4 results
The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…
Objectives: For patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) enrolled in this study, the objectives are as follows:Primary: -To assess the efficacy of bardoxolone methyl relative to placebo.Secondary…
To provide continuing open-label treatment with bardoxolone methyl as part of this extended access program while collecting ongoing safety and tolerability data of bardoxolone methyl.
To determine non-inferiority of preoperative 6 weeks of VCE to VAD in the overall metastatic rapid response rate (MetRR) in newly diagnosed stage 4 childhood renal tumours. The MetRR will include the pulmonary response rate (PRR) and the response…