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GR181413A/AT1001

Phase 1

Fabry Disease | Small molecule | Rare Disease |Amicus Therapeutics, Inc.|Last Updated: Dec 18, 2013

Success Probability

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Market & Valuation

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Trial Design

RandomizedPLACEBO_CONTROLLED
Total Trials1
Total Enrollment14

FDA Designations

No designations recorded

Clinical trial landscape

GR181413A/AT1001 · 1 trial · 1 indication

Phase 1 1
NCT01853852A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese SubjectsFabry Disease
COMPLETED14 Analytics
PHASE1COMPLETED
A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
Fabry Disease
NCT0185385214
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Study Endpoints

Primary Endpoints

Profile of plasma pharmacokinetics
0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose

AUC, Cmax, tmax, Tlast , t1/2, CL/F, Vz/F

Number of Participants with adverse events
up to 24 hr
Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
0, 24h post dose
Change from baseline in vital signs (blood pressure and heart rate)
0 ,1 , 2, 3, 4, 5, 6, 24h post dose
Change from baseline in 12-lead ECG
0, i, 2, 3, 6, 24h post dose
Profile of urine pharmacokinetics
0-4, 4-10, 10-12, 12-24h post dose

Ae, CLr, %Fx

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Study Design & Arms

AllocationRANDOMIZED
MaskingSINGLE
ModelCROSSOVER

Treatment Arms

ArmTypeDescription
50 mgEXPERIMENTALGR181413A/AT1001
150 mgEXPERIMENTALGR181413A/AT1001
450 mgEXPERIMENTALGR181413A/AT1001
PlaceboPLACEBO_COMPARATORplacebo

Interventions

NameTypeDescription
GR181413A/AT1001 solutionDRUGPowder for reconstitution
GR181413A/AT1001 capsuleDRUGSize 2, hard gelatin capsule, white opaque / blue opaque
Potable waterOTHERMatched, Size 2, hard gelatin capsule, white opaque/blue opaque
Placebo capsuleDRUGSolution matched
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Eligibility Criteria

Age Range20 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Healthy as determined by a responsible and experienced physician, based on a medical evaluation including medical history, physical examination, laboratory tests and cardiac monitoring. A subject with a clinical abnormality or laboratory parameters outside the reference range...

Countries:Australia
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Frequently asked questions about GR181413A/AT1001

What is GR181413A/AT1001 used for?

GR181413A/AT1001 is an investigational small molecule being developed for Fabry disease, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes GR181413A/AT1001?

GR181413A/AT1001 is being developed by Amicus Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol FOLD.

What phase is GR181413A/AT1001 in?

GR181413A/AT1001 is in Phase 1 clinical development. One Phase 1 trial has been completed, and the drug remains investigational, meaning it is not yet approved for any use.

What clinical trials is GR181413A/AT1001 in?

GR181413A/AT1001 has one completed Phase 1 trial, NCT01853852, which was a randomized, single-blind, four-period crossover, placebo-controlled, dose-escalation study. The trial evaluated the safety and pharmacokinetics of single oral doses in healthy Japanese subjects and enrolled 14 participants.

Is GR181413A/AT1001 the same as AT1001?

Yes, GR181413A/AT1001 is also known as AT1001. Both names refer to the same investigational small molecule being developed for Fabry disease.