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Completed

NCT Number: NCT05036512

A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants

This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Harry Perkins Institute of Medical Research, Nedlands, Western Australia, Australia

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About this study

This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Healthy males and females ≥ 18 to ≤ 55 years of age
  • Body mass index ≥ 18.0 to ≤ 30.0 kg/m2
  • Body weight ≥ 50 kg at screening and Day -1

Exclusion criteria

  • Positive pregnancy test or currently breastfeeding.

Treatment and study plan

GBT021601

Drug

Administered orally with water as a single dose in the morning.

Other names: Placebo

Primary outcomes

  1. Safety, as assessed by frequency and severity of adverse events (AEs)

    Time frame: 119 days from screening Part A, 134 days from screening Part B

    AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.

  2. Safety, as assessed by changes in Heart Rate.

    Time frame: 119 days from screening Part A, 134 days from screening Part B

    Number of participants with changes in heart rate (bpm) as compared to baseline.

  3. Safety, as assessed by changes in eGFR

    Time frame: 119 days from screening Part A, 134 days from screening Part B

    Number of participants with changes in eGFR from baseline

  4. Safety, as assessed by changes in alanine aminotransferase (ALT)

    Time frame: 119 days from screening Part A, 134 days from screening Part B

    Number of participants with changes in alanine aminotransferase (ALT)

  5. Safety, as assessed by changes in Blood pressure

    Time frame: 119 days from screening Part A, 134 days from screening Part B

    Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood

  6. Plasma concentration

    Time frame: 119 days from screening Part A

    Time of Cmax

  7. Plasma concentration

    Time frame: 134 days from screening Part B

    Cmax on D1-D15

Secondary outcomes

  1. Determine whole blood concentration of GBT021601

    Time frame: 119 days from screening Part A

    Hemoximetry will be used to assess oxygen saturation in whole blood by generating oxygen equilibrium curves (OECs) which relate the extent of Hb-O2 saturation to the partial pressure of O2 (pO2) and measure the binding affinity of O2 to Hb.

  2. Determine plasma concentration of GBT021601.

    Time frame: 134 days from screening Part B

    With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).

  3. Safety, as assessed by changes in QTcF

    Time frame: 119 days from screening Part A, 134 days from screening Part B

    Number of participants with changes in the QTcF interval from baseline

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.

Important dates

Study start
2020
Primary completion
2022
Study completion
2023
First posted
Sep 5, 2021
Registry last updated
Jul 25, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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