View clinical trials related to Disease.
Filter by:The primary objective of this study is to collect whole blood from patients diagnosed with Glycogen storage disease type 1B, which will be used to support the investigation of potential therapies that address the genetic basis of this disease.
The primary aim of this pilot study is to assess the feasibility, acceptability, and preliminary evidence of efficacy of a self-guided, cognitive behavioral therapy (CBT)-based mobile app intervention (SparkRx) for symptoms of depression among adolescents being treated in specialty medical care settings at Children's Hospital of Los Angeles (CHLA).
This trial is comprised of a 4-week randomized, double-blind, placebo-controlled treatment period followed by an optional 8-week open-label extension (OLE) period. This trial will evaluate the efficacy and safety of oral PRAX-114 flexibly dosed at 40 to 60 mg for 4 weeks compared to placebo in adults with PTSD. The OLE period consisting of treatment with 40 mg PRAX-114 for 8 weeks will provide additional efficacy and safety data.
The primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric participants with glycogen storage disease type III (GSD III).
The purpose of the study is to examine whether an investigational medication called ketamine along with psychotherapy is an effective treatment for depression in participants with a history of opioid addiction who have not abused opioids in at least 3 months. Participants will receive ketamine through intramuscular injection along with psychotherapy weekly for 8 weeks. Participation for eligible subjects who decide to enroll (including post-medication follow-up visits) will last about 16 weeks or 4 months.
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss.
The purpose of this study is to determine the interest of the use of EMDR (Eye Movement Desensitization and Reprocessing) psychotherapy in the management of psychotic disorders, in particular schizophrenic disorders.
Patients with Developmental Language Disorder (DLD) or Autism Spectrum Disorder (ASD) have communicational difficulties to adapt their language to context and to interlocutors. These difficulties have long term impacts on education and social life of these patients. Speech language therapists (SLT) helps child and teenagers with pragmatic and communicational disorders. Nevertheless, few research evaluated the efficacy of such interventions. In the present research, the students will do a literature review to identify efficacy intervention's strategies of pragmatic disorders. Then, 2 to 5 patients, aged from 8 to 14 years old, with ASD or DLD and pragmatic disorders, and who haven't intellectual disorder will be recruited. They will come to the faculty of psychology of UCLouvain before the beginning of the intervention. In this pretest session, parents will answer questionnaires et children will have tests in order to evaluate their pragmatic abilities (during 30 minutes), like conversations, role plays, communication referential tasks, etc. This evaluation will be video recorded to code pragmatic abilities. Then, a group intervention of ten sessions will be proposed (1 session per week, 10 weeks). Activities will include role plays, conversations, etc. At the end of the intervention period, a post test session, like the pretest session, will be proposed to evaluate intervention efficacy. The design is multiple study cases. This is the best design to control efficacy of these kind of interventions considering the inter-individual variability of DLD and ASD patients. This study is original since few research evaluated the efficiency of pragmatic intervention. Some studies demonstrated the efficacy of pragmatic intervention in DLD and ASD patients but theses researches were conducted in English-speaking countries. Furthermore, the present research will propose a group intervention, which weren't proposed in the literature to our knowledge (individual intervention). If the group intervention is efficacy, the cost-benefice ratio would be interested.
The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).
To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) who had participated in the primary study (XPF-009-301).