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GSK1223249

Phase 1

Amyotrophic Lateral Sclerosis | Small molecule | Neurology |GSK plc|Last Updated: Sep 20, 2017

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment76
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00875446First Time in Human Study of GSK1223249 in Amyotrophic Lateral SclerosisPHASE1 COMPLETED 76 — — May 13, 2009Sep 9, 2011Sep 20, 201712 United States, France +2
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Study Endpoints
Primary Endpoints
Adverse events, vital signs, standard physical examination, ECG and standard clinical laboratory tests (haematology and biochemistry). Evaluation of safety will include any adverse effects on Immunogenicity, ALSFRS-R, SVC, Muscle Strength and MUNE.
12-16 Weeks
Secondary Endpoints
GSK1223249: Single dose PK (Part 1): AUC(0-∞), AUC(0-t), AUC(0-Week 4), %AUCex, Cmax, tlast, CL, Vss, MRT, λz and t½.Repeat dose PK (Part 2): GSK1223249 AUC(0-Week 4), Cmax and after the second dose, AUC(0-t), AUC(0-∞),λz and t½.
12-16 Weeks
Presence of antibodies to GSK1223249 will be assessed in serum samples using immuno-electrochemiluminescent assay.
At least 16 weeks
ALSFRS-R scores, Manual Muscle Strength test and Slow Inspirational Vital Capacity (SVC)
12-16Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Subjects receiving GSK1223249EXPERIMENTALEligible subjects will receive sequential dose of intravenous infusion of GSK1223249 with a starting dose of 0.01 milligram per kilogram followed by 0.1, 0.5, 1, 2.5, 5, 7.5, and 15 milligrams per kilograms.
Subjects receiving placeboPLACEBO_COMPARATOREligible subjects will receive intravenous infusion of placebo.
Interventions
NameTypeDescription
PLACEBODRUGPlacebo
GSK1223249DRUGI.V Infusion
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Patients with diagnosis of familial or sporadic ALS, defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the revised World Federation of Neurology El Escorial criteria \[Rix Brooks 2000\]. * Onset o...

Countries:United StatesFranceItalyUnited Kingdom
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