The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics
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 2 Trials 
25 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Phan, Han
RESPOND, NCT04488133 / 2020-003492-18: A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

Active, not recruiting
4
46
Europe, US, RoW
Nusinersen, ISIS 396443, BIIB058, Spinraza
Biogen
Muscular Atrophy, Spinal
10/25
10/25
VNS4PWS, NCT06144645: A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People with PWS

Recruiting
3
102
US
tVNS, intermittent stimulation, tVNS R, manufactured by tVNS Technologies, intermittent stimulation, tVNS, continuous stimulation, tVNS R, manufactured by tVNS Technologies, continuous stimulation
Foundation for Prader-Willi Research, RTI International
Prader-Willi Syndrome
12/26
12/27
RECONNECT, NCT04977986 / 2021-002542-33: Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults with Fragile X Syndrome

Recruiting
3
250
Europe, US, RoW
ZYN002 - transdermal gel, cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery), Placebo, Placebo Comparator, Matching Placebo
Zynerba Pharmaceuticals, Inc., Zynerba Pharmaceuticals, Inc.
Fragile X Syndrome
05/25
05/25
NCT06366464: A Study of Pitolisant in Patients with Prader-Willi Syndrome

Recruiting
3
134
US
Pitolisant tablet, pitolisant, Placebo tablet, placebo
Harmony Biosciences, LLC
Prader-Willi Syndrome
07/26
07/27
EBS-101-TD-391, NCT06021522: A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

Recruiting
3
150
Europe, Canada, US, RoW
Ecopipam, Ecopipam Hydrochloride
Emalex Biosciences Inc.
Tourette Syndrome
11/24
03/27
MIS51ON, NCT03992430 / 2018-001762-42: A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD)

Active, not recruiting
3
160
Europe, US, RoW
Eteplirsen, AVI-4658, EXONDYS 51, EXONDYS
Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne
10/26
10/26
HOPE-3, NCT05126758: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, not recruiting
3
104
US
Deramiocel (CAP-1002), Cardiosphere-Derived Cells (CDCs), Placebo
Capricor Inc.
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
12/25
12/27
D1AMOND, NCT05615220: Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

Active, not recruiting
3
196
Europe, Canada, US, RoW
Ecopipam Hydrochloride
Emalex Biosciences Inc.
Tourette Disorder
12/24
12/24
REBUILD-2, NCT04616326 / 2018-004622-28: A Study of Galcanezumab (LY2951742) in Participants 12 to 17 Years of Age With Chronic Migraine

Recruiting
3
300
Europe, Japan, US, RoW
Galcanezumab, LY2951742, Placebo
Eli Lilly and Company
Chronic Migraine
07/25
03/26
REBUILD, NCT03432286 / 2017-004351-23: A Study of Galcanezumab (LY2951742) in Participants 6 to 17 Years of Age With Episodic Migraine

Recruiting
3
325
Europe, Japan, US, RoW
Galcanezumab, LY2951742, Placebo
Eli Lilly and Company
Episodic Migraine
11/25
11/26
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Recruiting
3
45
Europe, Japan, US
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
Fortify, NCT05775848: Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

Active, not recruiting
3
81
Europe, US, RoW
BBP-418 (ribitol), Placebo
ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
07/27
07/27
NCT04336826 / 2020-000980-21: A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Completed
2
6
US
Ataluren, PTC124
PTC Therapeutics
Nonsene Mutation Duchenne Muscular Dystrophy
08/23
08/23
NCT04886518: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Hourglass Jul 2022 - Dec 2022 : Efficacy and safety data for treatment of EDS in patients with Myotonic Dystrophy Type 1
Active, not recruiting
2
30
Canada, US
Pitolisant Oral Tablet, Placebo oral tablet
Harmony Biosciences, LLC
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
10/23
10/24
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
PWS-001, NCT05879614: An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome

Withdrawn
2
20
US
NNZ-2591, Cyclo-L-Glycyl-L-2-Allylproline
Neuren Pharmaceuticals Limited
Prader-Willi Syndrome
06/25
06/25
NCT06332807: AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

Recruiting
1/2
12
US
NGGT002
NGGT INC.
Phenylketonurias
12/30
12/30
DELIVER, NCT05524883 / 2021-005478-24: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Recruiting
1/2
88
Europe, Canada, US, RoW
DYNE-251, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Duchenne Muscular Dystrophy (DMD)
11/29
11/29
CDLink, NCT04972604: CureDuchenne Link®: A Resource for Research

Recruiting
N/A
5000
US
CureDuchenne
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
07/31
07/31
NCT05683379: AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD

Recruiting
N/A
200
US
AAV8 DetectCDx
REGENXBIO Inc.
Duchenne Muscular Dystrophy
12/24
12/24
Lozano, Reymundo
NCT05358886: A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

Recruiting
3
150
US
BPN14770/ zatolmilast, Placebo
Tetra Discovery Partners
Fragile X Syndrome
06/25
09/25
RECONNECT, NCT04977986 / 2021-002542-33: Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults with Fragile X Syndrome

Recruiting
3
250
Europe, US, RoW
ZYN002 - transdermal gel, cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery), Placebo, Placebo Comparator, Matching Placebo
Zynerba Pharmaceuticals, Inc., Zynerba Pharmaceuticals, Inc.
Fragile X Syndrome
05/25
05/25
NCT05163808: A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

Recruiting
2/3
150
US
zatolmilast, BPN14770, Placebo
Tetra Discovery Partners
Fragile X Syndrome
09/25
12/25
NCT03718923: FOXP1 Syndrome: The Seaver Autism Center for Research and Treatment is Characterizing FOXP1-related Neurodevelopmental Disorders Using Genetic, Medical, and Neuropsychological Measures.

Recruiting
N/A
50
US
Icahn School of Medicine at Mount Sinai, The Seaver Foundation, Autism Science Foundation, Research Foundation for Mental Hygiene, Inc.
FOXP1, Mental Retardation With Language Impairment and With or Without Autistic Features, Autism Spectrum Disorder
04/26
04/26
Aarem, Anniek M Van
RECONNECT, NCT04977986 / 2021-002542-33: Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults with Fragile X Syndrome

Recruiting
3
250
Europe, US, RoW
ZYN002 - transdermal gel, cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery), Placebo, Placebo Comparator, Matching Placebo
Zynerba Pharmaceuticals, Inc., Zynerba Pharmaceuticals, Inc.
Fragile X Syndrome
05/25
05/25

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