17 results
During the last several years, substantial progress has been made regarding the biologic characterization of the acute leukemias. The traditional understanding that myeloid leukemic cells are developmentally similar to their normal hematopoietic…
The main purpose of the study is to evaluate the safety and tolerability (any toxicities and side effects) of SAR405838 and Pimasertib and that includes identifying the maximum safe and tolerable dose of the study drugs. Also to assess the anti-…
To evaluate the efficacy of pasireotide LAR and everolimus alone or in combination in progressive patients with a well differentiated neuroendocrine tumor of the lung or thymus.
PrimaryTo evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:* Incidence and severity of adverse events and serious adverse events* Changes in vital signs, laboratory assessments, and ECGs*…
Primary objective:To investigate the safety of turoctocog alfa pegol during continuous use for prevention and treatment of bleeding episodes of previously turoctocog alfa pegol treated severe haemophilia A patients.Secondary objectives:To…
Primary objectives* To determine the Overall Response Rate (ORR) in patients with (FGFR3MUT) advanced urothelial carcinoma treated with TKI258* To determine the Overall Response Rate (ORR) in patients with (FGFR3WT) advanced urothelial carcinoma…
The purpose of the study is to collect long-term data of safety, tolerability, and efficacy to support the submission of marketing authorisation of one-month dosing regimen of degarelix for treatment of prostate cancer.Primary Objective:•To evaluate…
The primary objective of this study is to demonstrate the superior antiviral efficacy of the combination of peginterferon alpha-2a plus telbivudine vs peginterferon alpha-2a monotherapy as demonstrated by HBV DNA non-detectability using the COBAS…
The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and femaleparticipants less than or equal to 6 months of age with IOPD.
Primary objectiveTo confirm the effect on glycaemic control of once weekly insulin icodec in combination with insulin aspart, in subjects with T1D. This includes comparing the difference in change from baseline in HbA1c between once weekly insulin…
Primary objectives:- To confirm the haemostatic effect of Mim8 as treatment prophylaxis for adult and adolescent patients with haemophilia A with or without inhibitors.Secondary objectives:- To investigate safety of Mim8 prophylaxis in adults and…
Primary objective: To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.Secondary objectives• To investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without…
Participants enrolling from study CLNP023B12301• Primary Objective: To evaluate the long-term safety and tolerability of iptacopan in participants with C3G
The primary objective is to evaluate the long-term safety and tolerability of iptacopan in eligible participants. The primary clinical question of interest is: what is the long-term safety and tolerability of iptacopan in IgAN participants including…
Primary objectiveTo demonstrate the effect on glycaemic control of once weekly insulin icodec in combination with insulin aspart, with or without non-insulin anti-diabetic drugs, in subjects with T2D on a basal-bolus regimen. This includes comparing…
Run-in phase I: To confirm the safety and tolerability of BIBF 1120 up to a dose level of 200 mg b.i.d added to a standard dose of cisplatin/gemcitabine in first line NSCLC patients with squamous cell histology. Pharmacokinetics of BIBF 1120 and…
To investigate safety and tolerability and to collect exploratory efficacy data of long-term treatment with Org 50081 in adult patients with chronic primary insomnia.