
Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects...
Short Bowel SyndromeThe purpose of this study is to evaluate the safety and efficacy of an investigational treatment (teduglutide) in Japanese patients with PN-dependent SBS. This study will also look at how teduglutide moves through the body (pharmacokinetics).

Efficacy and Safety Study of GB-0998 for Guillain-Barré Syndrome
Guillain-Barré SyndromeThis study will carry out to assess the efficacy of GB-0998 (intravenous immunoglobulin;400mg/kg/day for five days) in the treatment of the Guillain-Barré Syndrome based on the changes in Hughes Functional Grade (FG) as primary endpoint, and in addition, to assess the safety of GB-0998.

Acceptance and Commitment Group Therapy for Adolescents With a Range of Functional Somatic Syndromes...
Somatization DisorderSomatoform DisordersBackground: An increasing number of adolescents report recurrent functional somatic symptoms. Some experience persistent symptoms and may receive functional somatic syndromes (FSS) diagnoses (i.e. symptoms not attributable to any known conventionally defined physical disease), characterised by severe disability and reduced quality of life. The aim of this study is to: Develop an Acceptance and Commitment Therapy (ACT)-based group intervention for adolescents with severe FSS (conceptualized as Bodily Distress Syndrome (BDS), see detailed description). Examine the efficacy of group based ACT in adolescents (aged 15-19 years) with severe FSS. The ACT-based treatment, with 9 sessions of group therapy and one follow up meeting is compared to standard treatment/enhanced usual care, which is one single advisory consultation. The study includes approximately 120 patients.

Decitabine and Cytarabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia,...
Chronic Myelomonocytic Leukemia-2Myelodysplastic Syndrome2 moreThis clinical trial studies decitabine and cytarabine in treating older patients with newly diagnosed acute myeloid leukemia, myelodysplastic syndrome that is likely to come back or spread to other places in the body, or myeloproliferative neoplasm. Drugs used in chemotherapy, such as decitabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving decitabine and cytarabine may work better than standard therapies in treating cancers of the bone marrow and blood cells, such as acute myeloid leukemia, myelodysplastic syndrome, or myeloproliferative neoplasm.

Reverse Transcriptase Inhibitors in AGS
Aicardi-Goutières Syndrome (AGS)The purpose of this study is to determine if treatment with reverse transcriptase inhibitors returns the interferon signature observed in patients with AGS to normal levels.

Aerobic Exercise in Primary Sjögren's Syndrome
Sjogren's SyndromeThe purpose of this sudy is to investigate the effects of a regular aerobic exercise on aerobic capacity, fatigue, depression, quality of life, perception of symptoms and disease activity in women with pSS.

Phenotypic Specific Communication Intervention for Children With Down Syndrome
Language ImpairmentDown SyndromeChildren with Down syndrome (DS) have greater difficulty acquiring expressive language than is predicted by their general cognitive abilities and language comprehension (Miller, 1999). To date, interventions to improve communication outcomes for children with DS have met with only modest success. The proposed study is a randomized controlled trial of an early communication intervention combining two evidence-based treatments that teach expressive communication (Enhanced Milieu Teaching; EMT) and joint attention/symbolic play (Joint Attention Symbolic Play Engagement and Regulation; JASPER) using a dual mode (words +AAC). The intervention (a) teaches the foundations of communication (joint attention, play), (b) builds on positive child characteristics of social attention, (c) uses naturalistic strategies to increase the rate and complexity of communication and increase task engagement, (d) addresses the potential value of adding visual support (AAC) to spoken communication, and (e) includes parents as implementers of the intervention to promote generalization across settings and activities, and to ensure maintenance over time. It is hypothesized that children with DS enrolled in the intervention will have better language and communication skills at the end of treatment and followup than children in the BAU comparison group.The study will enroll 82 30 to 54 month old children with DS who have fewer than 20 words. Examining predictors of response to treatment and the effects of treatment on executive functioning will inform both theory and practice.

Treatment of Chronic Deep Vein Thrombosis (DVT) and Post-Thrombotic Syndrome (PTS) With the EkoSonic®...
Deep Vein ThrombosisPost-thrombotic SyndromeTo evaluate the efficacy of ultrasound accelerated thrombolysis using the EkoSonic® Endovascular System with standard infusion of thrombolytic drug for post-thrombotic syndrome from chronic venous occlusion.

Efficacy of Botulinum Toxin In Scleroderma-Associated Raynaud's Syndrome
SclerodermaRaynaud's SyndromeThis is a randomized, double-blinded, clinical trial assessing the therapeutic efficacy of Botulinum toxin A (Onabotulinumtoxin A) in treating scleroderma-associated Raynaud's syndrome. Each patient will undergo injection with a treatment dose of Botulinum toxin A in one randomly-selected hand, and the contralateral hand will be injected with sterile saline (placebo) to serve as a control. Study participants at the first study visit will complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive laser Doppler imaging to assess blood flow. After this initial assessment, the patients will undergo peri-arterial injection of Botulinum toxin A in one hand, and of sterile saline solution (placebo) in the other, in a randomized, blinded manner. Patient will report the severity of their Raynaud's symptoms weekly over the four month study period. At one month post-injection, the patient will complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive laser Doppler imaging. At four months post-injection, the patient will again complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive laser Doppler imaging. In addition, patient will be given the option of one week post-injection visit, at which point the same assessment will be performed. At the conclusion of the study, unblinding will occur.

Efficacy Study of Local Steroid Injection and Wrist Splinting for Carpal Tunnel Syndrome
Carpal Tunnel SyndromeLocal Steroid Injection1 moreCarpal tunnel syndrome is caused by the compression of median nerve at the wrist where it passes through a narrow space called carpal tunnel formed by the wrist bones, ligaments and tendons. The common symptoms include numbness and pain over the areas supplied by the median nerve namely the thumb, the index finger, the middle finger and the outer part of the ring finger. There can be loss of sensation, weakness or muscle atrophy in severe cases. The functions of the affected hands may be greatly impaired. Carpal tunnel syndrome is very common. It can lead to significant economic impact both to the affected individual and the society either by the direct treatment cost and indirectly from the working ability loss. Despite the vast burden, there is no consensus regarding its treatment so far. Surgery is generally effective and often reserved for severe cases. There are many nonsurgical treatment options. Local steroid injection and wrist splinting are among the commonest and with more evidence. Local steroid injection into the carpal tunnel can reduce the inflammation and swelling. Wrist splinting can maintain the wrist at its neutral position where the pressure at the carpal tunnel is the least. However, there are only very few studies comparing these two treatments directly. Patients complaining of finger numbness who have been confirmed to have carpal tunnel syndrome by nerve conduction test are invited to participate in the study. The patients who agreed to be recruited are asked about their basic informations and the details of the carpal tunnel syndrome symptoms. Their hands will be examined. They are asked to fill in a questionnaire specific for assessing the symptom severity and the functional status of patients with carpal tunnel syndrome. They will then be assigned to one of the two treatment groups randomly and receive the respective treatment. They need to come back for follow-up at one month and to fill in the questionnaire again. The study hypothesis is local steroid injection is more effective than wrist splinting in treating carpal tunnel syndrome.