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Phase IIa Dose-Expansion and Biomarker Study of OPB-111077

Phase 2
Conditions
NPC
Refractory Tumor
Interventions
Registration Number
NCT03158324
Lead Sponsor
National University Hospital, Singapore
Brief Summary

This is a phase IIa open-label, non-randomized dose-expansion study of OPB-111077 in patients with advanced, treatment refractory cancers who have biopsy-amenable lesions at study entry.

Detailed Description

This is a phase IIa open-label, non-randomized dose-expansion study of OPB-111077 in patients with advanced, treatment refractory cancers who have biopsy-amenable lesions at study entry. Patients on the proposed study will be treated with the RPII dose of OPB-111077 (600mg on a 4 days-on, 3 days-off per week schedule). They will be enrolled in two parallel cohorts: i. patients with tumors predicted to be dependent on oxidative phosphorylation metabolism or oncogene addicted tumors which have developed resistance to primary TKI therapy, or ii. patients with nasopharyngeal carcinoma

Each cohort will contain 11-26 patients, over a period of 12-36 months. Subjects will receive OPB-111077 in 28-day cycles till disease progression or intolerable toxicity. Mandatory tumour biopsies will be performed at baseline and on cycle 1 day 15 (where feasible and accessible). Circulating biomarker blood sampling will be performed on days 1, 11 and 15 of cycle 1, and upon completion of OPB-111077 dosing. Pharmacokinetics blood sampling will be performed on days 11 and 15 of cycle 1. Safety assessments will be performed on cycle 1 day 1, cycle 1 day 8, cycle 1 day 15, bi-weekly till week 8, then monthly thereafter and response assessments will be performed every 8 weeks. Metabolic response assessment by PET/CT will be performed after 2 cycles of treatment, while radiologic response assessment will be performed after every 2 cycles of OPB-111077 from cycle 4 onwards.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
52
Inclusion Criteria
  1. Histologically confirmed, locally recurrent or metastatic solid tumors, who have failed standard treatment
  2. Subjects with NPC will be eligible as long as they have received prior platinum therapy
  3. Subjects with other types of solid tumors will be eligible if: i) their archival tumor sample shows over-expression oxidative phosphorylation markers e.g., PGC-1α/ SIRT1 or ii) they have oncogene-addicted cancers (e.g., EGFR mutation-positive NSCLC, EML4-ALK fusion NSCLC, BRAF-mutant melanomas, GIST, RET-driven thyroid cancers) which have become resistant to primary TKI therapy
  4. All subjects must have at least one tumour lesion (primary or metastatic) that is suitable for free-hand or image-guided biopsy at baseline.
  5. Age ≥ 21 years, Eastern Cooperative Oncology Group (ECOG) performance status < 1
  6. Adequate bone marrow, liver and renal function
  7. Baseline serum lactate ¬<3mmol/l
  8. Capable of swallowing tablets
  9. Recovery from any previous drug- or procedure-related toxicity to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 Grade 0 or 1 (except alopecia), or to baseline preceding the prior treatment.
  10. Signed informed consent obtained before any study specific procedure. Subjects must be able to understand and be willing to sign the written informed consent.
Exclusion Criteria
  1. Chemotherapy, radiotherapy, surgery, immunotherapy or other therapy within 3 weeks of starting investigational medicinal product (IMP).
  2. Use of any prohibited medications (CYP3A4 inhibitors and inducers) or medications which may predispose to lactic acidosis (e.g., metformin, nucleoside analogue reverse within 1 week prior to start of study drug administration
  3. Pregnancy or breastfeeding.
  4. Women of childbearing potential not employing adequate contraception. Women of childbearing potential must have a pregnancy test performed a maximum of 7 days before start of study medication, and a negative result must be documented before start of study medication. Women of childbearing potential and men, must agree to use adequate contraception (barrier method of birth control) upon signing the informed consent form until at least 3 months after the last study drug administration.
  5. Known or suspected allergy to the investigational agent or any agent given in association with this study.
  6. Concurrent cancer which is distinct in primary site or histology from the cancer being evaluated in this study EXCEPT cervical carcinoma in situ, treated basal cell carcinoma, superficial bladder tumours (Ta, Tis & T1) or any cancer curatively treated less than 3 years prior to study entry.
  7. Interstitial lung disease with ongoing signs and symptoms at the time of screening.
  8. Patients with CTCAE Grade 2 or higher peripheral neuropathy.
  9. History of significant cardiac disease: congestive cardiac failure > NYHA class II, ongoing unstable angina, new-onset angina or myocardial infarction within the past 3 months

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Advanced refractory solid tumorsOPB-111077Patients with advanced refractory solid tumors will be enrolled.
Primary Outcome Measures
NameTimeMethod
Objective response rates3 years

This will be calculated as the percentage of evaluable patients achieving complete and partial response with OPB-111077 treatment, according to the RECIST 1.1 criteria

Secondary Outcome Measures
NameTimeMethod
Metabolic response rates3 years

This will be calculated as the percentage of evaluable patients achieving complete and partial metabolic response on 18F\]-FDG PET/CT after 2 cycles of OPB-111077, as determined by the EORTC PET response criteria.

Progression free survival3 years

This is defined as the time from the start of study treatment to documented progression of disease or death.

Haematologic and non-haematologic toxicities (all grades)3 years

To evaluate the haematologic and non-haematologic toxicities of OPB-111077

Trial Locations

Locations (1)

National University Hospital, Singapore

🇸🇬

Singapore, Singapore

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