University of Massachusetts
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 5 Trials 
8 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Berry-Kravis, Elizabeth
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
NCT05367960: An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Enrolling by invitation
3
300
US
Zatolmilast/ BPN14770
Tetra Discovery Partners
Fragile X Syndrome
09/27
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
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
REVEAL, NCT05606614: Safety and Efficacy of TSHA-102 in Adolescent and Adult Females with Rett Syndrome ( Adult Study)

Recruiting
1/2
18
Canada, US
TSHA-102
Taysha Gene Therapies, Inc.
Rett Syndrome
01/25
01/32
NCT06152237: Safety and Efficacy of TSHA-102 in Pediatric Females with Rett Syndrome (REVEAL Pediatric Study)

Recruiting
1/2
20
Europe, Canada, US
TSHA-102
Taysha Gene Therapies, Inc.
Rett Syndrome
11/28
11/31
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
NCT04476862: Cerliponase Alfa Observational Study in the US

Active, not recruiting
N/A
35
US
Cerliponase Alfa, Brineura, Administration Kit
BioMarin Pharmaceutical
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
08/30
08/30

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