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Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
voxelotor
Placebo
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease

Eligibility Criteria

12 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female study participants with sickle cell disease
  2. Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months.
  3. Age 12 to 65 years
  4. Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening
  5. For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF.

Exclusion Criteria:

  1. More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit
  2. Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 60 days of signing the ICF
  3. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to signing the ICF)
  4. Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × upper limit of normal
  5. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30 mL/min/1.73 m^2 or on chronic dialysis

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Active Comparator

Placebo Comparator

Arm Label

Dose 1

Dose 2

Placebo

Arm Description

voxelotor

voxelotor

Placebo

Outcomes

Primary Outcome Measures

Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24
Number of participants with increase in Hb >1 g/dL from Baseline to Week 24

Secondary Outcome Measures

Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate
Number of Vaso-Occlusive Crisis (VOC) events averaged per year.
Percentage Change From Baseline in Hemolysis Measures
Percentage change from Baseline to week 24 in unconjugated bilirubin
Percentage Change From Baseline in Hemolysis Measures
Percentage change from Baseline to week 24 in the absolute reticulocyte which is used to estimate the degree of effective erythropoiesis. This values is important in Sickle Cell Disease and was reported by the central laboratory.
Percentage Change From Baseline in Hemolysis Measures
Percentage change from Baseline to week 24 in reticulocytes % which is a % of total Red Blood Cells (RBCs).
Percentage Change From Baseline in Hemolysis Measures
Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)

Full Information

First Posted
January 27, 2017
Last Updated
July 17, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT03036813
Brief Title
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Acronym
GBT_HOPE
Official Title
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
December 2016 (Actual)
Primary Completion Date
October 8, 2019 (Actual)
Study Completion Date
October 8, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Detailed Description
This is a randomized, placebo-controlled, double blind, parallel group, multicenter study of participants, age 12 to 65 years, with SCD. The key purpose for the study is to establish efficacy and safety of voxelotor as compared with placebo.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Masking Description
This study is a double-blind study.
Allocation
Randomized
Enrollment
449 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dose 1
Arm Type
Active Comparator
Arm Description
voxelotor
Arm Title
Dose 2
Arm Type
Active Comparator
Arm Description
voxelotor
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
voxelotor
Other Intervention Name(s)
GBT440
Intervention Type
Other
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24
Description
Number of participants with increase in Hb >1 g/dL from Baseline to Week 24
Time Frame
Baseline to Week 24
Secondary Outcome Measure Information:
Title
Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate
Description
Number of Vaso-Occlusive Crisis (VOC) events averaged per year.
Time Frame
Baseline to Week 72
Title
Percentage Change From Baseline in Hemolysis Measures
Description
Percentage change from Baseline to week 24 in unconjugated bilirubin
Time Frame
Baseline to Week 24
Title
Percentage Change From Baseline in Hemolysis Measures
Description
Percentage change from Baseline to week 24 in the absolute reticulocyte which is used to estimate the degree of effective erythropoiesis. This values is important in Sickle Cell Disease and was reported by the central laboratory.
Time Frame
Baseline to Week 24
Title
Percentage Change From Baseline in Hemolysis Measures
Description
Percentage change from Baseline to week 24 in reticulocytes % which is a % of total Red Blood Cells (RBCs).
Time Frame
Baseline to Week 24
Title
Percentage Change From Baseline in Hemolysis Measures
Description
Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)
Time Frame
Baseline to Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female study participants with sickle cell disease Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months. Age 12 to 65 years Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF. Exclusion Criteria: More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 60 days of signing the ICF Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to signing the ICF) Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × upper limit of normal Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30 mL/min/1.73 m^2 or on chronic dialysis
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35205
Country
United States
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36693
Country
United States
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72204
Country
United States
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
City
Newark
State/Province
New Jersey
ZIP/Postal Code
07112
Country
United States
City
Bronx
State/Province
New York
ZIP/Postal Code
11501
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15219
Country
United States
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
City
Toronto
ZIP/Postal Code
M5G 2C4
Country
Canada
City
Alexandria
ZIP/Postal Code
21131
Country
Egypt
City
Cairo
ZIP/Postal Code
11566
Country
Egypt
City
Cairo
Country
Egypt
City
Zagazig
ZIP/Postal Code
44519
Country
Egypt
City
Créteil
ZIP/Postal Code
94010
Country
France
City
Paris
ZIP/Postal Code
75743
Country
France
City
Paris
ZIP/Postal Code
75908
Country
France
City
Monza
State/Province
Milano
ZIP/Postal Code
20900
Country
Italy
City
Padova
ZIP/Postal Code
35128
Country
Italy
City
Verona
ZIP/Postal Code
37134
Country
Italy
City
Kingston
ZIP/Postal Code
JMAAW15
Country
Jamaica
City
Nairobi
ZIP/Postal Code
42325-00100
Country
Kenya
City
Nairobi
ZIP/Postal Code
47855
Country
Kenya
City
Nairobi
ZIP/Postal Code
59857-00200
Country
Kenya
City
Siaya
ZIP/Postal Code
144-40600
Country
Kenya
City
Beirut
ZIP/Postal Code
11072020
Country
Lebanon
City
Beirut
ZIP/Postal Code
1136044
Country
Lebanon
City
Tripoli
ZIP/Postal Code
1434
Country
Lebanon
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
City
Den Haag
ZIP/Postal Code
2545 CH
Country
Netherlands
City
Rotterdam
ZIP/Postal Code
3015 AA
Country
Netherlands
City
Muscat
ZIP/Postal Code
123
Country
Oman
City
Adana
ZIP/Postal Code
01130
Country
Turkey
City
Kayseri
ZIP/Postal Code
38039
Country
Turkey
City
Mersin
ZIP/Postal Code
33342
Country
Turkey
City
London
ZIP/Postal Code
E11BB
Country
United Kingdom
City
London
ZIP/Postal Code
E96SR
Country
United Kingdom
City
London
ZIP/Postal Code
SE17EH
Country
United Kingdom
City
London
ZIP/Postal Code
SE59NU
Country
United Kingdom
City
London
ZIP/Postal Code
W12 0HS
Country
United Kingdom
City
London
ZIP/Postal Code
WC1N3BG
Country
United Kingdom
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
33838113
Citation
Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.
Results Reference
derived
PubMed Identifier
33476432
Citation
Minniti CP, Knight-Madden J, Tonda M, Gray S, Lehrer-Graiwer J, Biemond BJ. The impact of voxelotor treatment on leg ulcers in patients with sickle cell disease. Am J Hematol. 2021 Apr 1;96(4):E126-E128. doi: 10.1002/ajh.26101. Epub 2021 Feb 19. No abstract available.
Results Reference
derived
PubMed Identifier
31199090
Citation
Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.
Results Reference
derived
PubMed Identifier
28337324
Citation
Metcalf B, Chuang C, Dufu K, Patel MP, Silva-Garcia A, Johnson C, Lu Q, Partridge JR, Patskovska L, Patskovsky Y, Almo SC, Jacobson MP, Hua L, Xu Q, Gwaltney SL 2nd, Yee C, Harris J, Morgan BP, James J, Xu D, Hutchaleelaha A, Paulvannan K, Oksenberg D, Li Z. Discovery of GBT440, an Orally Bioavailable R-State Stabilizer of Sickle Cell Hemoglobin. ACS Med Chem Lett. 2017 Jan 23;8(3):321-326. doi: 10.1021/acsmedchemlett.6b00491. eCollection 2017 Mar 9.
Results Reference
derived
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT440-031
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

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