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NCT ID: NCT05915910 Terminated - Clinical trials for Glycogen Storage Disease Type IB

Prospective Collection of Biospecimen in Pediatric Patients and Adult Guardians Diagnosed With Glycogen Storage Disease Type 1B (GSD1b)

Start date: April 27, 2023
Phase:
Study type: Observational

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.

NCT ID: NCT05461599 Terminated - Depression Clinical Trials

Digital Therapeutic for Adolescent Depressive Symptoms in Cardiology and Gastroenterology

Start date: August 25, 2022
Phase: N/A
Study type: Interventional

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).

NCT ID: NCT05260541 Terminated - Clinical trials for Post Traumatic Stress Disorder

A Clinical Trial of PRAX-114 in Participants With Post-Traumatic Stress Disorder

Start date: January 25, 2022
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT05196165 Terminated - Clinical trials for Glycogen Storage Disease Type III

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

Start date: May 20, 2022
Phase:
Study type: Observational

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).

NCT ID: NCT05176717 Terminated - Clinical trials for Retinitis Pigmentosa

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Start date: December 15, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

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.

NCT ID: NCT05164289 Terminated - Clinical trials for Schizophrenic Disorders

Interest of EMDR Schizophrenic Disorders

EMDRpsychose
Start date: December 10, 2021
Phase:
Study type: Observational [Patient Registry]

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.

NCT ID: NCT05127863 Terminated - Clinical trials for Autism Spectrum Disorder

Efficacy of a Pragmatic Intervention to Improve Adaptation to Context and Interlocutor

Start date: December 1, 2021
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT04990388 Terminated - Clinical trials for Glycogen Storage Disease Type III

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Start date: October 18, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).

NCT ID: NCT04912856 Terminated - Epilepsy Clinical Trials

An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE

EPIK-OLE
Start date: August 17, 2021
Phase: Phase 3
Study type: Interventional

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).

NCT ID: NCT04812106 Terminated - Clinical trials for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Start date: July 25, 2022
Phase:
Study type: Observational

The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or caregivers) to self-report information.