Clinical trial NCT03573882: Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Official title: An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Sickle Cell Disease
Keywords: Open Label Extension
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2018-06-29
Enrollment: 179
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials

Detailed description: This open label extension (OLE), multi-center study will be conducted at approximately 100 clinical sites globally and will be available to eligible participants from study GBT440-031.

The study will enroll participants from GBT440-031 (approximately 435) under any of the following conditions:

* Participant has completed 72 weeks of treatment regardless of dose selection for GBT440-031
* Dose selection has occurred for GBT440-031 and participant is on non-selected dose on GBT440-031
* GBT440-031 study interim data analysis and/or study modifications have occurred
* GBT440-031 study has completed

The objective of this open-label extension (OLE) study is to assess the long-term safety and treatment effect of voxelotor in participants who have completed treatment in study GBT440-031, using the following parameters:

1. Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
2. Frequency of sickle cell disease (SCD)-related complications.
3. Hemolytic anemia as measured by hematological laboratory parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin).

All participants will receive daily voxelotor treatment.

Participants may receive study drug as long they continue to receive clinical benefit which outweighs risk as determined by the Investigator and/or until the participant has access to voxelotor from an alternative source (i.e., commercialization or through a managed access program).

Eligibility criteria: Inclusion Criteria:

* 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.

* Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
* 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 use barrier methods of contraception to 30 days after the last dose of study drug.
* 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:

* Female who is breast-feeding or pregnant.
* Participant withdrew consent from Study GBT440-031.
* Participant was lost to follow-up from Study GBT440-031.
* Participant requiring chronic dialysis.
* 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.

Primary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
timeFrame: Five Years
measure: Frequency of sickle cell-related complications
timeFrame: Five Years
Secondary outcomes:
measure: Response in Hemolytic Anemia
timeFrame: Five Years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Primary Care Clinic, PA
state: Arkansas
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Miami
country: United States
facility: Jackson Memorial Hospital (Investigational Drug Services)
state: Florida
city: Miami
country: United States
facility: Jackson Memorial Hospital
state: Florida
city: Miami
country: United States
facility: University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta at Hughes Spalding
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Scottish Rite
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Scottish Rite
state: Georgia
city: Chicago
country: United States
facility: University of Illinois at Chicago Clinical Research Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois Hospital and Health Science System
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia and Thrombosis Center
state: Indiana
city: Baton Rouge
country: United States
facility: St. Jude Affiliate Clinic Baton Rouge
state: Louisiana
city: Baton Rouge
country: United States
facility: Our Lady of the Lake Physician Group-Medical Oncology
state: Louisiana
city: New Orleans
country: United States
facility: University Medical Center New Orleans
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins University/Sickle Cell Infusion Center
state: Maryland
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital Research Pharmacy
state: Massachusetts
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Newark
country: United States
facility: Newark Beth Israel Medical Center
state: New Jersey
city: Bronx
country: United States
facility: Montefiore - Einstein Center for Cancer Care
state: New York
city: New York
country: United States
facility: Columbia University Medical Center - Herbert Irving Pavilion
state: New York
city: Chapel Hill
country: United States
facility: UNC Hospitals
state: North Carolina
city: Chapel Hill
country: United States
facility: Clinical and Translational Research Center (CTRC)
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Durham
country: United States
facility: Investigational Drug Service, Duke University Hospital
state: North Carolina
city: Oklahoma City
country: United States
facility: Lynn Health Science Institute
state: Oklahoma
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center (UPMC)
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina - Comprehensive Sickle Cell Clinic
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina: Investigational Drug Services Pharmacy
state: South Carolina
city: Memphis
country: United States
facility: Methodist Comprehensive Sickle Cell Clinic
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital - Investigational Pharmacy
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Richmond
country: United States
facility: Clinical Research Services Unit- Virginia Commonwealth University
state: Virginia
city: Toronto
country: Canada
facility: Toronto General Hospital-University Health Network
state: Ontario
city: Alexandria
country: Egypt
facility: Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
city: Alexandria
country: Egypt
facility: Alexandria Clinical Research Center, Faculty of Medicine
city: Alsharkia
country: Egypt
facility: Zagazig University Hospital
city: Cairo
country: Egypt
facility: Abu El Rich Hospital,Cairo University Hospital
city: Cairo
country: Egypt
facility: Ain Shams University Hospital
city: Cairo
country: Egypt
facility: The Egyptian Thalassemia Association ( E.T.A)
city: Paris
country: France
facility: Hôpital Européen Georges Pompidou - Medecine Interne
city: Paris
country: France
facility: Hôpital Européen Georges Pompidou
state: Île-de-france
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
state: Padova Veneto
city: Siaya
country: Kenya
facility: Centres for Disease Control and Prevention
state: Kisumu
city: Nairobi
country: Kenya
facility: KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
city: Nairobi
country: Kenya
facility: Gertrude's Children's Hospital
city: Beirut
country: Lebanon
facility: American University of Beirut
city: Tripoli
country: Lebanon
facility: Nini Hospital
city: Amsterdam
country: Netherlands
facility: Academic Medical Center(AMC)
city: Rotterdam
country: Netherlands
facility: ErasmusMC
city: Muscat
country: Oman
facility: Sultan Qaboos University Hospital
city: Istanbul
country: Turkey
facility: Erciyes Universitesi Tip Fakultesi Hastanesi
state: İ̇stanbul
city: Adana
country: Turkey
facility: Adana Acibadem Hospital
city: Kayseri
country: Turkey
facility: Erciyes Universitesi Tip Fakultesi
city: Mersin
country: Turkey
facility: Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
city: London
country: United Kingdom
facility: Barts Health NHS Trust
state: Greater London
city: London
country: United Kingdom
facility: Homerton University Hospital NHS Foundation Trust
state: Greater London
city: Great Maze Pond
country: United Kingdom
facility: Guys and St. Thomas Hospital NHS Foundation Trust
state: London
city: Great Maze Pond
country: United Kingdom
facility: Guys and STt Thomas NHS Foundation Trust
state: London
city: London
country: United Kingdom
facility: King's College Hospital
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust, Hammersmith Hospital
city: London
country: United Kingdom
facility: McMillan Cancer Centre
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: