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ANAVEX2-73

Phase 3

Rett Syndrome | Small molecule | Rare Disease |Anavex Life Sciences Corp.|Last Updated: Aug 1, 2022

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment64
FDA Designations
FAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASE
Clinical trial landscape

ANAVEX2-73 · 5 trials · 3 indications

Phase 3 1Phase 2 4
NCT03941444ANAVEX2-73 Study in Patients With Rett SyndromeRett Syndrome
COMPLETED33 Analytics
PHASE3COMPLETED
ANAVEX2-73 Study in Patients With Rett Syndrome
Rett SyndromeUnlock trial analytics
Study Endpoints
Primary Endpoints
RSBQ
7 weeks

Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score

Incidence of Adverse Events
7 weeks
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
48 weeks

To continue assessing the safety and tolerability of ANAVEX2-73

Maximum Plasma Concentration [Cmax] of ANAVEX2-73
7 weeks

PK of ANAVEX2-73 and metabolite

Area Under the Curve [AUC] of ANAVEX2-73
7 weeks

PK of ANAVEX2-73 and metabolite

Lipid panel
7 weeks

Significant laboratory findings

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Up to 208 weeks

Safety

To determine maximum tolerated dose of Anavex2-73.
36 Days
Secondary Endpoints
CGI-I
7 weeks
Anxiety, Depression, and Mood Scale (ADAMS)
7 weeks
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
7 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Active armEXPERIMENTALANAVEX2-73 liquid oral solution
Placebo armPLACEBO_COMPARATORPlacebo liquid oral solution
ANAVEX2-73 ActiveEXPERIMENTALOral capsules
ANAVEX2-73 Oral as assigned in ANAVEX2-73-002EXPERIMENTAL -
Anavex2-73 oral then the Anavex2-73 intravenous formulationEXPERIMENTALParticipants first receive Anavex2-73 hard gelatin capsule each morning with a light breakfast for 11 days. After a washout period of 11 days they then receive the Anavex2-73 intravenous formulation each morning with a light breakfast for 11 days.
Anavex2-73 intravenous then the Anavex2-73 oral formulationEXPERIMENTALParticipants first receive Anavex2-73 intravenous formulation each morning with a light breakfast for 11 days. After a washout period of 11 days then they receive the Anavex2-73 hard gelatin capsule each morning with a light breakfast for 11 days.
Anavex2-73 30 mg oral formulationEXPERIMENTALParticipants will receive the 30 mg Anavex2-73 hard gelatin capsule orally once daily for 52 weeks.
Anavex2-73 50 mg oral formulationEXPERIMENTALParticipants will receive the 50 mg Anavex2-73 hard gelatin capsule orally once daily for 52 weeks.
Interventions
NameTypeDescription
ANAVEX2-73DRUGLiquid oral solution
PlaceboDRUGLiquid oral solution
ANAVEX2-73 OralDRUG -
ANAVEX2-73 IntravenousDRUG -
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Eligibility Criteria
Age Range18 Years to 45 Years
SexFEMALE
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: * Aged ≥ 18 years, inclusive. * Diagnosis of classic RTT, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation. * Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks. * If on antiepileptic drugs (AEDs), 1-4 AEDs ...

Countries:AustraliaUnited KingdomSpainUnited States
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