A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies
Completed
Conditions studied: Molybdenum Cofactor Deficiency, Isolated Sulfite Oxidase Deficiency
In brief
Primary objective: 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
Key facts
- Study ID
- NCT01735188
- Run by
- Origin Biosciences
- People needed
- 65
- Starts
- 2013-08-01
- Expected to finish
- 2016-06-01
- Last updated by the study team
- 2019-03-19
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Both living and deceased patients of any age will be considered for study inclusion.
- Diagnosis of MoCD or isolated SOX deficiency
- Documented informed consent
You may not qualify if…
- MoCD Type A patient who was in Study ALX-MCD-501
- Deceased patients with unknown genotype (as of Amendment 4)
Where it is running
- 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
- 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)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.