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Hemoglobin Modifiers

Voxelotor for Sickle Cell Disease (HOPE-KIDS Trial)

Phase 2

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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.

Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0thal)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to week 48

Awards & highlights

No Placebo-Only Group

Summary

This trial consists of four parts, each with different goals. Part A is to study how voxelotor works in the body in children with sickle cell disease. Part B is to study how voxelotor works in the body in adolescents with sickle cell disease. Part C is to study how voxelotor affects kids with sickle cell disease. Part D is to study how voxelotor affects infants with sickle cell disease.

Who is the study for?

This trial is for children with Sickle Cell Disease. Different age groups are eligible: 6-17 years for Part A, 12-17 years for Part B, 4-17 years for Part C, and 6 months to <4 years for Part D. Participants need stable hemoglobin levels and those on hydroxyurea must have a consistent dose prior to the study. Children who've had recent medical crises or require chronic transfusion therapy cannot join.

What is being tested?

The trial tests Voxelotor's effects in pediatric patients with Sickle Cell Disease across four parts: single-dose PK (Part A), multiple-dose safety/efficacy (Parts B & C), and hematological effects in very young children (Part D). It aims to understand how the drug works in different age groups and dosing schedules.

What are the potential side effects?

While specific side effects of Voxelotor in this trial aren't listed, common ones from previous studies include headache, diarrhea, abdominal pain, nausea, fatigue, rash and fever. Side effects can vary based on individual reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My hemoglobin level is 10.5 g/dL or less for certain study parts, and if I'm 12-17, my TCD velocity is ≥ 140 cm/sec.

Select...

I have either HbSS or HbS β0thal.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part A: Pharmacokinetic profile of voxelotor including maximum concentration

Part A: Pharmacokinetic profile of voxelotor including the time taken to reach the maximum concentration

Part A: Pharmacokinetic profile of voxelotor including the total drug concentration over time

+3 more

Secondary study objectives

Part C: Time to initial Hemoglobin response

Part D: Time to initial Hemoglobin response

Side effects data

From 2022 Phase 4 trial • 25 Patients • NCT04400487

32%

Sickle cell anaemia with crisis

Acute chest syndrome

100%

80%

60%

40%

20%

Study treatment Arm

Voxelotor: SCD Related

Voxelotor: Non-SCD Related

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VoxelotorExperimental Treatment1 Intervention

Subjects 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)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Voxelotor

2023

Completed Phase 4

~90

0 / 2Eligibility criteria met