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A Phase 2/3 Study in Adult and Pediatric Participants With SCD

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ClinicalTrials.gov Identifier: NCT05431088
Recruitment Status : Recruiting
First Posted : June 24, 2022
Last Update Posted : April 8, 2024
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Osivelotor Phase 2 Phase 3

Detailed Description:

This is a three-part, multicenter, Phase 2/3 study of orally administered osivelotor in participants with sickle cell disease (SCD).

Part A will evaluate the safety, tolerability, and efficacy of osivelotor in adult participants with SCD to determine an optimal dose.

Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent 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 osivelotor administered to pediatric participants.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 517 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Part B only
Primary Purpose: Treatment
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
Actual Study Start Date : September 22, 2022
Estimated Primary Completion Date : October 2, 2026
Estimated Study Completion Date : October 2, 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Part A

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.

 Drug: Osivelotor
Tablets which contain drug substance
Other Name: PF-07940367 or GBT021601
Placebo Comparator: Part B
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
 Drug: Osivelotor
Tablets which contain drug substance
Other Name: PF-07940367 or GBT021601
Experimental: Part C
100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data
 Drug: Osivelotor
Tablets which contain drug substance
Other Name: PF-07940367 or GBT021601


Outcome Measures
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Primary Outcome Measures :
  1. Part A [ Time Frame: Through week 12 ]
    Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in osivelotor concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).

  2. Part B [ Time Frame: Through week 48 ]

    Co-primary endpoints: Hb response (increase from baseline of >1 g/dL) at Week 48 (based on average of Hb levels at Week 40 and Week 48) and the Annualized rate of VOC through end of Week 48.

    A VOC is defined as an acute episode of pain that:

    • Has no medically determined cause other than a vaso-occlusive event, and
    • Results in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), and
    • Requires parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.

    Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, priapism, and dactylitis that meet the requirements listed above will be included in this co-primary endpoint.


  3. Part C [ Time Frame: Through Week 6 ]

    Assess the PK AUC0-24 after multiple dose administration of osivelotor in whole blood and plasma.

    • Cmax and Cmin
    • Accumulation ratios based on Cmax and AUC0-24. -% Hb occupancy

  4. Part C [ Time Frame: Through Week 2 ]
    Assess the PK AUC0-last, AUC0-inf, and Cmax after single dose of osivelotor in whole blood and plasma.


Eligibility Criteria
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Ages Eligible for Study:   6 Months to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05431088


Contacts
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Contact: Pfizer Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
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Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Additional Information:

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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT05431088    
Other Study ID Numbers: GBT021601-021
C5351004 ( Other Identifier: Alias Study Number )
2023-508766-14-00 ( Registry Identifier: CTIS (EU) )
First Posted: June 24, 2022    Key Record Dates
Last Update Posted: April 8, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn