View clinical trials related to Syndrome.
Filter by:Specific Aim 1: As shown in the literature, exercise can be a useful tool for treating and improving functional GI disorders. Therefore, the aim is to estimate the effectiveness of an exercise intervention on IBS symptoms as measured by a reduction of at least 50 score points on the IBS- Symptom Severity Scale (IBS-SSS) questionnaire.
To evaluate the efficacy and safety of two dosing schemes of oral masitinib versus matching placebo in the treatment of patients suffering from severe MCAS with handicap unresponsive to optimal symptomatic treatment.
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation. In total, up to 8 participants, with a minimum of 3 adults, will be enrolled in the trial. The study will be comprised of the following periods for each participant: - a Screening period of up to 6 weeks (42 days) - a total Treatment Period of 8 weeks (60 days) - a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.
This research study will determine whether orally administered ADX-629 is safe and has biochemical efficacy in patients with Sjögren-Larsson syndrome (SLS), a rare inherited disorder of fatty aldehyde metabolism The disease is caused by bi-allelic mutations in ALDH3A2, which results in deficient activity of fatty aldehyde dehydrogenase (FALDH) and leads to the build-up of harmful long-chain (C16-C20) aldehydes and alcohols. Accumulation of these lipids and their metabolic products in skin, brain and eyes is responsible for the symptoms, which persist lifelong. ADX-629 is an aldehyde trapping agent that is expected to eliminate fatty aldehydes and negate aldehyde toxicity, improve the biochemical abnormalities and have clinical efficacy for SLS. The primary objective of this clinical protocol is to determine whether ADX-629 is safe and tolerable for use in SLS subjects. The secondary objective is to determine the efficacy of ADX-629 in reversing the biochemical abnormalities in SLS. Exploratory objectives are to evaluate the short-term clinical effects of ADX-629 on neurologic, cutaneous and ophthalmologic disease in SLS. Patients will be treated with ADX-629 for 12 weeks and monitored for safety and biochemical efficacy.
The purpose of this study is to describe and compare the kinematic deficiencies specifically associated with each of the 3 main clinical phenotypes of patellofemoral pain syndrome. The prevalence of patellofemoral pain is high with a high rate of chronicity and recurrence and an overrepresentation of young, athletic and female populations. There are multiple classifications of patellofemoral pain syndrome. A pragmatic classification distinguishes 3 main clinical phenotypes of patellofemoral pain syndrome: with objectively displaceable patella, with extra-patellar alignment problems and without alignment problems. The pathophysiology of patellofemoral pain syndrome is multifactorial involving static and dynamic dysfunctions of the hip, knee and foot, which remain incompletely elucidated to date. The links between the clinical and biomechanical aspects are still unclear and the kinematic and neuromuscular deficiencies associated with the 3 main clinical phenotypes are poorly understood. A validated non-invasive device allows the 3D evaluation of femorotibial rotations during walking.
Patients suffering from facet-joint-syndrome associated with lower-back pain will be treated with MRI-guided High Intensity Focused Ultrasound thermal therapy for pain relief.
Inherited bone marrow failure syndromes (IBMFSs) are a diverse collection of genetic illnesses characterized by various degrees of peripheral cytopenias due to defective single-lineage or multi-lineage hematopoiesis, it can manifest itself at birth or later in life.
The primary objective is to determine possible implications of deviant tear film quality for optimal refractive precision in patients scheduled for cataract surgery. The study consist of three separate arms where cataract patients will be separated in to Dry Eye Disease (DED) positive and negative groups. The DED positive group will be further subdivided into two different treatment groups (with focus on prolonged use of lubricant and preservative free eye drops). Tear film samples will be taken before treatment, after treatment and after surgical treatment and compared.
Short bowel syndrome (SBS) is a rare but challenging condition in which patients have insufficient bowel length to meet fluid, electrolyte, and nutrient requirements without parenteral support. The purpose of this study is to determine how well dietary fiber is tolerated in patients with short bowel syndrome compared to patients without short bowel syndrome based on assessment of gastrointestinal symptoms, and corresponding changes in microbiome composition and metabolomics.
This is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.