University of Minnesota Fragile X Clinic (Voyager Clinic)
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 7 Trials 
28 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Coffey, Barbara J
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
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
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
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
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
Fundora, Cynthia
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
Esler, Amy
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
Rambeck, Desirae
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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