Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease

NCT04255875 | Completed Phase 1 FDA Drug

• 1 other identifier: C4071001
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 16

Brief Summary

This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in healthy participants and participants with sickle cell disease.

Conditions

Healthy; Sickle Cell Anemia

Keywords

Safety; Tolerability; Single ascending dose; Multiple dose; Pharmacokinetics; Phase 1; First in human; First in patient

Outcome Measures

Primary Outcomes (6)

• Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs

  Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs

  Day 1 up to Day 85 (SAD) or Day 113 (MD)

• Percentage of subjects with laboratory abnormalities

  Percentage of subjects with laboratory abnormalities

  Day 1 up to Day 85 (SAD) or Day 113 (MD)

• Number of subjects with change from baseline in vital signs

  blood pressure, pulse rate, temperature, respiration rate

  Day 1 up to Day 85 (SAD) or Day 85 (MD)

• Number of subjects with change from baseline in electrocardiogram (ECG) parameters

  Number of subjects with change from baseline in electrocardiogram (ECG) parameters

  Day 1 up to Day 85 (SAD) or Day 85 (MD)

• Percentage of subjects with injection site reactions

  Percentage of subjects with injection site reactions

  Day 1 up to Day 11 post (SAD) Day 1 up to Day 85 (MD)

• Percentage of subjects with infusion site reactions

  Percentage of subjects with infusion site reactions

  Day 1 up to Day 11 post each dose (SD)

Secondary Outcomes (17)

• SAD: Single Dose PK /Cmax

  Day 1 up to Day 85

• SAD: Single Dose PK / DN Cmax

  Day 1 up to Day 85

• SAD: Single Dose PK / Tmax

  Day 1 up to Day 85

• SAD: Single Dose PK / AUClast

  Day 1 up to Day 85

• SAD: Single Dose PK / DN AUClast

  Day 1 up to Day 85

Study Arms (3)

Treatment Healthy Participants

EXPERIMENTAL

Participants will receive single ascending doses of subcutaneous (SC) or intravenous PF-07209326

Biological: PF-07209326

Placebo Healthy Participants

PLACEBO COMPARATOR

Participants will receive matching placebo

Biological: Placebo

Treatment for SCD

EXPERIMENTAL

Participants will receive a multiple dose of subcutaneous PF-07209326

Biological: PF-07209326

Interventions

Placebo BIOLOGICAL

Participants will receive matching placebo

Placebo Healthy Participants

PF-07209326 BIOLOGICAL

Participants will receive SC or IV single ascending doses

Treatment Healthy Participants

Eligibility Criteria

• Age: 16 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Body Mass Index (BMI) of 17.5 to 30.5 kg/m2; and a total body weight \>50 kg (110 lbs).

You may not qualify if:

• Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, immunocompromised (or known disorder of the immune system), cardiovascular, hepatic, psychiatric, neurological, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at the time of dosing).
• History of human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C; positive testing for HIV, hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), or hepatitis C antibody (HCVAb). Hepatitis B vaccination is allowed.
• History of active or latent tuberculosis (TB) regardless of treatment or positive QuantiFeron TB test.
• Participants with any of the following acute or chronic infections or infection history:
• Any infection requiring treatment within 2 weeks prior to the screening visit.
• Any infection requiring hospitalization, parenteral antimicrobial therapy within 30 days of the first dose of investigational product.
• Any infection judged to be an opportunistic infection, within the past 6 months of the first dose of the investigational product.
• Known active or history of frequent bacterial, viral, fungal, mycobacterial or other infections as determined by the PI.
• Participants with a fever within the last 7 days prior to dosing.
• Participants with a history of allergic or anaphylactic reaction to therapeutic or diagnostic protein.
• Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
• Participants between the ages of 16 and 70 years old with a confirmed diagnosis of stable sickle cell disease (HbSS or HBS β0 thalassemia).
• Medical history of ≥2 and ≤ 10 medical utilization VOCs in 12 months prior to screening.
• ≥75% of daily ePRO diary completion, over a minimum of 14 days during the screening period.
• Fully vaccinated for COVID-19 in accordance with the Center for Disease Control guidance prior to Screening or must be negative for SARS-CoV-2 by polymerase chain reaction (PCR) within 72 hours of the Day 1 visit.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (16)

New Haven Clinical Research Unit

New Haven, Connecticut, 06511, United States

Location

Howard University College of Medicine

Washington D.C., District of Columbia, 20060, United States

Location

Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States

Location

Lee Health - Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States

Location

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33023, United States

Location

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33024, United States

Location

Children's Healthcare of Atlanta - Egleston Hospital-Aflac Cancer and Blood Disorders Center

Atlanta, Georgia, 30322, United States

Location

University of Illinois at Chicago Clinical Research Center

Chicago, Illinois, 60612, United States

Location

University of Illinois at Chicago

Chicago, Illinois, 60612, United States

Location

Prism Research LLC dba Nucleus Network

Saint Paul, Minnesota, 55114, United States

Location

Columbia University Medical Center - Herbert Irving Pavilion

New York, New York, 10032, United States

Location

CUIMC Research Pharmacy

New York, New York, 10032, United States

Location

CUMC Research Pharmacy

New York, New York, 10032, United States

Location

UT Physicians Comprehensive Sickle Cell Center Houston

Houston, Texas, 77004, United States

Location

Memorial Hermann clinical research unit

Houston, Texas, 77030, United States

Location

UT Physicians Comprehensive Sickle Cell Center Houston

Houston, Texas, 77030, United States

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Masking will only be applicable to Part 1 of the study where Healthy participants will be enrolled and randomized to receive either PF-07209326 or to placebo. In Part 2 of the study, all eligible SCD participants will receive PF-07209326 and no masking will be required.
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2020
• First Posted: February 5, 2020
• Study Start: February 5, 2020
• Primary Completion: July 7, 2023
• Study Completion: July 7, 2023
• Last Updated: December 18, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (16)