Clinical trials: Intellectual Disabilities
Depression, Anxiety
BEhavioral Health Stratified Treatment (BEST) Study for Youth With Intellectual and/or Developmental Disabilities (IDD)
Many youth with disabilities and their families receive "care coordination services" from a state Maternal and Child Health Bureau (MCHB) agency.
- Conditions
- Depression, Anxiety, Transition
- Trial name
- BEST (NCT05520983)
- Testing
- Adapted Teens Achieving Mastery over Stress (TEAMS) Treatment, Adapted Teens Achieving Mastery over Stress (TEAMS) Prevention, Adapted Competent Adulthood Transition with Cognitive-behavioral & Interpersonal Training (CATCH-IT)
- Where
- United States
- Volunteers
- 780 people, healthy volunteers welcome
- Sponsor
- University of Illinois Chicago
Creatine Deficiency, X-linked
Observational Study of Males With Creatine Transporter Deficiency
The objectives of this study are to illustrate the clinical, neuro/electrophysiologic, biochemical, and developmental status and progression of patients with Creatine Transporter Deficiency (CTD) and to evaluate the utility of…
- Trial name
- Vigilan (NCT02931682)
- Where
- 10 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Down Syndrome, Subclinical Hypothyroidism
Treatment Trial of Subclinical Hypothyroidism in Down Syndrome
The purpose of this research study is to learn about the effects of treating subclinical hypothyroidism (SCH) with thyroid hormone replacement in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01832753
- Testing
- Levothyroxine
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Down Syndrome, Turner Syndrome
Hematological Profile in Children With Numerical Chromosomal Disorders
This cross-sectional study aims to evaluate the hematological profile and determine the frequency and pattern of hematological abnormalities in children with numerical chromosomal disorders attending the Pediatric Department of…
- Conditions
- Down Syndrome, Turner Syndrome, Klinefelter Syndrome, Trisomy 18
- Trial name
- NCT07807540
- Volunteers
- 158 people
- Sponsor
- Assiut University
Down Syndrome, Trisomy 21
Executive Function Play Opportunities for Children With Down Syndrome
The Executive Function Play Opportunities (EXPO) study is a sequential, multiple assignment, randomized trial (SMART) evaluating adaptive caregiver-mediated intervention pathways for improving executive function (EF) in children…
- Trial name
- EXPO (NCT07792382)
- Testing
- EXPO, EXPO Extended, EXPO Foundations
- Where
- 2 sites in United States, Italy
- Volunteers
- 150 people
- Sponsor
- Colorado State University
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Down Syndrome
Weight Bearing Exercises With and Without Jaffrey's Core Stability Exercise Training in Children With Down Syndrome.
Down syndrome also known as trisomy 21, is a genetic disorder caused by the presence of all or part of a third copy of chromosome 21.
- Trial name
- NCT05719662
- Testing
- Weight bearing exercises, Jaffrey's core stability exercises
- Where
- Pakistan
- Volunteers
- 18 people
- Sponsor
- Riphah International University
Prader Willi Syndrome, Obesity
Correlation of Hyperghrelinemia With Carotid Artery Intima-Media Thickness in Children With Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a genetic disorder characterized by childhood-onset obesity and endocrine dysfunction that leads to cardiovascular disability and early death within the first 3 decades of life.To assess the…
- Trial name
- NCT00474643
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Samsung Medical Center
Prader-Willi Syndrome
An Open-Label Study of DCCR Tablet in Patients With PWS
The primary objective of this study is to monitor the long-term safety of DCCR in PWS patients.
- Trial name
- NCT04086810
- Testing
- DCCR
- Sponsor
- Soleno Therapeutics, Inc.
Mucopolysaccharidosis II
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.
- Trial name
- NCT04573023
- Testing
- JR-141, Idursulfase, JR-141 or Idursulfase
- Where
- 26 sites in United States, Argentina and 10 more
- Volunteers
- 86 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Rett Syndrome
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett Syndrome in adolescent and adult females.
- Trial name
- NCT01703533
- Testing
- NNZ-2566
- Where
- 3 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neuren Pharmaceuticals Limited
Intellectual Disability, Healthy Subjects
Bladder and Bowel Functions, Participation and Quality of Life in Children With Intellectual Disabilities
Many neurodevelopmental, psychiatric, and medical disorders are commonly associated with intellectual disability. The presence of neurodevelopmental and psychiatric (NDP) comorbidities has been reported to negatively impact the…
- Trial name
- NCT07059858
- Where
- Turkey (Türkiye)
- Volunteers
- 102 people
- Sponsor
- Abant Izzet Baysal University
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below, to collect the safety information of JR-141 to evaluate the plasma pharmacokinetics of JR-141 to explore the efficacy of JR-141 on MPS…
- Trial name
- NCT03128593
- Testing
- JR-141
- Where
- 8 sites in Japan
- Volunteers
- 14 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Osteopenia, Spinal Muscular Atrophy
Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Children with neuromuscular disabilities and limited ambulation are at significant risk for decreased bone mineral density (BMD) and increased incidence of fracture.
- Conditions
- Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy
- Trial name
- NCT02428673
- Testing
- Assisted Standing Treatment Program
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Gillette Children's Specialty Healthcare
X-linked Mental Retardation
Clinical Phenotyping and Characterization of Neural Networks and Cognitive Processes Involved in Mental Retardation X-linked
X-linked Mental retardation (XLMR) represent 10% of the causes of mental retardation with a prevalence in both sexes around 1/296, i.e. 3.3 / 1000 births (Opitz et al., 1986).
- Trial name
- XLMR (NCT02854956)
- Testing
- Neuropsychological, cognitive and behavioral assessment
- Where
- France
- Volunteers
- 573 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Organic, Including Symptomatic, Mental Disorders, Substance Use
Exploring the Comorbidity Between Mental Disorders and General Medical Conditions
Mental disorders have been shown to be associated with a number of general medical conditions (also referred to as somatic or physical conditions).
- Conditions
- Organic, Including Symptomatic, Mental Disorders, Substance Use, Schizophrenia, Mood [Affective] Disorders
- Trial name
- COMO-GMC (NCT03847753)
- Testing
- Organic Disorders, Substance Use Disorders, Schizophrenia Disorders
- Volunteers
- 5,940,299 people
- Sponsor
- University of Aarhus
Down Syndrome
Role of Sleep Apnea in the Neuropsychological Function in Down Syndrome People
This is the first study about Neuropsychological function and OSAS in Taiwan. Although the correlation between OSAS and neuropsychological abnormalities had been reported, it is worth to understand more about the detailed domains…
- Trial name
- NCT03267602
- Testing
- CPAP
- Volunteers
- 45 people
- Sponsor
- National Taiwan University Hospital
Developmental Delay Disorder, Intellectual Disability
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in…
- Conditions
- Developmental Delay Disorder, Intellectual Disability, Rare Diseases
- Trial name
- NCT06860672
- Testing
- Dual vector DNA base editor
- Where
- China
- Volunteers
- 1 people
- Sponsor
- Yongguo Yu
Down Syndrome
Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy
Background: Down syndrome is a genetic disorder that can cause heart defects and other problems in the body. People with Down syndrome are more likely to have infections, autoimmunity, and blood diseases.
- Trial name
- NCT07416201
- Where
- United States
- Volunteers
- 700 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)