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AAT

Phase 2

Type 1 Diabetes | Small molecule | Metabolic |Kamada Ltd.|Last Updated: Jan 11, 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
NO_TREATMENT_CONTROLLEDBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01661192Long Term Treatment Effect of the Safety, Tolerability and Efficacy of AAT in Type 1 DiabetesPHASE2 COMPLETED 12Jan 1, 2013Jan 1, 2017Jan 11, 20172 Israel
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Study Endpoints
Primary Endpoints
Safety and tolerability of AAT in terms of adverse events and serious adverse events
At month 36

We will assess at each visit until final visit (month 36)the safety and tolerability of study drug in terms of adverse events and serious adverse events

Safety and tolerability of the AAT in terms of laboratory values
At month 36

We will assess at each visit until final visit (month 36)the safety and tolerability of study drug in terms of laboratory values

Secondary Endpoints
Beta cell function-AUC (Area Under the Curve) of stimulated C-Peptide from stimulated MMTT (mixed meal tolerance test)
at month 36
Percentage of patients that maintain stimulated peak C-peptide >=0.2 nmol/L
at month 36
Percentage of patients that achieve glycemic target of HbA1c <=7.5%
At month 36
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposePREVENTION
Treatment Arms
ArmTypeDescription
AAT( Alpha 1 Antitrypsin)EXPERIMENTALSubjects who maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L will continue treatment with AAT according to the dosage group they were allocated to at the previous study(40mg/kg or 60mg/kg or 80mg/kg), intravenously, once a week for 6 consecutive weeks, at 24-week intervals for a duration of \~54 weeks.
Follow up groupNO_INTERVENTIONSubjects who have not maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L or subjects with peak stimulated C -peptide secretion ≥ 0.2nmol/L who are reluctant to receive additional study drug, will be followed up only with no administration of investigational product
Interventions
NameTypeDescription
AAT( Alpha 1 Antitrypsin)DRUG -
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Eligibility Criteria
Age Range10 Years — 25 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Subject (or parent/guardian) willing and able to sign an informed consent * Ability to comply with all study requirements. * A patient that participated in Study 008 and received all doses of study medication, per protocol. * Evidence of clinically significant residual beta-ce...

Countries:Israel
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