A Phase 2A Study of ENV-101 in People with PTCH1-Mutated Advanced Solid Tumors


Full Title

A Phase 2, Multi-Center Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients with Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations


In this study, researchers are assessing two different doses of the investigational drug ENV-101 and evaluating the medication’s effectiveness in people with advanced solid tumors that contain a mutation in the PTCH1 gene. ENV-101 blocks a protein that is thought to be involved in the growth of cancer cells with the PTCH1 mutation. By blocking this protein, the medication may help slow or stop cancer growth. ENV-101 is taken orally (by mouth).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have an advanced solid tumor that has a PTCH1 mutation resulting in loss of PTCH1 function.
  • Patients’ cancers cannot be successfully treated with existing therapies.
  • The serious side effects of prior therapies must go away before ENV-101 is given.
  • Patients must be physically well enough that they are able to be mobile, take care of themselves, and engage in all but physically strenuous activities. For example, they must be well enough that they could carry out office work or light housework.
  • This study is for people age 18 and older.


For more information about this study and to ask about eligibility, please contact Dr. Yonina Murciano-Goroff at 646-888-4226.





Disease Status

Relapsed or Refractory


ClinicalTrials.gov ID