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An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.

Treatment of severe acute GVHD after allogeneic hematopoietic stem cell transplantation with steroids versus MSC and steroids.
A prospective double-blind placebo-controlled randomized phase III trial

To improve the response rate to treatment of severe acute GVHD (grade II-IV with gut involvement ) by adding infusion of Mesenchymal Stroma Cells to standard high dose prednisolone.

CONgenital Cytomegalovirus: Efficacy of antiviral treatment in a non-Randomized Trial with historical control group

Primary Objective: Investigate whether early valganciclovir treatment of children with SNHL of * 20 dB, unilateral or bilateral, and a confirmed congenital CMV infection can prevent deterioration of the hearing loss at follow-up (age 18 * 22 months…

ACNS0332: Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients - A Groupwide Phase III Study

The goal of this study is to determine whether radiosensitization with carboplatin or the addition of Isotretinoin to maintenance therapy improves cure rates for children with other than average risk medulloblastoma/PNET.

A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP, MULTI-CENTER TRIAL OF PREGABALIN AS ADJUNCTIVE THERAPY IN PEDIATRIC AND ADULT SUBJECTS WITH PRIMARY GENERALIZED TONIC-CLONIC SEIZURES

Determine efficiency and safety of two different doses of pregabalin as compared to placebo.

First international Inter-Group Study for classical Hodgkin's Lymphoma in Children and Adolescents

The objective of the study is to diminish the amount of chemotherapy and/ or radiotherapy for children with Hodgkin lymphoma who are likely to receive too much treatment with current treatment protocols.

A phase 3 prospective, uncontrolled, multicenter study evaluating pharmacokinetics, efficacy, safety, and immunogenicity of BAX 855 (PEGylated full-length Recombinant FVIII) in previously treated pediatric patients with severe hemophilia A

7.2 Primary ObjectiveThe primary objective is to assess the incidence of FVIII inhibitory antibodies(>=0.6 Bethesda units [BU] using the Nijmegen modification of the Bethesda assay).7.3 Secondary Objectives1. To evaluate the PK parameters of…

A Multicentre, Long-term Safety, Efficacy and Pharmacokinetics Study of Lubiprostone in Paediatric Subjects Aged *6 to <18 years with Functional Constipation

To assess the long-term safety, efficacy, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) when administered orally for 36 weeks in paediatric subjects with functional constipation. Evaluation of lubiprostone…

A Phase 3, Multicenter, Randomized, Double-blind Study to Determine the Safety and Efficacy of MMX Mesalamine/Mesalazine in Pediatric Subjects with Mild to Moderate Ulcerative Colitis, in both Acute and Maintenance Phases

Primary: The primary objective of the Double-blind Acute Phase of the study is to assess clinical response to MMX mesalamine/mesalazine between a low and high dose in children and adolescents aged 5-17 years with mild to moderate UC.The primary…

A randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.

To assess the efficacy of GWP42003-P as an adjunctive antiepileptic treatment compared with placebo, with respect to the percentage change from baseline during the treatment period of the study in convulsive seizure frequency. The dose response…

A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa

The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, RecessiveDystrophic, or Junctional non Herlitz Epidermolysis Bullosa.The primary endpoint is the complete closure of the…

Treatment of severe steroid-refractory acute GvHD with mesenchymal stromal cells.
A phase III randomized double-blind multi-center HOVON study.

Primary objective* To improve the response rate to treatment of severe steroid-refractory acute GvHD grade II-IV (with gut and/or liver involvement) by early addition of MSC to standardized second line treatmentSecondary objectives* To study the…

An open-label canakinumab (ACZ885) dose reduction or dose interval prolongation efficacy and safety study in patients with Systemic Juvenile Idiopathic Arthritis (SJIA)

Primary:To evaluate if the proportion of patients in clinical remission on canakinumab 4mg/kg (+/- concomitant NSAID only) who are able toremain on a reduced canakinumab dose (2mg/kg every 4 weeks) or prolonged canakinumab dose interval (4mg/kg…

A multicenter, multinational, randomized, controlled, open label study, performed in children with thermal burns, to evaluate the efficacy and safety of NexoBrid as compared to standard of care (SOC) treatment

To evaluate the safety and clinical benefit of NexoBrid in hospitalized children (0-18 years) with deep partial and/or full thickness thermal burns of 1-30% TBSA and to compare NexoBrid to standard of care (SOC).

An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa
(SD-101-6.0) in Patients with Epidermolysis Bullosa

The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidermolysis Bullosa (EB).

A 12-MONTH OPEN-LABEL STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PREGABALIN AS ADJUNCTIVE THERAPY IN PEDIATRIC SUBJECTS 1 MONTH TO 16 YEARS OF AGE WITH PARTIAL ONSET SEIZURES AND PEDIATRIC AND ADULT SUBJECTS 5 TO 65 YEARS OF AGE WITH PRIMARY GENERALIZED TONIC-CLONIC SEIZURES

To evaluate the long-term safety and tolerability of pregabalin in pediatric subjects 1 month through 16 years of age with partial onset seizures and pediatric and adult subjects 5 to 65 years of age with (PGTC) seizures.

A Phase 3 Efficacy and Safety Study of Ataluren
(PTC124 ®) in Patients with Nonsense Mutation Cystic
Fibrosis

To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)

An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A

An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A.…

A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis.

The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…

A randomized, double-blind, placebo controlled study of canakinumab in patients with Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), with subsequent randomized withdrawal/ dosing frequency reduction and open-label long term treatment epochs

Primary: The primary objective of the randomized treatment epoch and for the overall study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease…