There are about 9745 clinical studies being (or have been) conducted in Israel. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. Until recently, the mainstay of treatment for these patients was supportive medical care. However, advances in medical treatment focusing on gene replacement, gene enhancement, motor neuron protection and muscle enhancement is likely to change the management and prognosis of these patients in the future. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options.
Persistent developmental stuttering (PDS) is diagnosed when developmental stuttering persists beyond adolescence. Most stutterers experience vast improvement in stuttering during childhood and it generally disappears within five years. A minority of stutterers continue stuttering over age 18, often accompanied by social and personal difficulties. Following a report of a 75 year old woman, with severe Persistent developmental stuttering , who experienced significant improvement in her stuttering since treated by Ramipril for hypertension, we scrutinized the literature, and discovered that there is a physiological basis for this surprising reaction. Ace inhibitors, such as Ramipril, might in fact be successful for treating Persistent developmental stuttering . In theory, it seems that ACE inhibitors, such as Ramipril could improve stuttering by reducing striatum dopamine levels. 1. Stuttering is associated with high striatum dopamine levels 2. Angiotensin receptors are present in the striatum 3. Angiotensin causes elevated striatum dopamine levels 4. ACE inhibitors penetrate the blood brain barrier and reduce brain angiotensin II levels. Methods The study will begin as a pilot study in which 10 stuttering patients will be recruited for 12 weeks on open label Ramipril 1.25mg/d. If there is improvement in at least 2 of the stuttering patients, we will continue to the main study. Efficacy Evaluation: 1. The MINI Neuropsychiatric interview will be used to rule out major neuropsychiatric conditions 2. Stuttering evaluation 1. Stuttering Severity instrument Version 4 (SSI-4) (Riley 2009) 2. SLD :Percentage of stuttered syllables (Yairi 2015) 3. The Subjective Screening of Stuttering (SSS) 4. Speech Situation Checklist (Brutten 1975,1981) 3. Leibowitz Social Anxiety Scale (Leibowitz 1987) The efficacy evaluation will be performed by speech therapists. All evaluations will be will be recorded on video Safety evaluation: 1. Blood pressure: The average of three consecutive measures. Blood pressure will be measure in both arms on the first meeting, and thereafter on the arm with the highest measurements. 2. Orthostatic hypotension will be defined as a drop of 20mmHg systolic or 10mmHg diastolic, one and three minutes after standing from sitting position. 3. Creatinine clearance will be calculated by the MDRD method (Levy 2006) GFR, in mL/min per 1.73 m2 = 186.3 x SCr (exp[-1.154]) x Age (exp[-0.203]) x (0.742 if female) x (1.21 if black)
MOMENTUM is a randomized, double-blind, active control Phase 3 trial intended to confirm the differentiated clinical benefits of the investigational drug momelotinib (MMB) versus danazol (DAN) in symptomatic and anemic participants who have previously received an approved Janus kinase inhibitor (JAKi) therapy for myelofibrosis (MF). The purpose of this clinical study is to compare the effectiveness and safety of MMB to DAN in treating and reducing: 1) disease related symptoms, 2) the need for blood transfusions and 3) splenomegaly, in adults with primary MF, post-polycythemia vera MF or post-essential thrombocythemia MF. The study is planned in countries including, but not limited to: Australia, Austria, Belgium, Bulgaria, Canada, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, New Zealand, Poland, Romania, Singapore, South Korea, Spain, Sweden, Taiwan, United Kingdom (UK) and United States (US). Participants must be symptomatic with a Myelofibrosis Symptom Assessment Form (MFSAF) version (v) 4.0 Total Symptom Score of >= 10 at screening, and be anemic with hemoglobin (Hgb) < 10 gram/deciliter (g/dL). For participants with ongoing JAKi therapy at screening, JAKi therapy must be tapered over a period of at least 1 week, followed by a 2-week non-treatment washout interval prior to randomization. Participants will be randomized 2:1 to orally self-administer blinded treatment: MMB plus placebo or DAN plus placebo. Participants randomized to receive MMB who complete the randomized treatment period to the end of Week 24 may continue to receive MMB in the open-label extended treatment period to the end of Week 204 (a total period of treatment of approximately 4 years) if the participants tolerates and continues to benefit from MMB. Participants randomized to receive DAN may cross-over to MMB open-label treatment in the following circumstances: at the end of Week 24 if they complete the randomized treatment period; or at the end of Week 24 if they discontinue treatment with DAN but continue study assessments and do not receive prohibited medications including alternative active anti-MF therapy; or at any time during the randomized treatment period if they meet the protocol-defined criteria for radiographically confirmed symptomatic splenic progression. Participants randomized to receive DAN who are receiving clinical benefit at the end of Week 24 may choose to continue DAN therapy up to Week 48. The comparator treatment, DAN, is an approved medication in the US and in some other countries and is recommended by national guidelines as a treatment for anemia in MF.
This is a Phase 2/3 study that comprises 5 substudies designed to evaluate the efficacy, safety, and tolerability of oral etrasimod as therapy in adult participants with moderately to severely active Crohn's disease (CD) who are refractory or intolerant to at least 1 of the current therapies for CD (ie, corticosteroids, immunosuppressants, or biologics). The overall duration of this study is up to 282 weeks, inclusive of the Screening Period, Treatment Period of up to 274 weeks (Induction, Extension or Maintenance, and Long-term Extension Periods), and the 4-Week Follow-Up Period for safety assessment.
This registry is aimed to characterize patients with severe tricuspid regurgitation for the purpose of patient selection for interventional treatment and identify factors related to adverse outcomes.
The objective of the trial is to evaluate the efficacy of dasiglucagon in reducing glucose requirements in children with persistent congenital hyperinsulinism (CHI) requiring continuous intravenous (IV) glucose administration to prevent/manage hypoglycemia.
This protocol includes 2 standalone studies with randomization, data collection, analysis and reporting conducted independently. The main objectives of this protocol are: - To evaluate the efficacy of upadacitinib compared with placebo on reduction of signs and symptoms in adults with active axial spondyloarthritis (axSpA) including biologic disease-modifying antirheumatic drug inadequate responders (bDMARD-IR) ankylosing spondylitis (AS) (Study 1) and non-radiographic axial spondyloarthritis (nr-axSpA) (Study 2). - To assess the safety and tolerability of upadacitinib in adults with active axSpA including bDMARD-IR AS (Study 1) and nr-axSpA (Study 2). - To evaluate the safety and tolerability of upadacitinib in extended treatment in adult participants with active axSpA including bDMARD-IR AS who have completed the Double-Blind Period (Study 1) and nr-axSpA who have completed the Double-Blind Period (Study 2). - To evaluate the maintenance of disease control after withdrawal of upadacitinib.
This is an open-label, non-comparator, global, multi-center, long-term safety study for evaluating safety and tolerability of linerixibat in participants with cholestatic pruritus in primary biliary cholangitis (PBC) who participated in a prior clinical trial with linerixibat (BAT117123 [NCT01899703], 201000 GLIMMER [NCT02966834] (group 1) or 212620 GLISTEN [NCT00210418]) (group 2). All participants will receive open-label linerixibat for the duration of the study. The study duration is expected to last until the study's end or until linerixibat can be lawfully made available to participants. However, the total duration of study participation will vary by participant depending upon the time of entry relative to study end in their respective country.
This study will recruit families with a daughter with Rett syndrome living in either Australia, Denmark or Israel, and thereafter deliver individually designed participation programs using telehealth strategies. We will evaluate the effectiveness of the programs on reducing sedentary behaviours, increasing physical activity and increasing quality of life.
The purpose of this study is to see if the study drug, tirzepatide administered once weekly, is safe and effective as a treatment for Nonalcoholic Steatohepatitis (NASH).