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PTC596

Phase 1

Cancer | Small molecule | Oncology |PTC Therapeutics, Inc.|Last Updated: Dec 8, 2017

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment31

FDA Designations

No designations recorded

Clinical trial landscape

PTC596 · 1 trial · 1 indication

Phase 1 1
NCT02404480PTC596 in Patients With Advanced Solid TumorsCancer
COMPLETED31 Analytics
PHASE1COMPLETED
PTC596 in Patients With Advanced Solid Tumors
CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Dose-limiting toxicities
28 days

Determine the RP2D based on occurrence of DLTs and/or biological efficacy as determined by biomarker changes.

Secondary Endpoints

Adverse effects
28days
Time to Maximum Plasma Concentration (T max)
28days
Antitumor activity
28days
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1EXPERIMENTALPTC 596 administered twice daily- Dose level 0.65mg/kg
Cohort 2EXPERIMENTALPTC 596 administered twice daily-Dose level 1.3mg/kg
Cohort 3EXPERIMENTALPTC 596 administered twice daily-2.6mg/kg
Cohort 4EXPERIMENTALPTC 596 administered twice daily-Dose level 5.2mg/kg
Cohort 5EXPERIMENTALPTC 596 administered twice daily-Dose level 10mg/kg
Cohort 6EXPERIMENTALPTC 596 administered twice daily-Dose level 7mg/kg
Cohort 7 (Bio Marker cohort)EXPERIMENTALPTC 596 administered twice daily-Dose level 5.2mg/kg

Interventions

NameTypeDescription
PTC596DRUGPTC 596 will be administered orally twice a day until unmanageable toxicity, disease progression, or withdrawal of consent is noted
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Patients must have histologically or cytologically confirmed solid malignancy that is metastatic or unresectable, for which standard curative measures do not exist, that has progressed on at least one line of standard therapy or for which no standard therapies exists * Discont...

Countries:United StatesCanada
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Frequently asked questions about PTC596

What is PTC596 used for?

PTC596 is an investigational small molecule being developed for the treatment of cancer. It is being studied in patients with advanced solid tumors. The drug is currently in clinical development and has not been approved by regulatory authorities.

Who makes PTC596?

PTC596 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol PTCT. PTC Therapeutics is conducting clinical trials to evaluate the safety and efficacy of PTC596 in cancer patients.

What phase is PTC596 in?

PTC596 is in Phase 1 clinical development. A Phase 1 trial has been completed, which involved 31 patients with advanced solid tumors. The drug is still investigational and has not received regulatory approval for any indication.

What clinical trials is PTC596 in?

PTC596 has been studied in one clinical trial with the identifier NCT02404480, titled 'PTC596 in Patients With Advanced Solid Tumors'. This Phase 1 study was completed and enrolled 31 participants in the United States and Canada. The trial included adult patients aged 18 years and older.

Is PTC596 FDA approved?

PTC596 is not FDA approved. It is an investigational drug that has completed a Phase 1 clinical trial for advanced solid tumors. The drug remains in clinical development, and its safety and efficacy have not been established for regulatory approval.