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arbaclofen

Phase 3

Fragile X Syndrome | Small molecule | Rare Disease |cbdMD, Inc.|Last Updated: Jul 31, 2013

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment297

FDA Designations

No designations recorded

Clinical trial landscape

arbaclofen · 2 trials · 1 indication

Phase 3 2
NCT01325220Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X SyndromeFragile X Syndrome
COMPLETED172 Analytics
NCT01282268Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X SyndromeFragile X Syndrome
COMPLETED125 Analytics
PHASE3COMPLETED
Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Fragile X SyndromeUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome
Fragile X SyndromeUnlock trial analytics

Study Endpoints

Primary Endpoints

Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
at 8 weeks of treatment

This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal

Aberrant Behavior Checklist - FXS Social Avoidance Subscale
At 8 weeks of treatment
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
STX209 5 mg BIDACTIVE_COMPARATOR -
STX209 10 mg BIDACTIVE_COMPARATOR -
STX209 10 mg TIDACTIVE_COMPARATOR -
PlaceboPLACEBO_COMPARATOR -
ArbaclofenACTIVE_COMPARATOR -

Interventions

NameTypeDescription
arbaclofenDRUG5 mg bid
PlaceboDRUGtid
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Eligibility Criteria

Age Range5 Years to 11 Years
SexALL
Healthy VolunteersNo
Study Sites28

Inclusion Criteria: * Molecular documentation of the full FMR1 mutation * Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening. * Subjects with a history of seizure disorder must currently be receiving treatment with antiepileptics and must have been sei...

Countries:United States
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Frequently asked questions about arbaclofen

What is arbaclofen used for?

Arbaclofen is an investigational small molecule being developed for the treatment of Fragile X Syndrome, a rare genetic condition. It is currently in Phase 3 clinical development, meaning it has not yet been approved by regulatory authorities and remains under investigation for safety and efficacy in patients.

Who is developing arbaclofen?

Arbaclofen is being developed by cbdMD, Inc., a company publicly traded under the ticker symbol YCBD. The company is conducting clinical trials to evaluate the drug's potential as a treatment for Fragile X Syndrome, though the drug remains investigational and is not yet approved.

What phase is arbaclofen in?

Arbaclofen is in Phase 3 clinical development for Fragile X Syndrome. Two Phase 3 trials have been completed, with a total of 297 participants enrolled. The drug is still investigational and has not received FDA approval, as it remains under evaluation for safety and efficacy.

What clinical trials is arbaclofen in?

Arbaclofen has completed two Phase 3 clinical trials. NCT01282268 enrolled 125 adolescents and adults with Fragile X Syndrome, while NCT01325220 enrolled 172 children aged 5 years and older. Both trials were randomized, double-blind, and placebo-controlled, and both have been completed.

Is arbaclofen the same as STX209?

Yes, arbaclofen is also known as STX209. The clinical trials NCT01282268 and NCT01325220 both refer to the drug as STX209 (arbaclofen) in their titles, confirming that these names refer to the same investigational compound being studied for Fragile X Syndrome.

How does arbaclofen work?

Arbaclofen is a small molecule being studied for Fragile X Syndrome. The specific molecular target or mechanism of action has not been disclosed in the available clinical trial information, so its precise mode of action remains under investigation as part of ongoing research.