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Clinical trial

Congenital Muscle Disease Patient and Proxy Reported Outcome Study

NCT01403402 is registered in ClinicalTrials.gov with Cure CMD as lead sponsor.

NCT NCT01403402
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Registry record

As filed in ClinicalTrials.gov.

Title
Congenital Muscle Disease Patient and Proxy Reported Outcome Study
Lead sponsor
Cure CMD
Phase
Observational
Overall status
Recruiting
Start date
September 30, 2009
Completion date
September 30, 2029
Enrollment
4,000 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

alpha-dystroglycanopathy (congenital muscular dystrophy and abnormal glycosylation of dystroglycan with severe epilepsy), alpha-dystroglycanopathy (congenital muscular dystrophy with fatty liver and infantile-onset cataract caused by trappc11 mutations), alpha-dystroglycanopathy (congenital muscular dystrophy with hypoglycosylation of dystroglycan and epilepsy), alpha-dystroglycanopathy (congenital muscular dystrophy with hypoglycosylation of dystroglycan), alpha-dystroglycanopathy (dystroglycanopathy, congenital with or without mental retardation (formerly mdc1c)), alpha-dystroglycanopathy (fukuyama cmd), alpha-dystroglycanopathy (lgmdr09 fkrp related (formerly lgmd2i)), alpha-dystroglycanopathy (lgmdr11 pomt1 related (formerly lgmd2k)), alpha-dystroglycanopathy (lgmdr13 fktn related (formerly lgmd2m)), alpha-dystroglycanopathy (lgmdr14 pomt2 related (formerly lgmd2n)), alpha-dystroglycanopathy (lgmdr15 pomgnt1 related (formerly lgmd2o)), alpha-dystroglycanopathy (lgmdr19 gmppb related (formerly lgmd2t)), alpha-dystroglycanopathy (lgmdr20 ispd related (formerly lgmd2u)), alpha-dystroglycanopathy (lgmdr24 pomgnt2 related), alpha-dystroglycanopathy (muscle eye brain disease (meb)), alpha-dystroglycanopathy (walker warburg syndrome (wws)), choline kinase b receptor - chkb, collagen vi related disorders, collagen xii related disorders, congenital muscular dystrophy not otherwise specified (including merosin positive), congenital muscular dystrophy with cataracts and intellectual disability (mdccaid), congenital muscular dystrophy with itga7 (integrin alpha-7) deficiency, congenital muscular dystrophy with joint hyperlaxity, congenital muscular dystrophy with rigid spine related to acta1, congenital myasthenic syndrome, emery-dreifuss muscular dystrophy, golga2-related congenital muscle dystrophy with brain involvement, lgmdd01 - dnajb6 (formerly lgmd1d), lgmdd05 - collagen vi related bethlem myopathy (dominant), lgmdr07 - telethonin (tcap) related (formerly lgmd2g), lgmdr08 - trim related (formerly lgmd2h), lgmdr09 - fkrp related (formerly lgmd2i), lgmdr10 - titin (ttn) related (formerly lgmd2j), lgmdr11 - pomt1 related (formerly lgmd2k), lgmdr13 - fukutin (fktn) related (formerly lgmd2m), lgmdr14 - pomt2 related (formerly lgmd2n), lgmdr15 - pomgnt1 related (formerly lgmd2o), lgmdr16 - dag1 related dystroglycanopathy (formerly lgmd2p), lgmdr17 - plectin (plec) related (formerly lgmd2q), lgmdr18 - trappc11 related (formerly lgmd2s), lgmdr19 - gmppb related (formerly lgmd2t), lgmdr20 - ispd related (formerly lgmd2u), lgmdr22 - collagen vi related bethlem myopathy (recessive), lgmdr23 - lama2 related, lgmdr24 - pomgnt2 related, limb-girdle muscular dystrophy, lmna related disorders, merosin deficient cmd (full or partial), nesprin related md (syne1), selenon related disorders (previously known as sepn1), selenon related myopathy (aka sepn1), telethonin cmd