Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease (HOPE Kids 2)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04218084 |
Recruitment Status :
Recruiting
First Posted : January 6, 2020
Last Update Posted : December 24, 2020
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Condition or disease | Intervention/treatment | Phase |
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Sickle Cell Disease | Drug: Voxelotor Drug: Placebos | Phase 3 |
Expanded Access : Global Blood Therapeutics has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 224 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | Double-Blind, Placebo-Controlled |
Primary Purpose: | Treatment |
Official Title: | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease |
Actual Study Start Date : | February 3, 2020 |
Estimated Primary Completion Date : | March 2026 |
Estimated Study Completion Date : | March 2026 |

Arm | Intervention/treatment |
---|---|
Experimental: Voxelotor
Voxelotor 1500mg or equivalent daily as a tablet or as powder for oral suspension.
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Drug: Voxelotor
Participants are randomized 1:1 to receive voxelotor or placebo. |
Placebo Comparator: Placebo
Matching placebo.
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Drug: Placebos
Matching placebo. |
- TCD Measurement [ Time Frame: 24 weeks ]The change in time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow, as measured by TCD.
- Change in TCD Flow Velocity [ Time Frame: 48 and 96 weeks ]
- Conversion to Abnormal TCD Flow Velocity [ Time Frame: 24, 48, and 96 weeks ]Incidence of conversion to abnormal TCD flow velocity (≥ 200 cm/sec)
- Reversion to Normal TCD Flow Velocity [ Time Frame: 24, 48, and 96 weeks ]Incidence of reversion to normal TCD flow velocity (<170 cm/sec)
- TCD Flow Velocity Reduction [ Time Frame: 24, 48, and 96 weeks ]Proportion of participants with TCD flow velocity reduction ≥ 15 cm/sec
- Number of Participants with Change in Hemoglobin (Hb) [ Time Frame: 24, 48, and 96 weeks ]
- Change in Unconjugated Bilirubin [ Time Frame: 24, 48 and 96 weeks ]
- Change in Percent of Reticulocyte [ Time Frame: 24, 48, and 96 weeks ]
- Change in Absolute Reticulocyte [ Time Frame: 24, 48, and 96 weeks ]
- Change in Lactate Dehydrogenase (LDH) [ Time Frame: 24, 48, and 96 weeks ]

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Ages Eligible for Study: | 2 Years to 14 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female participants with Sickle Cell Anemia (SCA)
- TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥ 170 to < 200cm/sec during the Screening Period
- Hb ≥ 5.5 and ≤ 10.5 g/dL during screening
- For participants taking HU, the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the informed consent form (ICF) and/or assent form, and with no anticipated need for dose adjustments (other than weight based) or for initiation of HU for non-chronic use during the study, in the opinion of the Investigator
- Written informed parental/guardian consent and participant assent (where applicable) has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines.
Exclusion Criteria:
- Body weight < 5kg at the screening visit
- Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent (see Section 7.2.5.1 for the definition of VOC).
- More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit
- Stroke resulting in focal neurological deficit; previous silent infarcts are permitted.
- Known history or findings, including screening magnetic resonance imaging (MRI)/magnetic imaging angiography (MRA) findings, suggestive of significant cerebral vasculopathy (eg, moyamoya or significant vasculopathy)
- History of seizure disorder (History of febrile seizures is permissible if there have been no seizures within the 12 months prior to randomization).
- Has been treated with erythropoietin or other hematopoietic growth factors within 28 days of signing informed consent/assent or if, in the opinion of the Investigator, there is an anticipated need for such agents during the study
- RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion) or has received an RBC transfusion or exchange transfusion for any reason within 90 days of signing the informed consent/assent

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): NCT04218084
Contact: Margaret Tonda, PharmD | (650) 741-7761 | mtonda@gbt.com | |
Contact: Jenni Herber | (650) 826-2422 | jherber@gbt.com |

Responsible Party: | Global Blood Therapeutics |
ClinicalTrials.gov Identifier: | NCT04218084 |
Other Study ID Numbers: |
GBT440-032 |
First Posted: | January 6, 2020 Key Record Dates |
Last Update Posted: | December 24, 2020 |
Last Verified: | December 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Sickle Cell Disease Transcranial Doppler Ultrasound (TCD) |
Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia |
Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |