An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Phase 3

Recruiting

Conditions: Anemia
Sickel cell disease
10035534
10005330

Registration Number: NL-OMON52948

Lead Sponsor: Global Blood Therapeutics

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 19

Inclusion Criteria

1. Male or female study participants with SCD who participated and received
study treatment in Study GBT440-031.
Note: Participants in GBT440-031 who discontinued study drug due to an AE, but
who remained on study may be eligible for treatment in this study provided the
AE does not pose a risk for treatment with Voxelotor.
2. Females of child-bearing potential are required to have a negative urine
pregnancy test prior to dosing on Day 1.
3. Female participants of child-bearing potential must use highly effective
methods of contraception to 30 days after the last dose of study drug. Male
participants must continue to use barrier methods of contraception to 30 days
after the last dose of study drug.
4. Participant has provided written informed consent or assent (the ICF must be
reviewed and signed by each participant; in the case of pediatric participants,
both the consent of the participant*s legal representative or legal guardian,
and the participant*s assent must be obtained).

Exclusion Criteria

1. Female who is breast-feeding or pregnant.
2. Participant withdrew consent from Study GBT440-031.
3. Participant was lost to follow-up from Study GBT440-031.
4. Participant requiring chronic dialysis.
5. Any medical, psychological, safety, or behavioral conditions, which, in the
opinion of the Investigator, may confound safety interpretation, interfere with
compliance, or preclude informed consent.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>1.Safety endpoints include AEs, clinical laboratory tests, physical<br /><br>examinations and other clinical measures.<br /><br>2. Frequency of SCD-related complications with long-term dosing with Voxelotor.<br /><br>3.Hemolysis markers as measured by hematological laboratory parameters (e.g.<br /><br>hemoglobin, reticulocytes and unconjugated bilirubin). </p><br>

Secondary Outcome Measures