Psychological Concomitants of Morquio Syndrome (The MAP Study)
Cuvinte cheie
Abstract
Datele
Ultima verificare: | 11/30/2013 |
Primul depus: | 12/12/2012 |
Inscriere estimată trimisă: | 12/17/2012 |
Prima postare: | 12/18/2012 |
Ultima actualizare trimisă: | 12/01/2013 |
Ultima actualizare postată: | 12/03/2013 |
Data actuală de începere a studiului: | 06/30/2012 |
Data estimată de finalizare primară: | 09/30/2013 |
Data estimată de finalizare a studiului: | 09/30/2013 |
Stare sau boală
Fază
Criterii de eligibilitate
Vârste eligibile pentru studiu | 18 Years La 18 Years |
Sexe eligibile pentru studiu | All |
Metoda de eșantionare | Non-Probability Sample |
Acceptă voluntari sănătoși | da |
Criterii | Inclusion Criteria: 1. Documented clinical diagnosis of MPS IV based on clinical signs and symptoms of MPS IV and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IV. 2. Patient is at least 18 years old. 3. Patient is not currently receiving enzyme replacement therapy for MPS IV. 4. Patient must provide written, informed consent prior to study participation. Exclusion Criteria: 1. Previous treatment with ERT 2. Previous hematopoietic stem-cell transplant 3. Patient has a clinically significant disease (with the exception of symptoms of Morquio), including clinically significant immunologic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would confound the effects of Morquio upon study variables |
Rezultat
Măsuri de rezultate primare
1. ASEBA Self-Report [At enrollment, as a single-timepoint only]
Măsuri de rezultate secundare
1. Brief Pain Inventory [At enrollment, as a single-timepoint only]
2. SF-36 [At enrollment, as a single-timepoint only]