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CTAP101

Phase 3

Chronic Kidney Disease | Small molecule | Endocrine |Opko Health Inc.|Last Updated: Sep 29, 2016

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
RandomizedCONTROLLEDBiomarker
Total Trials2
Total Enrollment326
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02282813Extension Study of CTAP101-CL-3001 or CTAP101-CL-3002PHASE3 COMPLETED 298Apr 1, 2013May 1, 2015Sep 29, 2016 -
NCT00888069Pharmacokinetics and Safety Pilot Study of Single-Dose Oral and Intravenous CTAP101 in Stage 3 and 4 Chronic Kidney Disease SubjectsPHASE1 COMPLETED 28May 1, 2009Dec 1, 2009Sep 30, 201412 United States
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Study Endpoints
Primary Endpoints
Number of Participants in the Intent to Treat Population With a Mean Reduction in Plasma Intact Parathyroid Hormone (iPTH) of >/= 30% From Baseline Values at End of Treatment (EOT)
up to 6 months

Number of subjects in the intent to treat population with a mean reduction in plasma intact parathyroid hormone (iPTH) of \>/= 30% from pretreatment baseline values at end of treatment (EOT), classified as responders

To assess the PK and bioavailability of CTAP101 in Stage 3 and 4 CKD subjects with vitamin D insufficiency and SHPT
from 3 to 0 hours prior to dosing until 42 days post dosing
Secondary Endpoints
Number of Participants in the Per Protocol Population With Mean Reduction in Plasma Intact Parathyroid Hormone (iPTH) of >/= 30% From Baseline Values at End of Treatment (EOT)
up to 6 months
Number of Participants in the Intent to Treat Population With Normal Serum 25-hydroxyvitamin D at End of Treatment (EOT)
up to 6 months
Number of Participants in the Per Protocol Population With NormalSerum 25-hydroxyvitamin D at End of Treatment (EOT)
up to 6 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CTAP101 Capsules aloneEXPERIMENTALCTAP101 Capsules 30 or 60 mcg daily for up to 26 weeks
CTAP101 Capsules +calcitriolEXPERIMENTALCTAP101 Capsules 60 mcg +calcitriol daily for 14 weeks
CTAP101 Capsules +doxercalciferolEXPERIMENTALCTAP101 Capsules 60 mcg +doxercalciferol daily for 14 weeks
CTAP101 Capsules +paricalcitolEXPERIMENTALCTAP101 Capsules 60 mcg +paricalcitol daily for 14 weeks
Low Dose CTAP101 CapsulesEXPERIMENTALCTAP101 Capsules, 450 mcg dose
High Dose CTAP101 CapsulesEXPERIMENTALCTAP101 Capsules, 900 mcg dose
CTAP101 InjectionEXPERIMENTALIV injection, 448 mcg dose
Interventions
NameTypeDescription
CTAP101 CapsulesDRUGAt week 12, eligible subjects will continue to take 1-2 capsules (30 mcg each capsule) CTAP101 daily
CalcitriolDRUGAt week 12, eligible subjects will be randomized to also take calcitriol 0.25 mcg dose one capsule daily
DoxercalciferolDRUGAt week 12, eligible subjects will be randomized to also take doxercalciferol 0.5 mcg one capsule daily
ParicalcitolDRUGAt week 12, eligible subjects will be randomized to also take paricalcitol 1 mcg one capsule daily
CTAP101 InjectionDRUGsingle IV injection
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Completed either of the phase 3 double-blind, placebo-controlled studies, CTAP101-CL-3001 or CTAP101-CL-3002 * Taking no more than 1000 mg/day of elemental Ca and willing/able to remain on this dose for the duration of the study * Taking no more than 1600 IU/dose vitamin D (er...

Countries:United States
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