"An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase Alfa (human recombinant tissue non-specific alkaline phosphatase fusion protein) in Infants and Children ≤ 5 Years of Age with Hypophosphatasia (HPP). " Read more about "An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase Alfa (human recombinant tissue non-specific alkaline phosphatase fusion protein) in Infants and Children ≤ 5 Years of Age with Hypophosphatasia (HPP). "
An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency Read more about An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
A two part seamless, open label, multi-center study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with type 1 spinal muscular atrophy. Read more about A two part seamless, open label, multi-center study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with type 1 spinal muscular atrophy.
A three-arm, randomized, open-label, phase II study of everolimus in combination with exemestane versus everolimus alone versus capecitabine in the treatment of postmenupausal women with estrogen receptor postive, locally advanced, recurrent or metastatic breast cancer after recurrence or progression on prior letrozole or anastrozole. Read more about A three-arm, randomized, open-label, phase II study of everolimus in combination with exemestane versus everolimus alone versus capecitabine in the treatment of postmenupausal women with estrogen receptor postive, locally advanced, recurrent or metastatic breast cancer after recurrence or progression on prior letrozole or anastrozole.
A Randomized, Open-label, Multicentral, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet) Read more about A Randomized, Open-label, Multicentral, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
A randomized, double-blind, placebo-controlled phase II study to investigate the efficacy and safety of riociguat (0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg TID) in patients with symptomatic pulmonary hypertension associated with idiopathic interstitial pneumonias (IIP). Read more about A randomized, double-blind, placebo-controlled phase II study to investigate the efficacy and safety of riociguat (0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg TID) in patients with symptomatic pulmonary hypertension associated with idiopathic interstitial pneumonias (IIP).
A Randomized Phase II Trial for Patients With p16 Positive, Non-Smoking Associated, Locoregionally Advanced Oropharyngeal Cancer Read more about A Randomized Phase II Trial for Patients With p16 Positive, Non-Smoking Associated, Locoregionally Advanced Oropharyngeal Cancer
A phase II, multicenter, open label study of treatment escalation to ACVD (Adriamycin, Cyclophosphamide, Vinblastine and Dacarbazine) and Brentuximab-Vedotin in advanced-stage Hodgkin Lymphoma patients with a positive interim PET scan after 2 ABVD cycles (OPTIMIST study) Read more about A phase II, multicenter, open label study of treatment escalation to ACVD (Adriamycin, Cyclophosphamide, Vinblastine and Dacarbazine) and Brentuximab-Vedotin in advanced-stage Hodgkin Lymphoma patients with a positive interim PET scan after 2 ABVD cycles (OPTIMIST study)
"A Phase II open-label, single-arm, multi-center study of ruxolitinib added to corticosteroids in pediatric subjects with moderate and severe chronic graft vs. host disease after allogeneic stem cell transplantation" Read more about "A Phase II open-label, single-arm, multi-center study of ruxolitinib added to corticosteroids in pediatric subjects with moderate and severe chronic graft vs. host disease after allogeneic stem cell transplantation"
"A Phase 2/3, Randomized, Double-blind, Placebo- and Active-controlled, Parallel-group, Multicenter Protocol to Evaluate the Efficacy and Safety of Guselkumab in Participants with Moderately to Severely Active Crohn's Disease " Read more about "A Phase 2/3, Randomized, Double-blind, Placebo- and Active-controlled, Parallel-group, Multicenter Protocol to Evaluate the Efficacy and Safety of Guselkumab in Participants with Moderately to Severely Active Crohn's Disease "
