Clinical trials: Mental Retardation
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Depressive Disorder, Major
A Study of GW856553X For the Treatment of Depression
GW856553 is a novel compound, currently in development for the treatment of Major Depressive Disorder (MDD), and other indications.
- Trial name
- NCT00569062
- Testing
- GW856553X
- Where
- 15 sites in Estonia, India and 1 more
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Fragile X Syndrome
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
- Trial name
- NCT01482143
- Testing
- AFQ056
- Where
- 4 sites in United States, Spain
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
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.
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, Inc.
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 11 To 17
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of the cognitive dysfunction shown by children with Down syndrome, aged 11 to 17.
- Trial name
- NCT00754052
- Testing
- Aricept (donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.
Down Syndrome
A Study of ACI-24 in Adults With Down Syndrome
This study is a prospective multicenter, placebo-controlled, double-blind, randomized study to assess the effect of one dose ACI-24 versus placebo over a 74-week treatment period and 26-week safety follow-up period.
- Trial name
- NCT04373616
- Testing
- ACI-24
- Sponsor
- AC Immune SA
Fragile X Syndrome
Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001
The subjects that meet the inclusion and exclusion criteria and consent to participate in protocol 22001 will be offered participation in 22001A which will evaluate secreted protein before and after treatment with STX209 and…
- Trial name
- NCT00823368
- Testing
- STX209
- Where
- 11 sites in United States
- Sponsor
- Seaside Therapeutics, Inc.
Rett Syndrome
Analysis of the Glutathione Cycle in Children With Rett Syndrome
Analyis of the Glutathione Cycle in Children with Rett Syndrome
- Trial name
- NCT02360436
- Where
- United States
- Sponsor
- Edison Pharmaceuticals Inc
Down Syndrome, Fetal Aneuploidy
Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy
The specimen collection is designed for the purpose of the development of a noninvasive prenatal test for T21.
- Trial name
- NCT01429389
- Where
- 14 sites in United States, Canada
- Volunteers
- 2,000 people
- Sponsor
- Sequenom, Inc.
Aggressive Behavior in Fragile X Syndome
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
The goal of this clinical trial is to learn if drug SRX246 works to treat irritability, agitation, aggression and self-injury (IAAS) behaviors in adult males with Fragile X Syndrome (FXS).
- Trial name
- NCT07328529
- Testing
- SRX246
- Where
- 3 sites in United States
- Volunteers
- 80 people
- Sponsor
- Azevan Pharmaceuticals
Prader-Willi Syndrome
A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.
- Trial name
- NCT03440814
- Testing
- DCCR
- Where
- 29 sites in United States, United Kingdom
- Volunteers
- 127 people
- Sponsor
- Soleno Therapeutics, Inc.
Adrenomyeloneuropathy, Adrenoleukodystrophy
MD1003-AMN MD1003 in Adrenomyeloneuropathy
The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
- Trial name
- NCT02961803
- Testing
- MD1003 100 mg capsule
- Where
- 4 sites in France, Germany and 1 more
- Volunteers
- 67 people
- Sponsor
- MedDay Pharmaceuticals SA
Mucopolysaccharidosis II
Mucopolysaccharidosis Type II Natural History
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in…
- Trial name
- NCT03529786
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 36 people
- Sponsor
- REGENXBIO 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.
Down Syndrome (Trisomy 21)
AEF0217 in Participants With Down Syndrome
The goal of this clinical trial is to identify if AEF0217 show an improvement in adaptive behaviors (daily life activities) in adults and older adolescents with Down Syndrome. It will also learn about the safety of AEF0217.
- Trial name
- NCT07334912
- Testing
- AEF0217 100 µg, AEF0217 300 µg
- Where
- 10 sites in France, Italy and 1 more
- Volunteers
- 188 people
- Sponsor
- Aelis Farma
Cerebral Adrenoleukodystrophy (CALD)
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major…
- Trial name
- Stargazer (NCT06224413)
- Testing
- No Intervention
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Genetix Biotherapeutics Inc.
Down Syndrome, Adaptive Behavior
Life Skills Improved in Children With Down Syndrome After Using Assistive Technology
Enhancing independence and quality of life are key modifiable outcomes that are short- and long-term goals for children with Down syndrome (DS) and for their parents.
- Conditions
- Down Syndrome, Adaptive Behavior, Quality of Life
- Trial name
- NCT05343468
- Testing
- The MapHabit System
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- MapHabit, Inc.
Trisomy 13, Trisomy 18
High Risk Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our proprietary algorithm method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278874
- Where
- 4 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Down Syndrome
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome.
- Trial name
- NCT02024789
- Testing
- RG1662
- Where
- 37 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Hoffmann-La Roche