PTC596 in Patients With Advanced Solid Tumors

NCT02404480 | Completed Phase 1

• 1 other identifier: PTC596-ONC-001-AST
• Study Type: interventional
• Target: 31
• Locations: 2 countries
• Sites: 4

Brief Summary

This is a Phase 1, open-label, first-in-human, safety and pharmacokinetic study of PTC596 in patients with advanced cancer.

Conditions

Cancer

Outcome Measures

Primary Outcomes (1)

• Dose-limiting toxicities

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

  28 days

Secondary Outcomes (7)

• Adverse effects

  28days

• Time to Maximum Plasma Concentration (T max)

  28days

• Antitumor activity

  28days

• Maximum Plasma Concentration (C max)

  28 days

• Plasma Concentration at 24 hours

  28days

Study Arms (7)

Cohort 1

EXPERIMENTAL

PTC 596 administered twice daily- Dose level 0.65mg/kg

Drug: PTC596

Cohort 2

EXPERIMENTAL

PTC 596 administered twice daily-Dose level 1.3mg/kg

Drug: PTC596

Cohort 3

EXPERIMENTAL

PTC 596 administered twice daily-2.6mg/kg

Drug: PTC596

Cohort 4

EXPERIMENTAL

PTC 596 administered twice daily-Dose level 5.2mg/kg

Drug: PTC596

Cohort 5

EXPERIMENTAL

PTC 596 administered twice daily-Dose level 10mg/kg

Drug: PTC596

Cohort 6

EXPERIMENTAL

PTC 596 administered twice daily-Dose level 7mg/kg

Drug: PTC596

Cohort 7 (Bio Marker cohort)

EXPERIMENTAL

PTC 596 administered twice daily-Dose level 5.2mg/kg

Drug: PTC596

Interventions

PTC596 DRUG

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

Cohort 1; Cohort 2; Cohort 3; Cohort 4; Cohort 5; Cohort 6; Cohort 7 (Bio Marker cohort)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Discontinuation of all other therapies (including other investigational drugs, radiotherapy, or chemotherapy) for the treatment of cancer ≥4 weeks (≥6 weeks if nitrosoureas, ≥12 weeks if radiotherapy) before initiation of study treatment
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
• Life expectancy of at least 3 months
• A measured or estimated creatinine clearance (CrCl) ≥60 mL/min/1.73 m2

You may not qualify if:

• Prior bone marrow/hematopoietic stem cell transplantation
• History of solid organ, bone marrow, or progenitor cell transplantation
• History of major surgical procedure within 28 days prior to start of study treatment
• Evidence of ongoing systemic bacterial, fungal, or viral infection. Known human immunodeficiency virus (HIV) infection or acquired-immunodeficiency syndrome (AIDS)-related illness
• Any of the following in the past 6 months: myocardial infarction, unstable angina, coronary/peripheral artery bypass graft, congestive heart failure (New York Heart Association Class III or IV), cerebrovascular accident, transient ischemic attack, other arterial thromboembolic event, or pulmonary embolism

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (4)

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Duke University

Durham, North Carolina, 27708, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M4Y2H8, Canada

Location

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Related Links

• Characterization and Chromosomal localization of the human proto-oncogene
• Human protein atlas
• Exploratory assessment of dose proportionality
• Dose escalation study design example
• Increased polycomb-group oncogene BMI-1expression coorelates with poor prognosis in Hepatocellular carcinoma

MeSH Terms

Conditions

Neoplasms

Interventions

PTC596

Study Officials

• Edward O'Mara, MD

  PTC Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, first-in-human, safety and pharmacokinetic (PK)

Study Record Dates

• First Submitted: March 24, 2015
• First Posted: March 31, 2015
• Study Start: January 1, 2016
• Primary Completion: February 6, 2017
• Study Completion: February 6, 2017
• Last Updated: December 8, 2017

Record last verified: 2017-12

Locations

CA (1); US (3)