Clinical trials: Disabilities, Intellectual
Down Syndrome
Drumming Lessons' Influence on Children With Down Syndrome
The goal of this clinical trial is to learn if drumming lessons can increase self-control in children with Down syndrome. The main question it aims to answer is whether 2 months of drumming lessons can improve the behavioral…
- Trial name
- NCT07156318
- Testing
- 2 months of drumming lessons given by a professional drummer with extensive experience working with children with Down syndrome, Lessons after measurement
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt University
Smith-Magenis Syndrome (SMS)
Natural History Study of Smith-Magenis Syndrome
This study will examine how a rare disease called Smith-Magenis syndrome (SMS) affects people and how they change over time. SMS is caused by a small chromosome 17p11.2 deletion (missing piece).
- Trial name
- NCT00013559
- Where
- United States
- Volunteers
- 593 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Fragile X Syndrome
Trial of Sertraline to Treat Children With Fragile X Syndrome
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and…
- Trial name
- NCT01474746
- Testing
- Sertraline
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Randi J. Hagerman, MD
Prader-Willi Syndrome
Study of Emotion and Cognition Abilities of Children With PWS and Proposition of an Innovative Remediation
The present project project is divided in two parts. The primary aim of the part 1 of this study is to evaluate emotional control abilities of children with Prader-Willi syndrome (PWS) aged from 9 to 15 years and to study…
- Trial name
- PRACOM1 (NCT04526379)
- Testing
- Questionnaires, t-VNS
- Where
- France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Epilepsy, Intellectual Disability
Using Next-generation Sequencing in the Diagnosis of Epilepsy and/or Intellectual Disability in a Pediatric Cohorte
ABSTRACT Background and Aims: To determine the diagnostic performance of the epilepsy and intellectual disability panel used in the pediatric population, starting in June 2019, at the Regional University Hospital Center of Nancy…
- Trial name
- NCT05193890
- Testing
- retrospective analysis of a panel result
- Where
- France
- Volunteers
- 69 people
- Sponsor
- Central Hospital, Nancy, France
Hearing Loss, Mental Retardation
Postnatal HCMV Infection in Very Preterm Infants. Implications, Morbidity, Growth and Neurodevelopmental Outcomes.
The aim of this study is to investigate short and long term consequences from early postnatal HCMV infection transmitted via human milk in very preterm infants (birth weight < 1500 g or gestational age < 32 weeks).
- Conditions
- Hearing Loss, Mental Retardation, Microcephaly, Chorioretinitis
- Trial name
- NCT01151462
- Where
- Norway
- Sponsor
- Oslo University Hospital
Intellectual Disability, Caregiver Burden
Interactive Oral Health Educational Program for Children With Intellectual Disability and Their Caregivers
Conducting a research with an interactive oral health educational program and assessing knowledge of caregivers using a questionnaire will help in collecting information about the oral health educational program for children with…
- Trial name
- oral program (NCT06838221)
- Testing
- A Before and After study, before and after study
- Volunteers
- 30 people
- Sponsor
- Cairo University
Periodontitis, Trisomy 21
Periodontitis and Inflammation in Children With Down Syndrome/Trisomy 21: Study on Biological Samples
Since 2018, the Chicago Classification of Periodontal Diseases and Conditions, has listed Down syndrome (DS)/trisomy 21 (T21) as a systemic disease with periodontal implications.
- Trial name
- NT21 (NCT05970965)
- Testing
- Biological sampling
- Where
- France
- Volunteers
- 18 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Obesity, Over-weight
An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics for certain doses of beloranib in obese subjects with Prader-Willi Syndrome.
- Conditions
- Obesity, Over-weight, Prader-Willi Syndrome
- Trial name
- NCT01818921
- Testing
- ZGN-440 sterile diluent, 1.2 mg ZGN-440 for injectable suspension, 1.8 mg ZGN-440 for injectable suspension
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Zafgen, Inc.
Fragile X Syndrome
A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome
The purpose of this study is to determine the effectiveness and tolerability of aripiprazole in the treatment of children and adolescents with Fragile X Syndrome.
- Trial name
- NCT00420459
- Testing
- Aripiprazole
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Indiana University
Down Syndrome
Hippotherapy Versus Balance Therapy on Funvtional Mobility in Down Syndrome
Down Syndrome is a genetic condition caused by having an extra copy of chromosome 21 and 47 chromosomes instead of 46. A complete or partial extra copy of chromosome 21 is responsible for the anomaly and related structural and…
- Trial name
- NCT06502652
- Testing
- hippotherapy, balance training
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- Riphah International University
Down Syndrome
Promoting Health Through Parent Empowerment and the Activation of Routines (PrO-PEAR)
This study will examine the feasibility of the Promoting Health through Parent Empowerment and the Activation of Routines (Pro-PEAR) intervention using a open case-series for intervention optimization followed by a pilot RCT in…
- Trial name
- Pro-PEAR (NCT05020366)
- Testing
- Promoting Health through Parent Empowerment and the Activation of Routines, Enhanced Usual Care
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Pittsburgh
Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder
Motor Impairments in Children With Autism Spectrum Disorder: a Multimodal Approach
The general goal of the present proposal, Progetto MOSAICO, is the identification of a multimodal panel of neuropsychological, kinematic, neurophysiological, and genetic markers associated with motor abnormalities present in ASD.
- Conditions
- Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder, Intellectual Disability
- Trial name
- MOSAICO (NCT04769011)
- Testing
- Clinical evaluation, Neuropsychological evaluation, Neurophysiological evaluation
- Where
- Italy
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
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.
Prader-Willi Syndrome
A Study of Pitolisant in Patients With Prader-Willi Syndrome
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
- Trial name
- NCT06366464
- Testing
- Pitolisant tablet
- Where
- 57 sites in United States, Australia and 11 more
- Volunteers
- 134 people
- Sponsor
- Harmony Biosciences Management, Inc.
Rett Syndrome
An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome
Rett Syndrome (RTT) is a complex disorder resulting from mutations in a gene encoding the MeCP2 protein. Currently, there are no methods to fix the abnormal gene, however, animal studies suggest that the symptoms of RTT can be…
- Trial name
- NCT02562820
- Testing
- Intravenous infusion
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- The Cleveland Clinic
Autistic Disorder
Associative Memory in Autism Spectrum Disorder
The aim of this study is to investigate the neural substrates of associative memory in Autism Spectrum Disorder using both behavioural and EEG assessments.
- Trial name
- MEM-TA (NCT02265094)
- Testing
- Electroencephalography, Neuropsychological assessment
- Where
- France
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- University Hospital, Caen
Down Syndrome
Effects of Orofacial Therapy and Therapeutic Yoga in Children With Down Syndrome
The study aims to compare the effects of orofacial therapy and therapeutic yoga on swallowing, sleep habits, and quality of life in children with Down syndrome.
- Trial name
- NCT06352151
- Testing
- Routine Physiotherapy Group, Orofacial Treatment Group, Therapeutic Yoga Group
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hasan Kalyoncu University
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Down Syndrome
Proactive Speech and Language Intervention for Infants With Down Syndrome
Children with Down syndrome (DS) face life-long struggles with verbal communication. Babble and speech sound development is delayed, and speech can be difficult to understand.
- Trial name
- BBC-DS (NCT06450509)
- Testing
- Babble Boot Camp
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Arizona State University