A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies
NCT ID: NCT01735188
Last Updated: 2019-03-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
65 participants
OBSERVATIONAL
2013-08-31
2016-06-30
Brief Summary
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Characterize the natural history of MoCD type A in terms of survival
Secondary objectives:
1. Evaluate blood and urine for biochemical markers
2. Evaluate head circumference, seizure activity and neurologic outcomes
3. To evaluate brain MRI
4. Compare blood and urine analysis, head circumference, seizure activity and neurologic outcomes to MRI findings
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Detailed Description
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Conditions
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Study Design
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OTHER
OTHER
Study Groups
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Living
No interventions assigned to this group
Deceased
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of MoCD or isolated SOX deficiency
3. Documented informed consent
Exclusion Criteria
2. Deceased patients with unknown genotype (as of Amendment 4)
ALL
No
Sponsors
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Origin Biosciences
INDUSTRY
Responsible Party
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Locations
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King Fahad Medical City
Riyadh, , Saudi Arabia
Prince Sultan Riyadh Military Medical City
Riyadh, , Saudi Arabia
Germans Trias i Pujol University Hospital
Badalona, , Spain
Hospital Sant Joan de Déu Barcelona
Esplugues de Llobregat, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Clinico Universitario
Santiago de Compostela, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
La Rabta Hospital
Tunis, , Tunisia
Ihsan Dogramaci Children's Hospital
Ankara, , Turkey (Türkiye)
Akdeniz University Hospital
Antalya, , Turkey (Türkiye)
Massachusetts General Hospital
Boston, Massachusetts, United States
UVA Health System
Charlottesville, Virginia, United States
Woodinville Pediatrics PLLC
Woodinville, Washington, United States
Hospital for Sick Children
Toronto, , Canada
Universitätsklinikum Freiburg
Freiburg im Breisgau, , Germany
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, , Germany
TU Universitätsklinikum München
München, , Germany
Fachärztin für Kinder und Jugendmedizin
Offenbach, , Germany
HaEmek Medical Center
Afula, , Israel
Rambam Medical Center
Haifa, , Israel
Ospedale Pediatrico Bambin Gesu - Roma
Rome, , Italy
Wakayama Medical University
Wakayama, , Japan
Kuala Lumpur Hospital
Kuala, , Malaysia
Beatrix Children's Hospital
Groningen, , Netherlands
Leiden University Medical
Leiden, , Netherlands
The Children's Memorial Health Institute
Warsaw, , Poland
Çocuk Sağlığı ve Hastalıkları Anabilim Dalı
İzmit, , Turkey (Türkiye)
Kayseri Education and Research Hospital
Kayseri, , Turkey (Türkiye)
Birmingham Children's Hospital
Birmingham, , United Kingdom
Royal Hospital for Sick Children
Glasgow, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Manchester Academic Health Science Centre
Manchester, , United Kingdom
Countries
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Other Identifiers
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ALX-MCD-502
Identifier Type: REGISTRY
Identifier Source: secondary_id
ALX-MCD-502
Identifier Type: -
Identifier Source: org_study_id
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