Sickle Cell Disease MedDRA version: 21.0 Level: PT Classification code 10040644 Term: Sickle cell disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Voxelotor (500 mg)
Pharmaceutical Form: Tablet
INN or Proposed INN: VOXELOTOR
CAS Number: 1446321-46-5
Current Sponsor code: GBT440-038
Concentration unit: mg milligram(s)
Concentration
Sponsors
Global Blood Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of this open-label extension (OLE) is to assess the safety of, and SCD-related complications with, long-term treatment with voxelotor, in pediatric participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor pediatric clinical study, based on the following parameters: • Adverse events (AEs), clinical laboratory tests, physical examinations, and other clinical measures • Frequency of SCD-related complications;Secondary Objective: n/a;Primary end point(s): Safety • TEAEs and SAEs Sickle Cell Disease–Related Complications • Frequency of SCD-related complications;Timepoint(s) of evaluation of this end point: All participants who receive at least 1 dose of study drug in this study will be included in the safety population. Clinical laboratory test and vital sign results over time will be summarized using descriptive statistics. SCD-related complications over the course of the treatment period will be recorded for each participant. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): n/a;Timepoint(s) of evaluation of this end point: n/a | — |
Countries
Lebanon, United Kingdom, United States
Contacts
Public ContactClinical Trial Manager
Global Blood Therapeutics, Inc.
Outcome results
None listed