Clinical trial NCT04299048: Study to Assess the Safety and Tolerability of Repeated Doses of an Investigational New Drug in Patients With Cancer and Cachexia.
Official title: A PHASE 1B, 12-WEEK, OPEN-LABEL STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS FOLLOWING REPEATED SUBCUTANEOUS ADMINISTRATIONS OF PF-06946860 IN PATIENTS WITH CANCER AND CACHEXIA
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cachexia
Non-Small-Cell Lung Cancer
Pancreatic Cancer
Colorectal Cancer
Keywords: cachexia, non-small cell lung cancer, pancreatic cancer
colorectal cancer, weight loss, anorexia, muscle loss, fatigue
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2020-03-06
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: Study to assess the safety and tolerability of repeated doses of an investigational new drug in patients with cancer and cachexia.

Detailed description: This 12-week open-label study will explore how PF-06946860 is tolerated, the effects of the study drug, the best dose for treatment and how participants with non-small cell lung, pancreatic or colorectal cancer and cachexia feel after receiving repeated subcutaneous dosing. During the 12-week treatment period, study drug will be administered subcutaneously every 3 weeks for a total of 5 doses. There is a 12-week follow-up period following the last dose of study drug. Additional assessments include:

* body weight measurements
* blood pressure and heart rate measurements
* Lumbar Skeletal Muscle Index (LSMI) by CT scan
* Blood samples:

  * to evaluate safety,
  * to measure the amount of the study drug in the blood,
  * to evaluate if the study drug causes an immune response,
  * to examine the effects of the study drug on levels of a specific cytokine,
  * and for exploratory samples for bio banking.
* Measure the impact of the study drug on appetite, nausea, vomiting, fatigue, physical function, and health-related quality of life with questionnaires.
* Measure the impact of study drug on physical activity using wearable digital sensors.
* To evaluate the effect of study drug on ability to complete anti-tumor treatment and survival in participants with cancer and cachexia.
* To evaluate tumor size.

Eligibility criteria: Inclusion Criteria:

* Documented histologic or cytologic diagnosis of advanced metastatic NSCLC, advanced/unresectable pancreatic cancer, or metastatic colorectal cancer.
* Cachexia, defined by BMI \<20 kg/m2 with involuntary weight loss of \>2% within 6 months prior to screening or Involuntary weight loss of \>5% within 6 months prior to screening irrespective of BMI or If medical record documentation is unavailable, patient's report will suffice to estimate involuntary body weight loss.;
* Will receive the following for non-small cell lung cancer:

  * a platinum + pemetrexed ± pembrolizumab or
  * a platinum + nab paclitaxel or paclitaxel ± pembrolizumab or
  * pembrolizumab alone
* Will receive the following for pancreatic cancer:

  * FOLFIRINOX or
  * Nab-Paclitaxel + Gemcitabine
  * Gemcitabine
* Will receive the following for colorectal cancer:

  * FOLFOX +/- Biologic (Bevacizumab or Cetuximab/Panitumumab) or
  * FOLFIRI +/- Biologic (Bevacizumab or Cetuximab/Panitumumab) or
  * FOLFOXIRI +/- Biologic (Bevacizumab or Cetuximab/Panitumumab) or
  * Pembrolizumab for MSI-H • Will be entering the study at the first or second cycle of their current course of anti-cancer treatment/ therapy.
* Adequate renal and liver function.
* Signed informed consent.

Exclusion Criteria:

* All other forms of cancers not specified above unless currently considered cured (\>5 years without evidence of recurrence).
* Planned radiation therapy as part of the primary anti-tumor therapy regimen. However, localized radiation therapy for symptomatic relief is permitted
* Cachexia caused by other reasons: Severe COPD requiring use of home O2, heart failure or AIDS.
* known symptomatic brain metastases requiring steroids.
* Active hepatitis B or C virus.
* Confirmed positive HIV test.
* Current active reversible causes of decreased food intake.
* Receiving tube feedings or parenteral nutrition at Screening.
* Elevated blood pressure that cannot be controlled by medications.
* Women who are pregnant or breast-feeding

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
timeFrame: From Day 1 up to Week 24
measure: Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality)
timeFrame: From Day 1 up to Week 24
measure: Number of Participants With Post-Baseline Vital Signs Abnormalities
timeFrame: From Day 1 up to Week 24
measure: Number of Participants With Post-Baseline Electrocardiogram (ECG) Abnormalities
timeFrame: From Day 1 up to Week 24
Secondary outcomes:
measure: Serum Unbound Trough Concentrations (Ctrough) of PF-06946860
timeFrame: Pre-dose at Weeks 3, 6, 9 and 12, and at Week 15
measure: Serum Total Ctrough of PF-06946860
timeFrame: Pre-dose at Weeks 3, 6, 9 and 12, and at Week 15
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: Beverly Hills Cancer Center
state: California
city: Grand Junction
country: United States
facility: SCL Health Cancer Centers of Colorado - St. Mary's Hospital and Regional Medical Center
state: Colorado
city: Wheat Ridge
country: United States
facility: Lutheran Medical Center
state: Colorado
city: Tallahassee
country: United States
facility: Tallahassee Memorial Healthcare Cancer Center
state: Florida
city: Fort Wayne
country: United States
facility: Fort Wayne Medical Oncology and Hematology, Inc.
state: Indiana
city: Scarborough
country: United States
facility: New England Cancer Specialists
state: Maine
city: Bethesda
country: United States
facility: American Oncology Partners of Maryland, PA
state: Maryland
city: Germantown
country: United States
facility: American Oncology Partners of Maryland, PA
state: Maryland
city: Durham
country: United States
facility: Duke Cancer Center
state: North Carolina
city: Seattle
country: United States
facility: VA Puget Sound Health Care System
state: Washington
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: