Clinical trials: Disabilities, Intellectual
Fragile X Syndrome
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
This study is a multicenter, double-blind, randomized, placebo-controlled study to assess the safety and tolerability of 3 doses of MRM-3379 in male participants with Fragile X Syndrome ages 16 to 45 (inclusive).
- Trial name
- NCT07209462
- Testing
- Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379
- Where
- 18 sites in United States, Spain
- Volunteers
- 60 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Rett Syndrome
Open-Label Extension Study of Trofinetide for Rett Syndrome
To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome
- Trial name
- NCT04776746
- Testing
- trofinetide
- Where
- 21 sites in United States
- Volunteers
- 77 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Menkes Syndrome
Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome
It is a single-center, open, single-arm, non-randomized investigator-initiated trial evaluating the efficacy and safety of recombinant adeno-associated virus administration for patients with Menkes syndrome.
- Trial name
- NCT05507996
- Testing
- Recombinant adeno-associated virus administration
- Where
- China
- Volunteers
- 1 people
- Sponsor
- Kunming Hope of Health Hospital
Prader-Willi Syndrome
Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years
Positive results in preclinical and clinical studies in adults and infants with Prader-Willi syndrome lead investigators to set up a new study in children with Prader-Willi syndrome.
- Trial name
- Oxyjeune (NCT03114371)
- Testing
- Oxytocin
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Toulouse
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose…
- Conditions
- Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension
- Trial name
- POPS or POP02 (NCT04278404)
- Testing
- The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Duke University
Down Syndrome
Gait Parameters of Persons With Down Syndrome With and Without Orthotics Using GaitRite
This is a research study of persons with Down Syndrome between 2 and 80 years old who are independently walking. The purpose of this study is to see what effect orthotics have on how people with Down Syndrome walk.
- Trial name
- NCT01390558
- Testing
- custom orthotics
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Oregon Health and Science University
Lesch-Nyhan Disease
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam…
- Trial name
- NCT01065558
- Testing
- Ecopipam
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Psyadon Pharma
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Menkes Disease, Occipital Horn Syndrome
Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency
Menkes disease and occipital horn syndrome are two forms of copper deficiency that must be diagnosed and treated very early in life to prevent serious developmental problems.
- Conditions
- Menkes Disease, Occipital Horn Syndrome, Unexplained Copper Deficiency
- Trial name
- NCT00811785
- Testing
- Copper Histidine
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Cyprium Therapeutics, Inc.
Hepato-splenomegaly, Dysostosis Multiplex
Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)
Development of a new MS-based biomarker for the ear-ly and sensitive diagnosis of GM1/GM2 from blood
- Conditions
- Hepato-splenomegaly, Dysostosis Multiplex, Seizures, Mental Retardation
- Trial name
- BioGM1/BioGM2 (NCT02298647)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Dental Plaque, Dental Prophylaxis
Effectiveness of Customized Handle Toothbrush
People with special needs suffer from many difficulties, including mental, physical or motor, which increase the difficulty of obtaining good oral health.
- Conditions
- Dental Plaque, Dental Prophylaxis, Dental Care
- Trial name
- NCT04845633
- Testing
- Evaluation of the Effectiveness of Conventional Toothbrush in Dental Plaque Control in Healthy Children, Evaluation of the Effectiveness of Customized Handle Toothbrush in Dental Plaque Control in Healthy Children, Evaluation of the Effectiveness of Conventional Toothbrush in Dental Plaque Control in Children with Down Syndrome
- Where
- Syria
- Volunteers
- 48 people
- Sponsor
- Damascus University
Rett Syndrome
Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children. Rett syndrome is characterized by significant elevation in blood markers of oxidative stress.
- Trial name
- NCT01822249
- Testing
- EPI-743
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- Edison Pharmaceuticals Inc
Perinatal Condition, Neurodevelopmental Disorders
Perinatal and Psychological Correlates of Neurodevelopmental Disorders in Children.
The goal of this observational study is to investigate perinatal, cognitive and social-emotional correlates of neurodevelopmental disorders and educational difficulties in children and adolescents.
- Conditions
- Perinatal Condition, Neurodevelopmental Disorders, Social Functioning, Child Development
- Trial name
- NCT06215092
- Testing
- Psychological assessment
- Where
- Poland
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Bartosz M. Radtke
Down Syndrome, Cervical Spine Instability in Down Syndrome
Cervical Spine Abnormalities in Down Syndrome
The study aims to determine the prevalence,clinical presentations and radiographic cervical spine abnormalities associated Down syndrome in children at sohag university hospital
- Trial name
- NCT07260136
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Sohag University
Down Syndrome, Obstructive Sleep Apnea
Computer Models of Airways in Children and Young Adults With Sleep Apnea and Down Syndrome
The purpose of this research study is to develop a way of predicting with computers how surgery on the airway will affect night time breathing called Obstructive Sleep Apnea (OSA) in children with Down Syndrome.
- Trial name
- DYMOSA (NCT01902407)
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Disability, Intellectual
Importance of Multidimensional Assessments in Accompanying Schooling of Children With Mild to Moderate Intellectual Disability.
The EVAL'ID study aims to demonstrate the value of a multidimensional assessment in accompanying schooling of children with Intellectual Disability.
- Trial name
- EVAL'ID (NCT03088007)
- Testing
- Cognitive, adaptive, behavioral and academic assessment
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Hospices Civils de Lyon
Hunter Syndrome
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II) Patients
- Trial name
- NCT02044692
- Where
- South Korea
- Volunteers
- 34 people
- Sponsor
- Green Cross Corporation
Intellectual Developmental Disorder
Effectiveness of DIR/Floortime™ in Children With Intellectual Developmental Disorder
This randomized controlled trial aims to evaluate the effectiveness of Integrated Developmental, Individual-differences, Relationship-based (DIR)/Floortime™ intervention in improving functional emotional development and adaptive…
- Trial name
- DIR-IDD (NCT07758452)
- Testing
- DIR/Floortime™, Standard therapy
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- GIFT University
Psychopathology
Improving Mental Health Services for Economically Disadvantaged Children: Training Teachers
Children from low socioeconomic levels are more likely to have a mental disorder. However, they are less likely to receive appropriate treatment for that disorder than are children at higher socioeconomic levels.
- Trial name
- NCT00069563
- Testing
- Reaching Educators, Children, and Parents (RECAP)
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Down Syndrome
Application of LLM Care and Related Affective Computing Systems on Persons With Special Needs
Down syndrome (DS) is a genetic disorder characterized by specific physical characteristics (muscle degeneration) and cognitive phenotype (neurodegeneration caused by gene-overexpression that has affected memory, language, and…
- Trial name
- dsLLM (NCT04390321)
- Testing
- LLMcare
- Where
- Greece
- Volunteers
- 46 people
- Sponsor
- Aristotle University Of Thessaloniki