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

Pfizer

Status and phase: Completed; Phase 1

Conditions: Sickle Cell Disease

Treatments: Drug: GBT021601

Study type: Interventional

Funder types: Industry

Identifiers

NCT05036512

GBT021601-011

C5351001 (Other Identifier)

Details and patient eligibility

About

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.

Full description

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

Enrollment: 129 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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129 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Placebo as a tablet or capsule with dose based off of preceding cohort's data.

Treatment:

Drug: GBT021601

GBT021601

Experimental group

Description:

GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.

Treatment:

Drug: GBT021601