GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)

• Sponsor: Global Blood Therapeutics (Industry)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT05632354
• Collaborator: (none)
• Enrollment: 314
• Arm: 1
• Anticipated Duration (Months): 61.9

Study Details

Study Description

Brief Summary

An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease	Drug: open-label GBT021601	Phase 2/Phase 3

Detailed Description

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of GBT021601 Administered to Participants with Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 314 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-label Extension Study to Evaluate the Long-term Safety of GBT021601 Administered to Participants With Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial
• Anticipated Study Start Date: Jan 3, 2023
• Anticipated Primary Completion Date: Mar 1, 2027
• Anticipated Study Completion Date: Mar 1, 2028

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Treatment open-label GBT021601	Drug: open-label GBT021601; open-label GBT021601

Outcome Measures

Primary Outcome Measures

1. To evaluate the incidence of SCD treatment-emergent adverse events with the daily dosing of GBT021601 in participants with sickle cell disease [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Incidence of treatment-emergent adverse events.

2. To evaluate the effects of long-term use of GBT021601 on hemolytic anemia. [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change from baseline in hematological laboratory parameters.

3. To evaluate the long-term effects of GBT021601 treatment on inflammation [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change from baseline in adhesion of whole blood to microfluidic channels.

4. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) assessments [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change from baseline in QOL assessments including Patient Global Impression of Change (PGI-C).

5. To evaluate the change from baseline in hemoglobin [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change in hemoglobin of daily dosing of GBT021601 in participants with SCD

6. To evaluate the change from baseline in reticulocytes [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change in reticulocytes from baseline of daily dosing of GBT021601 in participants with SCD

7. To evaluate the long-term effects of GBT021601 treatment on inflammation [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change from baseline in adhesion of white blood cells to microfluidic channels.

8. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change from baseline in QOL assessments including Clinical Global Impression of Change (CGI-C).

9. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Change from baseline in QOL assessments including Patient Global Impression of Severity (PGI-S).

10. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

    Change from baseline in QOL assessments including Clinical Global Impression of Severity (CGI-S).

11. To evaluate change from baseline in lactate dehydrogenase [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

    Changes in lactate dehydrogenase from baseline of daily dosing of GBT021601 in participants with SCD

12. To evaluate change from baseline in unconjugated bilirubin [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

    Changes in unconjugated bilirubin from baseline of daily dosing of GBT021601 in participants with SCD

Secondary Outcome Measures

1. To evaluate the pharmacokinetic parameters of long-term exposure to GBT021601 [Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years]

   Trough and 1-to 2-hour post dose blood and plasma concentrations of GBT021601.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Months to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Male or female age 6 months or older with SCD who participated and received study drug or placebo in the previous GBT-Sponsored GBT021601 clinical study and remained on the previous study within 30 calendar days of the Day 1 visit for Study GBT021601-022.

Note: Participants who discontinued study drug in the originating study due to an TEAE, but who remained on study, may be eligible for treatment in this study provided the TEAE does not pose a risk for treatment with GBT021601.

1. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.

Note: Female participants who become of childbearing potential during the study must be willing to have negative urine pregnancy tests to remain in the study.

1. If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 120 days after the last dose of study drug.

2. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirements.

Exclusion Criteria:

• Participant withdrew consent or was noncompliant from the originating GBT021601 clinical study.

Current or recent use of voxelotor. Recent use is defined as within 10 days prior to Day 1.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Global Blood Therapeutics

Investigators

• Study Director: Adeyemi Adenola, MD, MPH, Global Blood Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Global Blood Therapeutics
• ClinicalTrials.gov Identifier: NCT05632354
• Other Study ID Numbers: GBT021601-022
• First Posted: Nov 30, 2022
• Last Update Posted: Nov 30, 2022
• Last Verified: Nov 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Anemia, Sickle Cell

Study Results

No Results Posted as of Nov 30, 2022