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Sickle Cell DiseasePhase 2HOPE-KIDSNCT02850406

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

A Phase 2 study testing Voxelotor in people with Sickle Cell Disease, run by Pfizer at 20 sites in 3 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years. Read more about this study

Who is it for?
Ages 6 months to 17 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
Voxelotor
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
147 participants
Where
20 sitesin 3 countries
Study dates
5 Jul 2016to 2 Oct 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0thal)
  • Age:
  • Part A - 6 to 17 years of age
  • Part B - 12 to 17 years of age
  • Part C - 4 to 17 years of age
  • Part D - 6 months to <4 years of age
Show all 13Show fewer
  • Hydroxyurea (HU) therapy:
  • Parts A, B, and C: A participant taking hydroxyurea (HU) may be enrolled if the dose has been stable for at least 3 months with no anticipated need for dose adjustment during the study and no sign of hematological toxicity.
  • Part D: A participant taking HU may be enrolled if the dose has been stable for at least 1 month. Titration to the maximum tolerated dose (MTD) is allowed during the study.
  • Hemoglobin (HB):
  • Part A - No restriction
  • Parts B, C, \& D - Hb ≤ 10.5 g/dL
  • For Part C only: Participants 12 to 17 years of age must have a TCD velocity of ≥ 140 cm/sec measured anytime during screening.

Who cannot join

  • Any one of the following requiring medical attention within 14 days of signing the Informed Consent Form (ICF):
  • Vaso-occlusive crisis (VOC)
  • Acute chest syndrome (ACS)
  • Splenic sequestration crisis
  • Dactylitis
  • Requires chronic transfusion therapy
Show all 9Show fewer
  • History of stroke or meeting criteria for primary stroke prophylaxis (history of two TCD measurements ≥ 200 cm/sec by non-imaging TCD or ≥185 cm/sec by TCDi).
  • Transfusion within 30 days prior to signing the ICF
  • Body weight <5 kg for 1 month prior to the screening visit and at the screening visit.

Trial locations and contacts

20 sites in 3 countries.

20 sites in 3 countries

United States13 sites

  • Children's Healthcare of Atlanta Scottish Rite

    Atlanta, Georgia, 30342

  • Our Lady of the Lake Children's Hospital (IP Address)

    Baton Rouge, Louisiana, 70809

  • Brentwood Clinic UCSF Benioff Children's Hospital Oakland

    Brentwood, California, 94513

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • University of Illinois at Chicago Clinical Research Center

    Chicago, Illinois, 60612

  • University Hospitals Cleveland Medical Center, Rainbow Babies & Children's Hospital

    Cleveland, Ohio, 44106

  • Brody School of Medicine at East Carolina University

    Greenville, North Carolina, 27834

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

  • Rutgers-Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 08903

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

United Kingdom4 sites

  • University College London Hospital, NHS Foundation Trust

    London, Greater London, NW1 2PG

  • Barts Health NHS Trust, The Royal London Hospital

    London, E1 1BB

  • Guy's and St Thoma's NHS Foundation Trust, Evelina London Children's Hospital

    London, SE1 7EH

  • Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital

    Manchester, M13 9WL

Other trials in United Kingdom

Lebanon3 sites

  • American University of Beirut - Medical Center

    Beirut

  • Rafik Hariri University Hospital

    Beirut

  • Nini Hospital

    Tripoli

Other trials in Lebanon

About this study

Official title: A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

This study consists of four parts, Parts A, B, C, and D.

  • Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
  • Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent participants with Sickle Cell Disease ages 12 to 17 years.
  • Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease ages 4 to 17 years.
  • Part D is a multiple dose, safety, tolerability, and PK study, which examines the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease ages 6 months to < 4 years.
Being tested
Voxelotor
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VoxelotorDrug

    • Part A: Voxelotor will be administered as oral capsules or tablets
    • Part B: Voxelotor will be administered as oral capsules or tablets
    • Part C: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension
    • Part D: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension
Study groups in NCT02850406
Study groupRoleWhat they receive
VoxelotorExperimentalSubjects to receive daily oral dosing of voxelotor according to which Part (A, B, C, or D), the subject is participating in: * Part A: Subjects to receive daily oral dosing of voxelotor for 1 day (single dose) * Part B: Subjects to receive daily oral dosing of voxelotor for up to 24 weeks (multiple dose) * Part C: Subjects to receive daily oral dosing of voxelotor for up to 48 weeks (1500mg or 1500mg equivalent dose) * Part D: Subjects to receive daily oral dosing of voxelotor for up to 48 weeks (1500mg equivalent dose)
Receives: Drug: Voxelotor

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  2. Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood

    AUC0-last was calculated using the linear/log trapezoid rule.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  3. Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood

    AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for whole blood and lambdaz=elimination rate constant.

    Measured: pre-dose, 2, 8, 24, 48, 96,168 and 336 hours post-dose

  4. Part B: Change From Baseline to Week 24 in Hemoglobin Level

    Measured: Baseline, Week 24

  5. Part C: Change From Baseline to Week 48 in Cerebral Blood Flow

    Cerebral blood flow was measured using transcranial Doppler (TCD) sonography. Change from baseline in cerebral blood flow as measured by the time-averaged mean of the maximum (TAMM) TCD velocity is reported.

    Measured: Baseline, Week 48

  6. Part D: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. TEAE was defined as an AE that emerged on or after initiation of study drug (having been absent pre-treatment), or an AE that existed pre-treatment and worsened on treatment (relative to the pre-treatment state) through 28 days after study drug discontinuation. An SAE was any AE that resulted in any of the following outcomes: death, life threatening adverse drug experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, congenital anomaly or birth defect, other important medical events. AEs were classified as SCD-related and non-SCD related.

    Measured: From start of study treatment up to 28 days after study treatment discontinuation (Up to 52 weeks)

Secondary outcomes (48)

  1. Part A: Maximum Concentration (Cmax) of Voxelotor in Plasma

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  2. Part A: Maximum Concentration (Cmax) of Voxelotor in Red Blood Cells (RBC)

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  3. Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Plasma

    AUC0-last was calculated using the linear/log trapezoid rule.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  4. Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in RBC

    AUC0-last was calculated using the linear/log trapezoid rule.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  5. Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Plasma

    AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for plasma and lambdaz is the elimination rate constant.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  6. Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in RBC

    AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  7. Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in Whole Blood

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  8. Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in Plasma

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  9. Part A: Time at Which Cmax Was Observed (Tmax) for Voxelotor in RBC

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  10. Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in Whole Blood

    T1/2 was the time measured for the drug concentration to decrease by one half.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  11. Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in Plasma

    T1/2 was the time measured for the drug concentration to decrease by one half.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  12. Part A: Terminal Elimination Half-Life (T1/2) for Voxelotor in RBC

    T1/2 was the time measured for the drug concentration to decrease by one half.

    Measured: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose

  13. Part A: Percentage Hemoglobin (Hb) Occupancy

    Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula:

    ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100.

    Measured: 15 days

  14. Part B: Percentage of Days With SCD Symptom Exacerbation During the First 24 Weeks of Treatment

    SCD symptoms were measured using the Patient Reported Outcome (PRO), Sickle Cell Disease Severity Measure (SCDSM) which was a self-administered 9-item questionnaire of SCD core symptoms including pain severity, frequency, and type, as well as fatigue and mental acuity, on a 4-point response scale that was completed daily using a handheld electronic device by the participants.

    Measured: From Day 1 up to 24 weeks

  15. Part B: Change From Baseline to Week 21 to 24 in the Sickle Cell Disease Severity Measure (SCDSM) Total Symptom Score (TSS)

    The SCDSM was a self-administered 9-item questionnaire of SCD core symptoms including pain severity, frequency, and type, as well as fatigue and mental acuity, on a 4-point response scale with a range of 0 (strongly disagree) to 4 (strongly agree) that was completed daily using a handheld electronic device by the participants. TSS was calculated as the sum of the 9-item questionnaire scores scaled to a 100-point scale with a range of 0 (no symptoms) to 100 (most severe symptoms). Baseline TSS was the average of the non-missing score during the Screening period. The average of change from baseline in SCDSM TSS score for the 4-week period (Week 21 to 24) is reported.

    Measured: Baseline, Weeks 21 to 24

  16. Part B: Percent Change From Baseline to Weeks 12 and 24 in Lactate Dehydrogenase (LDH)

    Measured: Baseline, Weeks 12 and 24

  17. Part B: Percent Change From Baseline to Weeks 12 and 24 in Indirect Bilirubin

    Measured: Baseline, Weeks 12 and 24

  18. Part B: Percent Change From Baseline to Weeks 12 and 24 in Percentage Reticulocytes

    Measured: Baseline, Weeks 12 and 24

  19. Part B: Cmax of Voxelotor in Whole Blood and Plasma

    Measured: Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24

  20. Part B: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Whole Blood and Plasma

    AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant.

    Measured: Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24

  21. Part B: Terminal Elimination Half-life of Voxelotor for Plasma and Whole Blood

    T1/2 was the time measured for the drug concentration to decrease by one half.

    Measured: Day 1 (pre-dose, 2, 8, 24 hours post-dose), pre-dose on Weeks 2, 4, 8, 12, 16, 20 and 24

  22. Part B: Accumulation Ratio (Rac) of Voxelotor for Plasma and Whole Blood

    Accumulation ratio was calculated as ratio of area under the concentration-time curve from time 0 to 24 hours (AUC0-24) at steady-state (Day 28) to AUC0-24 on Day 1.

    Measured: Day 1 (0 to 24 hours post-dose) and Day 28 (0 to 24 hours post-dose)

  23. Part B: Percentage Hemoglobin Occupancy

    Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula:

    ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100.

    Measured: Day 28

  24. Part B: Change From Baseline to Week 12 and 24 in Cerebral Blood Flow

    Cerebral blood flow was measured using TCD sonography. Change from baseline in cerebral blood flow as measured by TAMM TCD velocity is reported.

    Measured: Baseline, Weeks 12 and 24

  25. Part C: Change From Baseline to Weeks 24 and 48 in Hemoglobin Level

    Measured: Baseline, Weeks 24 and 48

  26. Part C: Percent Change From Baseline to Weeks 24 and 48 in LDH

    Measured: Baseline, Weeks 24 and 48

  27. Part C: Percent Change From Baseline to Weeks 24 and 48 in Indirect Bilirubin

    Measured: Baseline, Weeks 24 and 48

  28. Part C: Percent Change From Baseline to Weeks 24 and 48 in Percentage Reticulocytes

    Measured: Baseline, Weeks 24 and 48

  29. Part C: Change From Baseline to Week 24 in Cerebral Blood Flow

    Cerebral blood flow was measured using TCD sonography. Change from baseline in cerebral blood flow as measured by TAMM TCD velocity is reported.

    Measured: Baseline, Week 24

  30. Part C: Time to Initial Hemoglobin Response

    Time to initial Hb response was defined as the time from first dose of study treatment to the first occurrence of a change from baseline in Hb > 1 gram per deciliter (g/dL).

    Measured: From first dose of study treatment (Day 1) up to Week 48

  31. Part C: Cmax of Voxelotor for Plasma and Whole Blood

    Measured: Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48

  32. Part C: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Whole Blood and Plasma

    AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant.

    Measured: Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48

  33. Part C: Terminal Elimination Half-life (T1/2) of Voxelotor for Whole Blood and Plasma

    T1/2 was the time measured for the drug concentration to decrease by one half.

    Measured: Day 1 (15 minutes to 2 hours post-dose), pre-dose on Weeks 4, 8, 12, 16, 20, 24, 36 and 48

  34. Part C: Percentage Hemoglobin Occupancy of Voxelotor

    Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula:

    ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100.

    Measured: Day 28

  35. Part C: Percentage of Participants With Normal Transcranial Doppler (TCD) Flow Velocity at Week 48

    Normal TCD flow velocity was considered as < 170 centimeter per second (cm/sec) by non-imagining TCD or < 155 cm/sec by imaging transcranial Doppler (TCDi). Percentage of participants with normal TCD flow velocity at Week 48 by Baseline TCD group (i.e. Baseline normal TCD [<170 cm/sec] and Baseline conditional TCD [>=170 cm/sec] is reported.

    Measured: Week 48

  36. Part C: Annualized Incidence Rate of Vaso-occlusive Crisis (VOC) Events

    VOC events included preferred terms of sickle cell anaemia with crisis, acute chest syndrome, pneumonia necrotising and pneumonia. Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose.

    Measured: Up to Week 48

  37. Part C: Annualized Incidence Rate of Stroke Events

    Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose.

    Measured: Up to Week 48

  38. Part D: Cmax of Voxelotor for Plasma and Whole Blood

    Measured: Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48

  39. Part D: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor for Plasma and Whole Blood

    AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration and lambdaz is the elimination rate constant.

    Measured: Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48

  40. Part D: T1/2 of Voxelotor for Plasma and Whole Blood

    T1/2 was the time measured for the drug concentration to decrease by one half.

    Measured: Day 1 (anytime between 15 minutes to 2 hours post-dose), pre-dose on Weeks 2, 8, 12, 16, 24, 36 and 48

  41. Part D: Percentage Hemoglobin Occupancy

    Percentage hemoglobin occupancy (% Hb Occupancy) refers to the percentage of hemoglobin molecules within red blood cells that were bound to study drug. Concentration of voxelotor in whole blood and plasma was used to calculate %Hb occupancy. Percentage Hb occupancy within RBCs was estimated using the formula:

    ([Concentration of voxelotor in whole blood- {1-hematocrit}]*[Concentration of voxelotor in plasma/hematocrit])/5000*100.

    Measured: Day 28

  42. Part D: Change From Baseline to Weeks 24 and 48 in Hemoglobin Level

    Measured: Baseline, Weeks 24 and 48

  43. Part D: Percent Change From Baseline to Weeks 24 and 48 in LDH

    Measured: Baseline, Weeks 24 and 48

  44. Part D: Percent Change From Baseline to Weeks 24 and 48 in Indirect Bilirubin

    Measured: Baseline, Weeks 24 and 48

  45. Part D: Percent Change From Baseline to Weeks 24 and 48 in Reticulocytes Count

    Measured: Baseline, Weeks 24 and 48

  46. Part D: Time to Initial Hemoglobin Response

    Time to initial Hb response, defined as the time from first dose of study treatment to the first occurrence of a change from baseline in Hb > 1 g/dL.

    Measured: From first dose of study treatment (Day 1) up to Week 48

  47. Part D: Annualized Incidence Rate of VOC Events

    VOC events included preferred terms of 'Sickle cell anemia with crisis', 'Acute chest syndrome', 'Pneumonia necrotising,' and 'Pneumonia. Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose.

    Measured: Up to Week 48

  48. Part D: Annualized Incidence Rate of Stroke Events

    Annualized incidence rate was calculated as total number of events divided by total person years. Total person years=sum of participants treatment period in years, which covered the time from first dose to last dose.

    Measured: Up to Week 48

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Aug 2016 (estimated)
  2. Study start5 Jul 2016
  3. Primary completion2 Oct 2023
  4. Study completion2 Oct 2023

Why it stopped: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
147 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02850406
Sponsor's protocol ID
GBT440-007
Other IDs
C5341020
Acronym
HOPE-KIDS
Status last verified
Jul 2025
First submitted
1 Jul 2016
Last updated
15 Aug 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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.
Expanded access
Available

Publications

  • Estepp JH, Kalpatthi R, Woods G, Trompeter S, Liem RI, Sims K, Inati A, Inusa BPD, Campbell A, Piccone C, Abboud MR, Smith-Whitley K, Dixon S, Tonda M, Washington C, Griffin NM, Brown C. Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years. Pediatr Blood Cancer. 2022 Aug;69(8):e29716. doi: 10.1002/pbc.29716. Epub 2022 Apr 21.

    PubMed 35451176

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Voxelotor in people with Sickle Cell Disease, run by Pfizer at 20 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 15 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in United States, United Kingdom, Lebanon. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood (time frame: pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02850406, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2025.

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