Study to assess the safety of Voxelotor with long-term treatment in pediatric populatio

Phase 1

• Conditions: Sickle Cell Disease; MedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2019-003144-76-GB
• Lead Sponsor: Global Blood Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-10-14
• Last Update Posted: 5 January 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Participants who meet all of the following criteria will be eligible for study enrollment:
1. Male or female participant with SCD, aged = 4 to = 18 years, who participated and received study drug in a GBT-sponsored voxelotor pediatric clinical study
Note: Participants 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. Female participant of childbearing potential is required to have a negative urine pregnancy test prior to dosing on Day 1.
Note: Female participants who become childbearing during the study must be willing to have a negative urine pregnancy test to remain in the study.
3. If sexually active, female participant of childbearing potential must use highly effective methods of contraception until 30 days after the last dose of study drug. If sexually active, male participant must use barrier methods of contraception until 30 days after the last dose of study drug.
4. Participant has provided written assent (both the consent of the participant’s legal representative or legal guardian and the participant’s assent [where applicable] must be obtained)
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Participants meeting any of the following exclusion criteria will not be eligible for study enrollment:
1. Female participant who is breastfeeding or pregnant
2. Participant withdrew consent from a GBT-sponsored voxelotor pediatric clinical study
3. Participant was lost to follow-up from a GBT-sponsored voxelotor pediatric clinical study
4. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified