A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-05-21

Study Completion Date

2022-12-06

Brief Summary

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The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in participants with SCD, following single and multiple ascending doses.

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Detailed Description

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This is an open-label intrapatient single dose followed by a multiple dose escalation study in at least six (6) participants with SCD.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Single-dose Period (Part A)

Refer to Study Description

Group Type EXPERIMENTAL

GBT021601

Intervention Type DRUG

Tablets and capsules which contain GBT021601 drug substance

Multiple Ascending-dose Period (Part B and Part C)

Refer to Study Description

Group Type EXPERIMENTAL

GBT021601

Intervention Type DRUG

Tablets and capsules which contain GBT021601 drug substance

Interventions

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GBT021601

Tablets and capsules which contain GBT021601 drug substance

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male or Female with SCD
* Participants with SCD ages 18 to 60 years, inclusive.
* Participant has provided documented informed consent.
* Patients with stable and close to baseline hemoglobin value
* Patients on HU should be on stable dose for at least 90 days prior to signing ICF

Exclusion Criteria

* Patients had more than 10 VOC within 12 months of screening
* Patients who are pregnant or nursing
* Patients who receive RBC transfusion therapy regularly or received an RBC transfusion for any reason within 60 days of signing the ICF
* Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or within 24 days prior to Day 1 treatment

Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No