Phase 2 Study of PT2977 for Treatment of Von Hippel Lindau Disease-Ass

• Research type

  Research Study

• Full title

  An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of von Hippel-Lindau Disease-Associated Renal Cell Carcinoma

• IRAS ID

  245027

• Contact name

  Eamonn Maher

• Contact email

  erm1000@medschl.cam.ac.uk

• Sponsor organisation

  Peloton Therapeutics, Inc.

• Eudract number

  2018-000125-30

• Clinicaltrials.gov Identifier

  NCT03401788

• Duration of Study in the UK

  5 years, 0 months, days

• Research summary

  This is an open-label Phase 2 study, that will evaluate the efficacy and safety of PT2977 in patients with von Hippel-Lindau disease (VHL) who have at least 1 measurable (Renal cell carcinoma (RCC) tumor (as defined by RECIST 1.1).

  VHL disease is a hereditary cancer disease. Individuals affected with this disease have a germline mutation and/or deletion of the VHL gene, therefore at
  risk for developing tumors and fluid-filled sacs (cysts) in a number of organs.

  Renal cell carcinoma occurs in 30% to 70% of individuals with VHL disease and is the leading cause of mortality.
  
  In this Phase 2 clinical trial efficacy and safety of PT2977 will be evaluated in patients with VHL disease who have at least 1 measurable RCC tumor. After meeting all inclusion and no exclusion criteria, enrolled patients will be assigned to treatment with open-label PT2977. The treatment will be administered orally at a dose of 120mg once daily (three 40mg PT2977 tablet once daily). VHL diseased-associated non- RCC tumors, if present, will also be evaluated to assess tumors in individual organ system independently.
  
  This clinical trial is being sponsored by Peloton Therapeutics, Inc. The study will enroll approximately 50 patients and will take place at 2 sites in the United Kingdom.

  Patients will continue to receive PT2977 in the absence of unacceptable treatment-related toxicity or unequivocally disease progression. Patients will undergo clinical and laboratory evaluations according to the Schedule of Events (listed in the protocol). Patients who discontinue study treatment for any reason will undergo long term follow-up every 6 months for up to 3 years following enrollment of last patient into the study or until the patient either withdraws consent for follow-up or dies.

• REC name

  London - Westminster Research Ethics Committee

• REC reference

  18/LO/1104

• Date of REC Opinion

  7 Nov 2018

• REC opinion

  Further Information Favourable Opinion