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    Summary
    EudraCT Number:2025-000161-87
    Sponsor's Protocol Code Number:GBT440-042
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2025-06-13
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2025-000161-87
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy of Voxelotor for the Treatment of Leg Ulcers in Patients with Sickle Cell Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Resolution of Sickle Cell Leg Ulcers with Voxelotor (RESOLVE)
    A.4.1Sponsor's protocol code numberGBT440-042
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05561140
    A.5.4Other Identifiers
    Name:C5341026 Number:Alias study number
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPfizer Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPfizer
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer
    B.5.2Functional name of contact pointClinicalTrials.gov call center
    B.5.3 Address:
    B.5.3.1Street Address66 Hudson Blvd
    B.5.3.2Town/ cityNew York
    B.5.3.3Post code10001
    B.5.3.4CountryUnited States
    B.5.6E-mailClinicalTrials.gov_Inquiries@Pfizer.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Oxbryta
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Inc
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/16/1769
    D.3 Description of the IMP
    D.3.1Product nameVoxelotor
    D.3.2Product code GBT440/ PF-06759497
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVoxelotor
    D.3.9.3Other descriptive nameOxbryta
    D.3.9.4EV Substance CodeSUB190711
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    The treatment of leg ulcers in participants with sickle cell disease (SCD)
    E.1.1.1Medical condition in easily understood language
    The treatment of leg ulcers in participants with sickle cell disease (SCD)
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the effect of voxelotor and SOC compared to placebo and SOC on leg ulcer healing in participants ≥ 12 years of age with SCD, as measured by the proportion of participants achieving resolution of target ulcer(s) in each treatment group by Week 12.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the effect of voxelotor and SOC compared to placebo and SOC on: Time to resolution of target ulcer(s) , Change in total surface area of target ulcer(s) and Incidence of new ulcers

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female participants with documented diagnosis of SCD (sickle hemoglobin with 2 sickle cell genes [HbSS], sickle hemoglobin and one beta thalassemia gene [HbS/β0] thalassemia)
    - Age 12 years and older
    - At least one cutaneous ulcer on the lower extremity (leg, ankle, or dorsum of foot) that meets the following criteria:
    - Duration: ≥ 2 weeks and < 6 months at Screening, and Size: > 2 square centimeters (cm2) prior to randomization
    - Note: Participants with multiple ulcers are eligible for the study. For each participant, target ulcer(s) for evaluation in this study will be identified and tracked. To qualify as a target ulcer, the ulcer must meet the criteria above.
    - Participants, who if female and of child-bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of study drug and who if male, agree to use barrier methods of contraception from study start to 30 days after the last dose of study drug.
    - Females of child-bearing potential are required to have a negative pregnancy test before the administration of study drug.
    - Written informed consent (≥ 18 years) or parental/guardian consent and participant assent (≥ 12–17 years) per International Ethics Committee (IEC) policy and requirements, consistent with International Council for Harmonisation (ICH) guidelines.
    E.4Principal exclusion criteria
    - Target ulcer(s) healed by ≥ 25% during the SOC Run-in prior to randomization
    - Note: For participants with multiple ulcers, the participant is not eligible for the study if all target ulcers have healed by ≥ 25% during the SOC Runin.
    - Active infection/purulence at ulcer site, or exposed tendon or bone at the ulcer site, based on Investigator’s clinical judgment.
    - Current osteomyelitis at or near the ulcer site
    - Known vascular abnormalities that would preclude healing in the opinion of the Investigator (eg, pre-existing severe arterial insufficiency in the affected limb)
    - Serum albumin < 2.0 g/dL
    - Red blood cell (RBC) transfusion within 60 days of initiation of study drug
    - Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) during the study.
    - Planned elective surgery within the next 6 months.
    - Anemia due to bone marrow failure (eg, myelodysplasia)
    - Absolute reticulocyte count < 100 × 109/L
    - Screening alanine aminotransferase (ALT) > 4 × upper limit of normal (ULN)
    - Severe renal dysfunction (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 by Schwartz formula) or is on chronic dialysis.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be measured by the proportion of participants achieving resolution of the target ulcer(s), defined as skin reepithelialization confirmed at 2 consecutive study visits 2 weeks apart during the 12-week Randomized Treatment Period. To qualify as an event, ulcer re-epithelialization that is first assessed at Week 12 must be confirmed at Week 14. For participants with more than one target ulcer, all target ulcers must be confirmed resolved in order for the participant to be considered a responder.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12, Week 14
    E.5.2Secondary end point(s)
    Secondary endpoints in this study include a comparison of voxelotor and SOC versus placebo and SOC in:
    - Time (in days) to resolution of target ulcer(s) up to Week 12
    - Change from baseline in total surface area of target ulcer(s) at Week 12
    - Incidence of new ulcers by Week 12
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 12
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Nigeria
    Brazil
    Kenya
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 80
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 73
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients No
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children less than 18 years of age
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 80
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Brazil
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