Clinical trials: Disabilities Multiple
Prader-Willi Syndrome
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin).
- Trial name
- CARE-PWS (NCT03649477)
- Testing
- 3.2 mg intranasal carbetocin, 9.6 mg intranasal carbetocin
- Where
- 24 sites in United States, Australia and 1 more
- Volunteers
- 130 people
- Sponsor
- Levo Therapeutics, Inc.
Down Syndrome, Alzheimer Disease
The Promotion of Physical Activity for the Prevention of Alzheimer's Disease in Adults With Down Syndrome
The objectives of this study are to determine the feasibility and potential efficacy of remotely delivered group exercise sessions to increase daily moderate to vigorous physical activity in adults with Down syndrome, relative to…
- Trial name
- NCT04048759
- Testing
- Remote Low, Remote High, Personal Coach
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- University of Kansas Medical Center
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
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Aneuploidy
National Prevalence and Impact of Noninvasive Prenatal Testing
Information on pregnant women undergoing non-invasive prenatal testing (NIPT) at one of the Obstetrix Medical Groups Outpatient Centers between January 2012 and June 2014 will be retrospectively gathered and an analysis of the…
- Trial name
- NCT02284399
- Testing
- non-invasive prenatal testing
- Where
- 7 sites in United States
- Volunteers
- 4,488 people, healthy volunteers welcome
- Sponsor
- Obstetrix Medical Group
Hunter Syndrome, Mucopolysaccharidosis II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Trial name
- NCT00607386
- Testing
- Idursulfase
- Where
- 3 sites in Brazil, Poland and 1 more
- Volunteers
- 28 people
- Sponsor
- Shire
Chronic Pain
Pain, Psychiatric Disorders, and Disability Among Veterans With and Without Polytrauma
We believe information to be obtained from this proposed study will prove critical for planning future VA healthcare strategies and developing effective and efficient treatments targeting pain and emotional adjustment…
- Trial name
- NCT00645970
- Where
- United States
- Volunteers
- 359 people, healthy volunteers welcome
- Sponsor
- US Department of Veterans Affairs
Hypofibrinogenemia, Afibrinogenemia
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
The goal of the study is to determine whether the use of Human Fibrinogen Concentrate (RiaSTAP) will decrease blood loss and the need for component blood therapy in neonates and infants undergoing cardiopulmonary bypass.
- Conditions
- Hypofibrinogenemia, Afibrinogenemia, Bleeding Disorders
- Trial name
- RiaSTAP (NCT02822599)
- Testing
- RiaStAP, Saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Acute Myeloid Leukemia, Down Syndrome
Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome
This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome.
- Conditions
- Acute Myeloid Leukemia, Down Syndrome, Myelodysplastic Syndrome, Myeloid Leukemia Associated With Down Syndrome
- Trial name
- NCT02521493
- Testing
- Asparaginase, Asparaginase Erwinia chrysanthemi, Cytarabine
- Where
- 191 sites in United States, Australia and 4 more
- Volunteers
- 280 people
- Sponsor
- Children's Oncology Group
Prader-Willi Syndrome
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.
- Trial name
- NCT04697381
- Testing
- somatropin - GH naïve pediatric cohort, somatropin - GH treated cohort, somatropin - adult cohort
- Where
- 5 sites in Japan
- Volunteers
- 33 people
- Sponsor
- Pfizer
Developmental Disabilities, Weight Loss
Weight Management for Adolescents With IDD
The goal of this study is to explore the impact of two different diets (conventional vs. enhanced stop light) and two different delivery systems (face-to-face vs.
- Conditions
- Developmental Disabilities, Weight Loss, Body Weight, Down Syndrome
- Trial name
- IDDAdol (NCT02561754)
- Testing
- Enhanced Stop Light Diet (eSLD), Conventional Diet (CD), Face-to-face (FTF)
- Where
- 2 sites in United States
- Volunteers
- 110 people
- Sponsor
- University of Kansas Medical Center
Down Syndrome, Obstructive Sleep Apnea
Investigating the Experience of Living With Down Syndrome and Obstructive Sleep Apnea Syndrome (Stage 1)
To identify the perceptions, beliefs, and family-relevant outcomes regarding the treatment of obstructive sleep apnea syndrome (OSAS) with positive airway pressure (PAP) in children with Down's Syndrome (DS).
- Trial name
- NCT04124471
- Testing
- Open-ended semi-structured interviews
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Children's Hospital of Philadelphia
Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma
Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma
This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute…
- Conditions
- Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma
- Trial name
- NCT01190930
- Testing
- Cyclophosphamide, Cytarabine, Dexamethasone
- Where
- 243 sites in United States, Australia and 5 more
- Volunteers
- 9,350 people
- Sponsor
- Children's Oncology Group
Prader-Willi Syndrome
Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)
The purpose of this study is to investigate over a 6 month period the effect of octreotide therapy on food intake, sense of hunger, body weight, body composition, efficiency of burning calories, biomarkers of weight regulation…
- Trial name
- NCT00399893
- Testing
- Octreotide
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Duke University
Cannabis Use Disorder, Multiple Sclerosis
A Teleheath tDCS Approach to Decrease Cannabis Use
The study aims to evaluate the effect of Dorsolateral Prefrontal Cortex (DLPFC) Transcranial Direct Current Stimulation (tDCS) in decreasing distress and cannabis use.
- Trial name
- NCT05005013
- Testing
- Transcranial Direct Current Stimulation (tDCS), Mindfulness
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- NYU Langone Health
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 6 To 10
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of cognitive dysfunction shown by children with Down syndrome, aged 6 to 10 years.
- Trial name
- NCT00754013
- Testing
- Aricept (Donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Eisai Inc.
Respiratory Syncytial Virus Infection
Synagis® Liquid 50mg, 100mg for Intramuscular Injection Special Investigation in Immunocompromised Children With Synagis®
This post marketing observational study (PMOS) was conducted in Japan during the 2013-2014 and 2014-2015 Respiratory Syncytial Virus (RSV) seasons to assess the safety and effectiveness of palivizumab for the prevention of…
- Trial name
- NCT02016690
- Volunteers
- 312 people
- Sponsor
- AbbVie
Down Syndrome
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
This is a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter study of the safety and PK of ELND005 administered orally for 4 weeks.
- Trial name
- DS201 (NCT01791725)
- Testing
- ELND005
- Where
- 3 sites in United States
- Volunteers
- 23 people
- Sponsor
- OPKO Health, Inc.
Prader-Willi Syndrome
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
- Trial name
- NCT05153434
- Testing
- ARD-101
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Down Syndrome, Postural Balance
Balance Bike Training in Down Syndrome
Background: Children with Down syndrome (DS) present with hypotonia, impaired balance and poor gross motor proficiency which lead to difficulty learning new motor skills, decreased engagement in physical activity, and limited…
- Trial name
- NCT03677245
- Testing
- Strider Balance Bike
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Mississippi Medical Center