View clinical trials related to Syndrome.
Filter by:This Phase 3 trial will enroll participants diagnosed with Lennox-Gastaut Syndrome (LGS) who are still experiencing at least 4 motor seizures involving the trunk or extremities per week, despite ongoing treatment with up to 3 antiepileptic drugs (AEDs) and who meet inclusion/exclusion criteria. Following a 28-day baseline period, participants will begin an 84-day treatment period. Participants will be assigned to receive twice daily doses of placebo or cannabidiol oral solution at the highest dose determined to be safe in a previous trial. Following study completion, all participants will be invited to receive Cannabidiol Oral Solution in an open label extension study (under a separate protocol).
100 subjects who have a family history of pancreatic cancer (PC), or known genetic syndromes associated with increased risk of pancreatic cancer, will be followed for five years. This data will be used to determine the pancreatic cancer and precancerous lesion detection rate in High Risk Individuals (HRIs). Subjects may agree to annual imaging and annual biomarkers or to biomarkers only.
MP-101 will be evaluated in this study to see if it is safe and tolerable.
MP-101 will be evaluated in this study to see if it is safe, tolerable, and can help people with Short Bowel Syndrome. This study will also find out if taking MP-101 can improve the symptoms of Short Bowel Syndrome and reduce the number of times subjects experience bowel movements.
This is an open-label study of SNC-102 (acamprosate calcium sustained release tablet) in adult subjects with Tourette Syndrome. Subjects will be treated with oral doses of SNC-102 800 mg on a BID basis - before breakfast and at bedtime - for 4 weeks and the same subjects will be treated with SNC-102 1600mg in the morning and 800mg in the evening for an additional 4 weeks. Subjects will be assessed for changes in tic severity, safety, and pharmacokinetics. The study hypothesis is that treatment with SNC-102 will improve the tic severity in adult subjects with Tourette Syndrome.
Despite improvements in outcomes after Hematopoietic Cell Transplantation (HCT) for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), the risk of relapse remains high and is the most common cause of mortality after HCT. Moreover, treatment options for relapse after HCT are limited. Strategies to reduce relapse with maintenance therapy in patients who are at high risk are needed to improve survival. 5-aza is a hypomethylating agent that has shown immune modulating properties that may enhance the graft-versus-leukemia (GVL) effect, including upregulation of tumor-associated antigen and costimulatory molecule expression. Moreover, 5-aza has properties that suggest protection against graft-versus-host disease (GVHD) as well. Preliminary data shows that it is well tolerated and effective in clinical use for the treatment of AML or MDS relapse after HCT, as well as for maintenance therapy. This study will evaluate the use of 5-aza for maintenance after HCT in patients with AML or MDS with risk factors that are associated with a high risk for relapse.
Hypothesis: Myofascial temporomandibular joint disorder and related symptoms are associated with mandibular condyle and temporal fossa overloading secondary to temporalis and masseter muscle hyperactivity and spasm This study will examine the use of onabotulinumtoxinA (Botox) to treat myofascial pain disorder in patients with bruxism who frequently exhibit signs of temporomandibular joint disorder (TMD) in a double blind cross-over randomized clinical trial by injecting 50 units Botox in temporalis and masseter muscles in 32 patients with 6 months follow up. The objective of the proposed study will be to establish the safety and efficacy of botox in treating TMD associated with bruxism. By the time patients reach the specialists office, most have failed maximal conservative therapies, including non-chew diet, night guard, oral analgesics, muscle relaxants, physical therapy, and a portion have even undergone more invasive procedures, such as manipulation under anesthesia, arthroscopy, and arthroplasty surgeries with limited improvement or recurrence. Primary outcome of the study will be 50% reduction in pain. Secondary outcomes will be 50% reduction in surgical therapy, 25% increase in maximal inter-incisal opening (MIO). The investigators look forward to working with you on the proposed study
The purpose of this study is to investigate the effect on the frequency of tonic-clonic and clonic seizures of clobazam as adjunctive therapy compared to placebo after 16 weeks of treatment in paediatric patients aged ≥1 to ≤16 years with Dravet Syndrome.
The primary objective of the study is to determine whether Butrans Transdermal System (BTDS) reduces RLS symptom severity in patients with moderate to severe idiopathic RLS who are naïve to opiate treatment. The secondary objective of the study is to investigate the effects of BTDS on mood, sleep, and quality of life. The study will consist of nine visits. Depending on the need for medication titration, there may also be two scheduled telephone contacts. Visit 1: This is a screening visit to determine study eligibility. Eligible subjects who choose to participate must undergo medication washout as described in the detailed protocol between visits 1 and 2. Treatment period #1 (Visits 2 - 5; day 0 - 28): Baseline measures will be recorded and subjects randomized to treatment order at visit 2 (day 0). Study medication as well as rescue medication (l-dopa, a non-blinded active treatment to be used within a limited dose range as described in the detailed protocol) will be dispensed. Subjects will begin treatment period #1 immediately after this. The study medication will be titrated within the allowed range according to subject's reported symptoms during visit 3 (day 7), visit 4 (day 14), telephone contact (day 21). Visit 5 will occur on day 28 and will include assessment of outcome measures for the first treatment period. Visit 5 will also mark the beginning of the second treatment period. Treatment period #2 (Visits 6 - 8; day 28 - 56): Procedures will be similar to those described above during treatment period #1. Visit 8 will mark the end of the second treatment period during which outcome measures will be ascertained. Follow up visit (Visit 9; day 70): This will be a safety follow-up visit approximately two weeks after visit 8 for review of adverse events.
The Boston Keratoprosthesis type I (KPro) is a prosthetic cornea used to treat several causes of corneal blindness. Some categories of patients, including those with auto-immune diseases such as Stevens-Johnson syndrome, toxic epidermal necrolysis syndrome and mucous membrane pemphigoid, have a higher risk of failure for the KPro. Because of chronic inflammation, the cornea supporting the KPro may melt, leading to a higher risk of infection, loss of the KPro and loss of the eye. Infliximab is an antibody against tumor necrosis factor alpha and is used intravenously to control inflammation in several diseases. It has been used in some cases of corneal melting with significant success. This study's hypothesis is that infliximab can be successfully used as an eye drop (instead of the usual administration through veins) and that its regular use may prevent melt in eyes with a Boston Keratoprosthesis type I and underlying auto-immune disease.