Clinical trials: Intellectual Disabilities, open to volunteers
- Condition Intellectual Disabilities
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
Rett Syndrome
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
- Trial name
- NCT07503444
- Testing
- fenfluramine hydrochloride
- Where
- 40 sites in United States, Belgium and 10 more
- Volunteers
- 200 people
- Sponsor
- UCB BIOSCIENCES, 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.
Amyloid Plaque, Beta-Amyloid
A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease
- Trial name
- NCT05462106
- Testing
- ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a
- Where
- 26 sites in United States, Spain and 1 more
- Volunteers
- 304 people
- Sponsor
- AC Immune SA
Prader-Willi Syndrome
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS).
- Trial name
- NCT07266324
- Testing
- BMB-101
- Where
- 2 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Bright Minds Biosciences Pty Ltd
Rett Syndrome
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
The RETT REVOLUTION trial is a placebo-controlled, single-blinded, exploratory study with patients serving as their own control ("N of 1" trial design) where the safety and efficacy of vorinostat in the treatment of Rett syndrome…
- Trial name
- NCT07150013
- Testing
- Vorinostat (SAHA)
- Where
- Colombia
- Volunteers
- 15 people
- Sponsor
- Unravel Biosciences, Inc.
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Trial name
- NCT06430385
- Testing
- ION440
- Where
- 12 sites in United States, Austria and 3 more
- Volunteers
- 48 people
- Sponsor
- Ionis 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
Pregnant Individuals, Aneuploidy
The ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) Study is the Largest U.S.-Based Prospective Study Demonstrating a Circulating Fetal Cell (CFC) Based Approach to Non-invasive Fetal Risk Assessment
The goal of the ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) study is to compare the concordance of results of a novel non-invasive circulating fetal cell (CFC) assay to the results of…
- Conditions
- Pregnant Individuals, Aneuploidy, Down Syndrome (Trisomy 21), 22q11.2 Deletion Syndrome
- Trial name
- ADVANCE (NCT07643896)
- Where
- 9 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- BillionToOne Inc.
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.
ASD
Mental Imagery Therapy for Autism (MITA) - an Early Intervention Computerized Language Training Program for Children With Autism
Mental Imagery Therapy for Autism (MITA) is a highly innovative adaptive language therapy application for children with autism. MITA exercises are limitless in variations, therefore avoiding routinization.
- Trial name
- MITA-RCT (NCT07326423)
- Testing
- Mental Imagery Therapy for Autism (MITA), General executive function
- Where
- Brazil
- Volunteers
- 60 people
- Sponsor
- ImagiRation, LLC
Pediatric Obstructive Sleep Apnea, Down Syndrome (DS)
Pediatric Down Syndrome Post-Approval Study
The purpose of this Post Approval research study is to assess ongoing safety and effectiveness of the Inspire therapy in adolescents and young adults (age 13-18) with Down syndrome and severe sleep apnea.
- Trial name
- NCT06851338
- Testing
- Hypoglossal Nerve Stimulation
- Where
- 6 sites in United States
- Volunteers
- 60 people
- Sponsor
- Inspire Medical Systems, Inc.
MECP2 Duplication Syndrome
A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)
Methyl-CpG binding protein 2 (MECP2) is a dosage-sensitive, X-linked gene critical for central nervous system development and functional maintenance, which gain-of-function causes MECP2 duplication syndrome (MDS).
- Trial name
- HERO (NCT06615206)
- Testing
- HG204
- Where
- China
- Volunteers
- 6 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Prader-Willi Syndrome
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prospective observational study developed to generate more evidence of the safety profile, clinical characteristics and outcomes of patients with PWS treated with VYKAT XR.
- Trial name
- NCT07450664
- Testing
- VYKAT XR
- Where
- 25 sites in United States
- Volunteers
- 200 people
- Sponsor
- Soleno Therapeutics, Inc.
Fragile X Syndrome (FXS), Fragile X Syndrome
Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
- Trial name
- FXS-CTH120-01 (NCT07654114)
- Testing
- CTH120
- Where
- 2 sites in Spain
- Volunteers
- 30 people
- Sponsor
- Connecta Therapeutics, S.L.
Down Syndrome-Associated Alzheimer's Disease (DS-AD)
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran.
- Trial name
- NCT07636811
- Testing
- Mivelsiran
- Where
- 10 sites in United States, Spain
- Volunteers
- 58 people
- Sponsor
- Alnylam Pharmaceuticals
Intellectual Disability, Mild, Intellectual Disability, Mild to Moderate
PGx Medication Safety Reviews of Persons With IDD
The purpose of this study is to assess the feasibility of a comprehensive medication review and PGx initiative as part of the care process for individuals in the care of CP Unlimited.
- Conditions
- Intellectual Disability, Mild, Intellectual Disability, Mild to Moderate, Developmental Disability
- Trial name
- NCT07092852
- Testing
- Cheek swab for PGx testing, Medication safety review
- Where
- 2 sites in United States
- Volunteers
- 700 people
- Sponsor
- GalenusRx, Inc.
Mucopolysaccharidosis II
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects
An extension of Global Phase III open-label, multicenter designed to evaluate the Long-term safety and efficacy of study drug for the treatment of the MPS II.
- Trial name
- NCT05594992
- Testing
- JR-141
- Where
- 10 sites in United States, Argentina and 6 more
- Volunteers
- 80 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous…
- Trial name
- NCT06075537
- Testing
- tividenofusp alfa
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 99 people
- Sponsor
- Denali Therapeutics Inc.
RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design.
- Trial name
- TRANSCEND (NCT07257978)
- Testing
- NTI164
- Where
- Australia
- Volunteers
- 40 people
- Sponsor
- Fenix Innovation Group