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A phase 2, double-blind, randomized, placebo-controlled pharmacokinetic trial in two parallel groups to investigate possible drug- drug interactions between stiripentol or valproate and GWP42003-P in patients with epilepsy.

Primary Objective: To determine whether GWP42003-P affects the pharmacokinetic (PK) profile of stiripentol (STP) or valproate (VPA).Secondary Objective: To assess the safety and tolerability of GWP42003-P in the presence of STP or VPA.To assess…

An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia

PrimaryTo characterize the effect of 90 and 180 days of subcutaneous ALN-PCSSC on the percentage change from Day 1 inlow-density lipoprotein cholesterol (LDL-C) in subjects with homozygous familial hypercholesterolemiaSecondary* To assess the effect…

A THREE MONTH PROSPECTIVE OPEN LABEL STUDY OF THERAPY WITH FRAGMIN® (DALTEPARIN SODIUM INJECTION) IN CHILDREN WITH VENOUS THROMBOEMBOLISM WITH OR WITHOUT MALIGNANCIES.

The primary objective of this study is to:* To determine the PD profiles for treatment doses of dalteparin in pediatric subjects of different ages with or without cancer and VTE, using anti-Xa levels and a population PD analysis methodology;* To…

Treatment of congenital vascular malformations using Sirolimus: improving quality of Life

As patients with congenital vascular malformations often suffer from severe pain and morbidity due to the vascular malformation, quality of life is often signifcantly impaired. The primairy objective of the present study is to reduce pain complaints…

Randomized, double-blind, placebo controlled, multi-centre superiority phase II study to evaluate the safety, pharmacokinetic, efficacy of gabapentin liquid formulation as add-on to morphine in children from 3 months to less than 18 years of age experiencing severe chronic neuropathic or mixed pain.

The primary objective of this study is to assess the efficacy of gabapentin as add-on to morphine for the treatment of severe chronic neuropathic or mixed pain in children from 3 months to less than 18 years of age. Secondary objectives 1. To assess…

Pilot Randomized-controlled phase-IIa trial on the prevention of comorbid Depression and Obesity in Attention-deficit / Hyperactivity disorder

Primary objective: To assess the prevention of depressive symptoms by 10-weeks BLT and 10-weeks AEI vs. TAU, and to establish feasibility and effect sizes of these two kinds of interventions in combination with a m-Health app based reinforcement in…

Genistein as an add-on treatment for CF?

Our primary objective is to investigate whether a further gain in efficacy of oral Ivacaftor treatment can be reached by co-supplementation of genistein, as suggested by their highly synergistic action in intestinal organoidsOur secondary objectives…

Modulating regulatory T cell function in Juvenile Idiopathic Arthritis with Vitamin B3 (nicotinamide): a phase I/II trial focusing on safety and feasibility aspects in children with JIA.

NAM, well known as a dietary supplement, has also been extensively studied in humans in a variety of diseases in both children and adults. However, the safety, tolerability of NAM in children with JIA is yet unknown. Furthermore, studies performed…

The physiology of glucagon-like peptide-l receptor expression in patients with endogenous hyperinsulinism: correlation with histopathology

The main study aim is the in vivo and ex vivo investigation of the expression and distribution of the GLP-1R in the pancreas of children (< 16 years) with proven endogenous congenital hyperinsulinism who qualify forsurgery based on response…

A Phase 2, Randomized, Double-Blind, Dose-Ranging Study to Determine the Pharmacokinetics, Safety and Tolerability of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn*s Disease.
(A Phase 2, Randomized, Double-Blind, Dose-Ranging Study With Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn*s Disease).

Primary Objective:To evaluate vedolizumab PK in pediatric subjects with UC or CD.Secondary Objectives:* To assess the efficacy of vedolizumab IV in pediatric subjects with UC or CD.* To characterize the dose-response relationship of vedolizumab IV…

A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety and Efficacy of Avacopan (CCX168) in Patients with C3 Glomerulopathy

The primary objective is to evaluate the efficacy of avacopan compared to placebo based on histologic changes in kidney biopsies taken before and during treatment.The secondary objectives of this study include evaluation of:1. The safety of avacopan…

An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)

The primary objective of this study is to evaluate the long-term safety and tolerability of Diacerein 1% Ointment for 2 treatment cycles in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101.

A phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia (CETB115E2201)

To characterize the PK of eltrombopag at steady state after oral administration in pediatric patients with SAA.Secondary (key only, see protocol page 40-41 for all objectives): Safety and tolerability. Efficacy (overall response rate ORR).

A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1.

Primary:* Evaluate the long-term safety of multiple doses of ALN-GO1 in patients with PH1.Secundary:* Evaluate the pharmacodynamic (PD) effect of ALN-GO1 on urinary oxalate excretion* Characterize the effect of ALN-GO1 on markers of renal function.…

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of 3 Doses of MOTREM in Patients with Septic Shock. A Randomised, Double-blind, Two-stage, Placebo Controlled Study.

The objectives of the study are to: -To evaluate the safety and tolerability of MOTREM in patients with septic shock. -To evaluate the effects of MOTREM exposure over up to 5 days in patients with septic shock -To evaluate the PK/PD and dose/PD…

European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (ESMART)

This study has been transitioned to CTIS with ID 2024-514791-40-00 check the CTIS register for the current data. Based on results from a comparative review (Paoletti 2013), we hypothesize that if the toxicity profile and the PK parameters observed…

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication

Primary objective* To evaluate the efficacy in terms of overall survival (OS) of erlotinib, everolimus and dasatinib in combination with radiation therapy, in patients with a DIPG both: - compared to the other experimental treatments by comparisons…

A Phase 2b, Extension Study to Determine the Long-term Safety of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn*s Disease.;(Long-term Safety With Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or
Crohn*s Disease)

Primary Objective: • To determine the safety profile of long-term vedolizumab IV treatment in pediatric subjects with UC or CD.Secondary Objectives:• To determine the effect of long-term vedolizumab IV treatment on time to major inflammatory bowel…

Comparison of the beta cell mass during and shortly after the honeymoon phase of type 1 diabetes using Ga-68-exendin PET

The study objective is to measure beta cell mass and function in subjects with type 1 diabetes during and shortly after the honeymoon phase, to determine whether the change in metabolic control is mainly caused by a decrease in the total number of…

An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study]

To compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily in subjects with EBS.To compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS in:* Safety and tolerability* Pruritus * Pain…