Down Syndrome Clinical Trial
Official title:
Mechanistic Investigation of Therapies for Down Syndrome Regression Disorder
Individuals with Down syndrome (DS) have an increased risk of numerous co-occurring conditions, including the neuropsychiatric condition known as Down Syndrome Regression Disorder (DSRD). A DSRD diagnosis often includes a sub-acute onset of catatonia, mutism, depersonalization, loss of ability to perform activities of daily living, hallucinations, delusions, and aggression and is most commonly observed in adolescents and young adults. The study evaluates the safety and efficacy of three currently prescribed therapies: lorazepam, intravenous immunoglobulin (IVIG) and tofacitinib.
Recent published case reports and clinical experience of the investigators indicate Down Syndrome Regression Disorder (DSRD) may be successfully treated with immune-modulating therapies, in addition to current pharmacologic options. This study is a multidimensional clinical trial designed to advance the understanding of the etiology of DSRD and to evaluate the safety and efficacy of three distinct therapeutic approaches to treating DSRD: (1) the benzodiazepine lorazepam (Ativan™) (2) intravenous immunoglobulin (IVIG, Gammagard™) or (3) the JAK inhibitor tofacitinib (Xeljanz™). Participants will be randomized into one of the three treatment arms above for the 12-week study period, with a subset of participants undergoing an initial 12-week observational period. Specific Aims: 1. To define the relative safety profile of lorazepam, IVIG, and tofacitinib in DSRD. 2. To compare the efficacy of lorazepam, IVIG, and tofacitinib in DSRD. 3. To investigate potential mechanisms underlying DSRD and its response to therapies. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT04854122 -
Blood Flow Regulation in Individuals With Down Syndrome - Training Study
|
N/A | |
| Completed |
NCT04020302 -
Self-Monitoring Shopping Intervention
|
N/A | |
| Recruiting |
NCT01950624 -
DS-Connect {TM}: The Down Syndrome Registry
|
||
| Completed |
NCT04751136 -
the Effect of Cerebrolysin on Physical and Mental Functions of Down Syndrome
|
Phase 2 | |
| Completed |
NCT04767412 -
Inspiratory Muscle Training and Physical Fitness in Children With Down Syndrome Randomized Control Trial
|
N/A | |
| Completed |
NCT04536506 -
Bobath and Vojta Therapy for DS
|
N/A | |
| Not yet recruiting |
NCT04037579 -
Protocol for a Non-randomized Survey in Down Syndrome People Who Practice Sports. Self and Observers´ Perception.
|
||
| Completed |
NCT02882698 -
Performance Analysis in Down Syndrome on Mobile Phone
|
N/A | |
| Completed |
NCT01791725 -
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
|
Phase 2 | |
| Unknown status |
NCT01975545 -
Fluor Varnish With Silver Nanoparticles for Dental Remineralization in Patients With Trisomy 21
|
Phase 2 | |
| Completed |
NCT01808508 -
Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome
|
N/A | |
| Terminated |
NCT00754052 -
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
|
Phase 3 | |
| Terminated |
NCT00754013 -
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
|
Phase 3 | |
| Completed |
NCT01313325 -
Hippotherapy to Improve the Balance of Children With Movement Disorders
|
N/A | |
| Completed |
NCT01256112 -
Parent Supported Weight Reduction in Down Syndrome
|
N/A | |
| Completed |
NCT01594346 -
Multicenter Vitamin E Trial in Aging Persons With Down Syndrome
|
Phase 3 | |
| Completed |
NCT05343468 -
Life Skills Improved in Children With Down Syndrome After Using Assistive Technology
|
N/A | |
| Suspended |
NCT05755464 -
Evaluation of Patients With Down Syndrome Compliance to Dental Therapy
|
||
| Recruiting |
NCT04022460 -
Using Personal Mobile Technology to Identify Obstructive Sleep Apnea in Children With Down Syndrome (UPLOAD)
|
||
| Completed |
NCT04818437 -
Effect of Core Stability Exercises and Balance Training in Postural Control Among Down Syndrome
|
N/A |