search
Back to results

GBT021601-021: A Study in Adult and Pediatric Participants With SCD

Primary Purpose

Sickle Cell Disease

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
GBT021601
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

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

Inclusion Criteria:

Part A, Part B, and Part C:

  • Male or female with SCD
  • Participants with stable Hb value as judged by the Investigator
  • For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

  • Participants with SCD ages 12 to 65 years, inclusive
  • Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

Exclusion Criteria:

Part A, Part B, and Part C:

  • Participants who had more than 10 VOC within 12 months of screening
  • Female participant who is breastfeeding or pregnant
  • Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
  • Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF

Sites / Locations

  • Lynn Institute of the Ozarks
  • Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center
  • Edward Jenner Research Group LLC.Recruiting
  • Kidz Hematology / Oncology a division of Kidz Medical
  • St. Mary's Medical Center
  • Alpha Clinical Research GeorgiaRecruiting
  • University of Illinois Hospital and Health Sciences System - Hospital Pharmacy Services
  • University of Illinois Hospital and Health Sciences System
  • University of Illinois at Chicago Clinical Research Center
  • LSU Health Baton Rouge-North ClinicRecruiting
  • Our lady of the Lake Hospital
  • Our Lady of the Lake Hopistal, Inc
  • Our Lady of the Lake Hopistal, IncRecruiting
  • Our Lady of the Lake Hospital, Inc In-Patient PharmacyRecruiting
  • Our Lady of the Lake Physicians Group, Medical OncologyRecruiting
  • University Medical Center New OrleansRecruiting
  • Mississippi Center for Advanced MedicineRecruiting
  • UT Physicians Comprehensive Sickle Cell ClinicRecruiting
  • McGovern Medical School/Health Science Center HoustonRecruiting
  • Memorial Hermann - TMC Investigational Drugs, IDS PharmacyRecruiting
  • Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)Recruiting
  • UT Physicians Comprehensive Sickle Cell ClinicRecruiting
  • Inova Schar Cancer Institute Infusion PharmacyRecruiting
  • Inova Schar Cancer InstituteRecruiting
  • Virginia Commonwealth University- Clinical Research Services Unit
  • Virginia Commonwealth University
  • Gertrude's Children's Hospital
  • Nini Hospital s.a:l
  • University College Hospital IbadanRecruiting
  • Aminu Kano Teaching HospitalRecruiting
  • Lagos University Teaching HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Placebo Comparator

Experimental

Arm Label

Part A

Part B

Part C

Arm Description

Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg. Participants will then receive maintenance once daily doses through Week 12.

Following the selection of the optimal safe and effective dose from Part A of the study, Part B of the study will assess the efficacy and safety of 48 weeks of the optimal dose, compared to placebo

100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data

Outcomes

Primary Outcome Measures

Part A
Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in GBT021601 concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Part B
Proportion of participants with an increase from baseline of >1 g/dL in Hb at week 48 as measured by change in GBT021601 concentrations from baseline or percentage change from baseline of clinical measures of anemia (hemoglobin) and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Part C
Assess the pharmacokinetics (PK), while observing maximum concentration (Cmax) after a single dose as measured by a noncompartmental PK analysis or population PK analysis using nonlinear mixed-effect modeling. To characterize GBT021601 PK in plasma and whole blood following a single dose.
Part C
Assess the PK, while observing minimum concentration (Cmin) and Cmax after multiple dose administration as measured by a noncompartmental PK analysis or population PK analysis using nonlinear mixed-effect modeling will be performed to characterize GBT021601 PK in plasma and whole blood following multiple doses.

Secondary Outcome Measures

Full Information

First Posted
June 21, 2022
Last Updated
October 7, 2023
Sponsor
Pfizer
search

1. Study Identification

Unique Protocol Identification Number
NCT05431088
Brief Title
GBT021601-021: A Study in Adult and Pediatric Participants With SCD
Official Title
A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants With Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants With Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 22, 2022 (Actual)
Primary Completion Date
June 13, 2026 (Anticipated)
Study Completion Date
June 13, 2026 (Anticipated)

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
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of GBT021601.
Detailed Description
This is a three-part, multicenter, Phase 2/3 study of orally administered GBT021601 in participants with sickle cell disease (SCD). Part A will evaluate the safety, tolerability, and efficacy of GBT021601 in adult participants with SCD to determine an optimal dose. Part B will evaluate the efficacy of GBT021601 versus placebo in adult and pediatric participants with SCD for 48 weeks. Part C will evaluate the pharmacokinetics (PK) and safety of single and multiple doses (MD) of open-label single arm GBT021601 administered to pediatric participants.

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 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Part B only
Allocation
Randomized
Enrollment
517 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Part A
Arm Type
Active Comparator
Arm Description
Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg. Participants will then receive maintenance once daily doses through Week 12.
Arm Title
Part B
Arm Type
Placebo Comparator
Arm Description
Following the selection of the optimal safe and effective dose from Part A of the study, Part B of the study will assess the efficacy and safety of 48 weeks of the optimal dose, compared to placebo
Arm Title
Part C
Arm Type
Experimental
Arm Description
100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data
Intervention Type
Drug
Intervention Name(s)
GBT021601
Intervention Description
Tablets which contain GBT021601 drug substance
Primary Outcome Measure Information:
Title
Part A
Description
Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in GBT021601 concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Time Frame
Through week 12
Title
Part B
Description
Proportion of participants with an increase from baseline of >1 g/dL in Hb at week 48 as measured by change in GBT021601 concentrations from baseline or percentage change from baseline of clinical measures of anemia (hemoglobin) and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Time Frame
Through week 48
Title
Part C
Description
Assess the pharmacokinetics (PK), while observing maximum concentration (Cmax) after a single dose as measured by a noncompartmental PK analysis or population PK analysis using nonlinear mixed-effect modeling. To characterize GBT021601 PK in plasma and whole blood following a single dose.
Time Frame
Through Week 6
Title
Part C
Description
Assess the PK, while observing minimum concentration (Cmin) and Cmax after multiple dose administration as measured by a noncompartmental PK analysis or population PK analysis using nonlinear mixed-effect modeling will be performed to characterize GBT021601 PK in plasma and whole blood following multiple doses.
Time Frame
Through Week 2

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Part A, Part B, and Part C: Male or female with SCD Participants with stable Hb value as judged by the Investigator For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator. Part B: Participants with SCD ages 12 to 65 years, inclusive Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening. Exclusion Criteria: Part A, Part B, and Part C: Participants who had more than 10 VOC within 12 months of screening Female participant who is breastfeeding or pregnant Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1 Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Pfizer Pfizer CT.gov Call Center
Phone
1-800-718-1021
Email
ClinicalTrials.gov_Inquiries@pfizer.com
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:
Facility Name
Lynn Institute of the Ozarks
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72204
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center
City
Torrance
State/Province
California
ZIP/Postal Code
90502
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Edward Jenner Research Group LLC.
City
Miami
State/Province
Florida
ZIP/Postal Code
33317
Country
United States
Individual Site Status
Recruiting
Facility Name
Kidz Hematology / Oncology a division of Kidz Medical
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33407
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
St. Mary's Medical Center
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33407
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Alpha Clinical Research Georgia
City
Dunwoody
State/Province
Georgia
ZIP/Postal Code
30350
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Illinois Hospital and Health Sciences System - Hospital Pharmacy Services
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612-4333
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University of Illinois Hospital and Health Sciences System
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612-7232
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University of Illinois at Chicago Clinical Research Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
LSU Health Baton Rouge-North Clinic
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70805
Country
United States
Individual Site Status
Recruiting
Facility Name
Our lady of the Lake Hospital
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70805
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Our Lady of the Lake Hopistal, Inc
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Our Lady of the Lake Hopistal, Inc
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Individual Site Status
Recruiting
Facility Name
Our Lady of the Lake Hospital, Inc In-Patient Pharmacy
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Individual Site Status
Recruiting
Facility Name
Our Lady of the Lake Physicians Group, Medical Oncology
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Individual Site Status
Recruiting
Facility Name
University Medical Center New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Individual Site Status
Recruiting
Facility Name
Mississippi Center for Advanced Medicine
City
Madison
State/Province
Mississippi
ZIP/Postal Code
39110
Country
United States
Individual Site Status
Recruiting
Facility Name
UT Physicians Comprehensive Sickle Cell Clinic
City
Houston
State/Province
Texas
ZIP/Postal Code
77004
Country
United States
Individual Site Status
Recruiting
Facility Name
McGovern Medical School/Health Science Center Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
UT Physicians Comprehensive Sickle Cell Clinic
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
Inova Schar Cancer Institute Infusion Pharmacy
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22031
Country
United States
Individual Site Status
Recruiting
Facility Name
Inova Schar Cancer Institute
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22031
Country
United States
Individual Site Status
Recruiting
Facility Name
Virginia Commonwealth University- Clinical Research Services Unit
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Gertrude's Children's Hospital
City
Nairobi
ZIP/Postal Code
00100
Country
Kenya
Individual Site Status
Not yet recruiting
Facility Name
Nini Hospital s.a:l
City
Tripoli
Country
Lebanon
Individual Site Status
Not yet recruiting
Facility Name
University College Hospital Ibadan
City
Ibadan
State/Province
Oyo/ibadan North
ZIP/Postal Code
200212
Country
Nigeria
Individual Site Status
Recruiting
Facility Name
Aminu Kano Teaching Hospital
City
Kano
ZIP/Postal Code
700233
Country
Nigeria
Individual Site Status
Recruiting
Facility Name
Lagos University Teaching Hospital
City
Lagos
ZIP/Postal Code
100254
Country
Nigeria
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT021601-021
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

GBT021601-021: A Study in Adult and Pediatric Participants With SCD

We'll reach out to this number within 24 hrs