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A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…

Gait retraining by real-time feedback in patients with knee osteoarthritis
(KneeMo Feedback Study )

A) To evaluate the use of real-time visual and audio feedback on the knee adduction moment and on kinematic patterns during gait in patients with knee osteoarthritis to decrease the biomechanical load on the knee via implicit learning and explicit…

A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

Primary objective:To assess the efficacy of BMN 045 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives:To assess the safety and tolerability of BMN 045 after 48 weeks of treatment in all study…

Phase 2 Study, Multicenter, Open-Label Extension (OLE) Study in Rheumatoid Arthritis Subjects Who Have Completed a Preceding Phase 2 Randomized Controlled Trial (RCT) with ABT-494

To evaluate the long-term safety, tolerability, and efficacy of ABT-494 in RA subjects who have completed Study M13-550 or Study M13-537 Phase 2 RCT with ABT-494.

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophy

Primary objective: To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives: - To assess the safety and tolerability of BMN 053 after single…

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…

Phase II trial of Linsitinib (anti-IGF-1R/IR) in patients with relapsed and/or refractory Ewing Sarcoma

Primary ObjectivesTo determine the pharmacodynamic effect of linsitinib in the tumourTo evaluate the safety and tolerability of linsitinibSecondary ObjectivesTo determine the clinical outcomeTo conduct pharmacokinetic assays with linsitinib…

Mesenchymal stromal cells for treatment of drug resistant pediatric Juvenile idiopathic arthritis

To find out if intravenous MSC is a safe treatment for children with therapy-resistant JIA

Pilot study on effectiveness and safety of Shape Memory Scoliosis Correction Instrument

Primary Objectives:• show effectiveness of the SMSC instrument in 3D correction of the spine prior to placement of the implants.The hypothesis is that to achieve de-rotation of the spine without failure of the pedicle screws, de-rotation has to be…

Phase II study of nilotinib efficacy in pigmented villo-nodular synovitis/tenosynovial giant cell tumor (PVNS/TGCT)

PRIMARY OBJECTIVEThe primary objective of the study will be to determine the efficacy of 12 weeks of nilotinib treatment as measured by the non progression rate (Complete response + Partial Response + Stable disease according to Response Evaluation…

A phase II study of ARA 290 as therapeutic strategy in rheumatoid arthritis

This is an open label proof of principle study, in 14 patients. The objective of the study is to test the efficacy and safety of ARA 290 in patients with active rheumatoid arthritis. To assess the efficacy, disease activity is examined based on…

Randomized controlled trial of taping patients with a shoulder subacromial impingement syndrome

To examine the effectiveness of taping shoulders with a subacromial impingement syndrome using a rondomized controlled trial.

A Phase 2, Multicenter, Randomized, Active-Controlled, Parallel-Group, Dose-Finding and Safety Study of Recombinant Human Bone Morphogenetic Protein-2 (rhBMP-2)/Calcium Phosphate Matrix (CPM) in Subjects With Decreased Bone Mineral Density.

Onset of effect of systemic therapy to enhance bone density (eg, bisphosphonates) is not immediate and may not adequately protect subjects with decreased bone density from fracture.Bisphosphonate therapy will typically require 2 to 3 years of active…

Arm training in boys with Duchenne Muscular Dystrophy

To determine whether 3D arm training with arm-support is effective in delaying the loss of arm functions in daily activities from an early age onwards in boys with DMD.

Assessment of asymmetrical innervation of the right and left m. puborectalis as a possible cause of idiopathic incontinence, by using concentric needle electrodes in an electromyography investigation of the pelvic floor muscles.

Is it possible to make a discrimination in muscle activity between the right and left puborectalis muscle by using concentric needle electrodes in an electromyography investigation of the pelvic floor muscles?When a asymmetry is found, what can be…

CT/MR guided cryoablation for painful bone metastasis: an observational trial.

The main objective of the study was to prove the effectiveness of cryoablation in palliating pain and correlate the patients with a matched group of the Dutch bone metastasis study. The secondary objectives are to determine the accuracy of cryoprobe…

A clinical, prospective feasibility study to assess the effectiveness and safety of micro-structured TCP granules as a bone augmentation material in sinus floor elevation procedures.

The primary objective is to determine whether micro-structured TCP granules provides sufficient bone volume as basis to insert endosseous dental implants in a two staged sinus floor elevation technique by benefiting from the clinical handling…

Eccentric Training of the Supraspinatus tendon in Shoulder Impingement syndrome. A pilot study

The main objective is to obtain more evidence in the effects of eccentric strengthening training of the supraspinatus tendon in SIS by comparing the effects eccentric strengthening training with the effects of usual care used in SIS. Secondary…

A Long-term Assessment of Safety and Physical Function with AMG 108 Subcutaneous Monthly Treatment in Subjects with Rheumatoid Arthritis

Primary Objective: The primary objective of the study is to assess long-term safety of AMG 108 (125 mg, 250 mg) SC in subjects with RA previously enrolled in study 20050168.Secondary Objective(s): The secondary objectives of the study are:* To…

The Effects of Epidermal Growth Factor Receptor (EGFR) Inhibition on Pulmonary Arterial Hypertension Associated with Systemic Sclerosis
A phase II controlled open-label safety and efficacy study.

The first objective is to evaluate the safety of cetuximab in patients with scleroderma associated PAH. The secondary objective is to assess efficacy.