Assessment of natural history in IBM and CCFDN
| Study group | Role | What they receive |
|---|---|---|
| Natural History | Assessment of natural history in IBM and CCFDN Receives: Other: Natural History |
What is being measured
The primary outcome is the main question the study is designed to answer.
Manual Muscle Strength assessed by Medical Research Council (MRC)
Retrospective
Measured: 6-months intervals
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2013 (estimated)
- Study startJun 2013
- Primary completionSep 2013
- Study completionOct 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inclusion Body Myositis, SporadicStudies 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
- Retrospective
- Enrolment
- 350 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ludwig-Maximilians - University of Munich
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Maggie Walter, Ludwig-Maximilians - University of Munich
- Investigators
- Maggie C Walter, MD, MA, Study chair, Friedrich-Baur-Institute, Dept. of Neurology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01902940
- Sponsor's protocol ID
- CCFDN-IBM-2013
- Other IDs
- CCFDN-IBM-2013
- Status last verified
- Aug 2015
- First submitted
- 3 Jul 2013
- Last updated
- 1 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Cohort Study Assessing the Natural Course in Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and Sporadic and Hereditary Inclusion Body Myopathies (IBM)
- ClinicalTrials.gov ID
- NCT01902940
- Acronym / sponsor ID
- CCFDN-IBM-2013
- Conditions
- Inclusion Body Myositis, Sporadic, Inclusion Body Myopathy, Autosomal-recessive, Inclusion Body Myopathy, Autosomal-dominant, Congenital Cataracts, Facial Dysmorphism, And Neuropathy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 350 participants (actual)
- Lead sponsor
- Ludwig-Maximilians - University of MunichUniversity, hospital or other
- Study start
- Jun 2013
- Primary completion
- Sep 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 18 Jul 2013 (estimated)
- 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.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Natural History | – | Other: Natural HistoryAssessment of natural history in IBM and CCFDN |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with CCFDN and IBM
Key inclusion criteria (1 listed)
- Clinical and/or genetic diagnosis of Congenital Cataract Facial Dysmorphism Neuropathy Syndrome (CCFDN) and sporadic and hereditary inclusion body myopathies (IBM)
Key exclusion criteria (1 listed)
- Additional neuromuscular diseases
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 |
|---|---|---|
| Natural HistoryAssessment of natural history in IBM and CCFDN | Other | Natural History |
Primary outcome
Manual Muscle Strength assessed by Medical Research Council (MRC)
Time frame: 6-months intervals
Retrospective
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2015.
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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.
Maggie C Walter, MD, MA study chair
Friedrich-Baur-Institute, Dept. of Neurology
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 Inclusion Body Myositis, Sporadic and Inclusion Body Myopathy, Autosomal-recessive, run by Ludwig-Maximilians - University of Munich at 1 site in Germany.
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: finished, as of the sponsor's last update on 1 Sep 2015. 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 Germany. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Manual Muscle Strength assessed by Medical Research Council (MRC) (time frame: 6-months intervals). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Sep 2015.