NCT01902940

Brief 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. 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
350

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jun 2013

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2013

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

July 3, 2013

Completed
15 days until next milestone

First Posted

Study publicly available on registry

July 18, 2013

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2013

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2013

Completed
Last Updated

September 1, 2015

Status Verified

August 1, 2015

Enrollment Period

3 months

First QC Date

July 3, 2013

Last Update Submit

August 31, 2015

Conditions

Inclusion Body Myositis, SporadicInclusion Body Myopathy, Autosomal-recessiveInclusion Body Myopathy, Autosomal-dominantCongenital Cataracts, Facial Dysmorphism, And Neuropathy

Outcome Measures

Primary Outcomes (1)

  • Manual Muscle Strength assessed by Medical Research Council (MRC)

    Retrospective

    6-months intervals

Study Arms (1)

Natural History

Assessment of natural history in IBM and CCFDN

Other: Natural History

Interventions

Natural HistoryOTHER

Assessment of natural history in IBM and CCFDN

Natural History

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with CCFDN and IBM

You may not qualify if:

  • Additional neuromuscular diseases

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Friedrich-Baur-Institut, Ludwig-Maximilians-University of Munich

Munich, Bavaria, 80336, Germany

Location

MeSH Terms

Conditions

Myositis, Inclusion BodyDistal myopathy, Nonaka typeInclusion body myopathy, autosomal dominantCongenital Cataracts, Facial Dysmorphism, And Neuropathy

Condition Hierarchy (Ancestors)

MyositisMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Study Officials

  • Maggie C Walter, MD, MA

    Friedrich-Baur-Institute, Dept. of Neurology

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor, MD, MA

Study Record Dates

First Submitted

July 3, 2013

First Posted

July 18, 2013

Study Start

June 1, 2013

Primary Completion

September 1, 2013

Study Completion

October 1, 2013

Last Updated

September 1, 2015

Record last verified: 2015-08

Locations

DE(1)