4 results
To evaluate the efficacy and toxicity of gemcitabine in combination wth 131I-MIBG in pediatric patients with relapsed or progressive neuroblastoma.
Primary:* To determine the efficacy profile of PA101 delivered via a high efficiency nebulizer (eFlow®,PARI) in comparison with placebo following 6 weeks of treatment in patients with indolentsystemic mastocytosis (ISM) who are symptomatic despite…
The objectives of the study are as follows:Primary:• Part 1: To assess the pharmacokinetics and relative bioavailability of three different formulations of PA101 (4% cromolyn sodium with and without mannitol, and 6% cromolyn sodium without mannitol…
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.