
Motor Intervention Whit the Intorus Tool on Children With Down Syndrome
Down SyndromeThe goal of this clinical trial is to test in people with down syndrome how does the INTORUS tool influence their psychomotor development. The main question it aims to answer is: - Does the INTORUS tool improve psychomotor development in people with Down Syndrome? The intervention program will be carried out during the Functional Habilitation sessions and will be carried out by the center's professionals. These sessions must be carried out in a quiet environment, without distractions so that the user can concentrate on carrying out the activities. It is important that the user attends the sessions in comfortable, sleeveless clothing, to allow the tool to slide smoothly through their upper limbs. Each session will consist of several exercises with their corresponding rest breaks described in the Intervention Protocol. Each session attendance will be recorded following an attendance record model created for the occasion. Participants will be divided into two groups randomly. The control group will receive an intervention treatment at the psychomotor level in the traditional way, following the protocols established in the center, and the intervention group will receive an intervention treatment at the psychomotor level with the INTORUS device. Randomization will be carried out using the Oxford Minimization and Randomization software.

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic...
Progressive Familial Intrahepatic CholestasisAlagille Syndrome1 moreThis study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Efficacy of Suvorexant in Patients With Effectively Treated Restless Legs Syndrome and Persistent...
Restless Legs SyndromeInsomnia1 moreThe investigators aim to determine the effect of suvorexant on actigraphically-derived total sleep time in patients with effectively treated restless legs syndrome with persistent insomnia in a two-arm, double-blind, randomized placebo-controlled crossover 2.5-month trial.

Safety and Efficacy of Drug-Coated Balloon for De-novo Lesions in Patients With Acute Coronary Syndromes...
Drug-Coated BalloonDe Novo Stenosis5 moreThe DCB-ACS trial is a prospective, multi-center, non-inferiority, randomized controlled trail. The purpose of this trial is to evaluate the safety and efficacy of drug-coated balloon(DCB) in de novo lesions for acute coronary syndromes (ACS) .

Acupuncture-like Transcutaneous Electrical Nerve Stimulation on Bowel Symptom and Quality of Life...
Acupuncture-like Transcutaneous Electrical Nerve StimulationIrritable Bowel SyndromeTo discuss the effects of improving the irritable bowel syndrome patients' quality of life and defecation after implementing acupuncture-like Transcutaneous Electrical nerve stimulation.

Clinical and Cyto-histological Evaluation of Fractional CO2 Laser Treatment on Genito-Urinary Syndromes...
Genitourinary Syndrome of MenopauseEstrogen deficiency can occur naturally during menopause or as a secondary effect of various treatments for breast or pelvic cancer and can lead to very disabling vulvovaginal symptoms, since it is associated with an anatomical and functional cellular modification of the urogenital sphere. These changes result in urogenital atrophy responsible for vaginal dryness, painful intercourse (dyspareunia), discomfort, itching and burning sensations, dysuria, urgency and incontinence. These symptoms, which significantly affect quality of life, are found in more than 40% of menopausal women and are grouped under the term Genitourinary Syndrome of Menopause (GSM). General or local estrogen-based treatments improve patients' symptoms, but remain contraindicated in women who have had breast cancer. Non-estrogenic local treatments are less effective, remain restrictive and are therefore often abandoned. The CO2 laser is currently part of the therapeutic arsenal for the management of patients with GSM. This device prevents and eliminates the effects of low estrogen levels on vaginal tissue by restoring the characteristic conditions of the vaginal mucosa of a woman of childbearing age. This simple treatment, which lasts only a few minutes, is safe and painless and has no serious side effects. It restores the tone and elasticity of the tissues, with positive effects on the quality of life and the couple's relationship. The investigators wish to evaluate the possible changes of the genital sphere in a longitudinal way (before, during and after the treatment), including the induced cytohistological changes, in patients with GSM who can benefit of this therapy.

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Alagille SyndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

Physical Training in Patients With POTS After Covid-19
Covid19Post-acute Covid-19 Syndrome1 moreCovid-19 has the potential to affect physical, cognitive and psychological functions in multiple ways. It has been clear that a significant proportion of patients with Covid-19 develop long-term symptoms. The term post-acute Covid-19 syndrome (PACS) is now used to describe the wide range of prolonged symptoms following the infection. Patients may need specialized rehabilitation to be able to meet the complex symptoms and problems that may arise. A more specific syndrome that seems to occur more frequently than expected in the group of non-hospitalized patients who have had Covid-19 is the postural orthostatic tachycardia syndrome (POTS). To evaluate the effects of physical training in patients with POTS after Covid-19 a single subject design will be used (the patient is their own control). Inidividual semistructured interviews will be performed to explore and describe the patients´ experiences of the rehabilitation intervention. Participants: Patients diagnosed with POTS after Covid-19 (N=30) will be included. Procedure and outcomes: The primary outcomes are physical activity and health-related quality of life. Secondary outcomes are: physical capacity, active standing test, Malmö-POTS-questionnaire, Anxiety and depression, fatigue, self-reported outcome measure of physical function and work ability. Initially measurements will be performed several times during a period of 2-4 weeks to obtain a baseline before the intervention starts. Then the included participants will undergo a specially designed physical training program that will be performed 3 times /week during a period of 12 weeks. The intervention of physical training will consist of different exercises to enhance muscle strength and endurance. The intervention will be individually adjusted with a progression in dose, intensity, and position. The exercise is based on a program used in a previous study. Measurements will then be repeated after completion of the intervention period. A qualitative approach, with semistructured interviews, will be used to explore the patients´ experiences of the intervention, after commence of the interventional trial.

Is Medicinal Cannabis an Effective Treatment for Tourette Syndrome in Adolescents? A Pilot Study...
Tourette Syndrome in AdolescenceThis is a single site, pilot double-blind, randomized, placebo-controlled, cross-over study of 10 participants comparing medicinal cannabis (THC:CBD 10:15 oil) with placebo in reducing tics in adolescents aged 12 - 18 years with severe Tourette Syndrome (TS). The primary objective of this pilot study is to evaluate all elements of the study design (recruitment strategy, study duration, study procedures, study medication tolerance and outcome measures) to assess if they are acceptable and feasible for the conduct of a full-scale randomized controlled trial of THC:CBD 10:15 oil to reduce tic severity in adolescents with TS. The secondary objective of this study is to collect preliminary data on the safety of oral THC:CBD 10:15 oil in adolescents aged 12 to 18 years with TS. As an exploratory aim data from clinician- and parent-rated measures will be compared across the phases to explore for a signal of efficacy on primary (tic reduction) and secondary (premonitory urges, obsessive compulsive behaviors, Attention Deficit Hyperactivity Disorder [ADHD] symptoms) outcome measures.

An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
Phelan-McDermid SyndromeA study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.