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Hemoglobin Modifiers

Voxelotor for Sickle Cell Disease (HOPE 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 24 and week 48
Awards & highlights

HOPE Trial 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.Check my eligibility
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.See study design
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.

HOPE Trial 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.
Select...
I have been on a stable dose of hydroxyurea for the required time without side effects.
Select...
I am within the age range specified for a part of the study.

HOPE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 24 and week 48
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 24 and week 48 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
Part A: Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Part B: Multiple Dose effect on Clinical Measures of Hemolysis
Part B: Pharmacokinetic profile of voxelotor including maximum concentration
+15 more

Side effects data

From 2022 Phase 4 trial • 25 Patients • NCT04400487
36%
Diarrhoea
24%
Nausea
24%
Headache
12%
Vomiting
8%
Abdominal pain
8%
COVID-19
4%
Skin irritation
4%
Arthritis
4%
Arthralgia
4%
Sinus tachycardia
4%
Urinary tract infection
4%
Acne
4%
Non-cardiac chest pain
4%
Gastrooesophageal reflux disease
4%
Dizziness
4%
Dyspnoea
4%
Constipation
4%
Cellulitis
4%
Neutrophil count increased
4%
Hypoxia
4%
Ovarian cyst
4%
Abdominal pain upper
4%
Fatigue
4%
Pulmonary embolism
4%
Rash
4%
Drug hypersensitivity
4%
Scrotal infection
4%
Pruritus
4%
Cough
4%
Lymphadenopathy
100%
80%
60%
40%
20%
0%
Study treatment Arm
Voxelotor: Non-SCD Related
Voxelotor: SCD Related

HOPE 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

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor
4,556 Previous Clinical Trials
10,907,535 Total Patients Enrolled
Global Blood TherapeuticsLead Sponsor
35 Previous Clinical Trials
4,895 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,467 Previous Clinical Trials
8,088,972 Total Patients Enrolled

Media Library

Voxelotor (Hemoglobin Modifiers) Clinical Trial Eligibility Overview. Trial Name: NCT02850406 — Phase 2
Sickle Cell Disease Research Study Groups: Voxelotor
Sickle Cell Disease Clinical Trial 2023: Voxelotor Highlights & Side Effects. Trial Name: NCT02850406 — Phase 2
Voxelotor (Hemoglobin Modifiers) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02850406 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How widespread is the implementation of this trial in our state?

"This research initiative is currently hosted at Children's Hospital of Pittsburgh, Ann & Robert Lurie Children's Hospital in Chicago, The Children's Hospital of Philadelphia and 6 other medical centres."

Answered by AI

Is this research initiative unique to the field?

"Global Blood Therapeutics funded the inaugural investigation of Voxelotor in 2016, which involved 155 participants. Following this initial study and its successful Phase 2 drug approval, 7 active clinical trials are now occurring across 39 cities within 14 nations."

Answered by AI

To what extent is this medical experiment being conducted with human subjects?

"Global Blood Therapeutics is responsible for the operation of this trial, which requires 155 participants that meet all necessary criteria. The Pittsburgh Children's Hospital of UPMC in Pennsylvania and Lurie Children's Hospital of Chicago in Illinois are two sites involved with the study."

Answered by AI

What criteria must potential participants satisfy to join this trial?

"For this trial, a total of 155 individuals between the ages 6 months and 17 years old with anemia or sickle cell disease are being admitted. Criteria for admittance include: gender (male/female), hemoglobin SS or S beta0 thalassemia, hydroxyurea dose must be stable for 3 months (Part A) and 1 month (Part D), haemoglobin level ≤ 10.5 g/dL, TCD velocity ≥ 140 cm/sec measured anytime during screening (applicable to Part C only)."

Answered by AI

Has the regulatory body given authorization to Voxelotor?

"Due to the phase 2 status of this medication, there is only limited evidence confirming its safety. Our team at Power has thus rated Voxelotor's safety as a 2 on our scale from 1 to 3."

Answered by AI

Do the qualifications for this clinical trial include individuals over thirty years of age?

"The requirements for this trial stipulate that those who can join must be between 6 Months to 17 years old. At present, there are 137 trials open specifically to minors and 88 geared towards senior citizens."

Answered by AI

What other investigations have been carried out related to the efficacy of Voxelotor?

"First validated in 2016 at the Royal London Hospital, voxelotor has been subject to 11 completed trials since. There are presently 7 open studies being conducted, a large portion of which originate from Pittsburgh in Pennsylvania."

Answered by AI

Is this trial still open to enrolling participants?

"Affirmative. The clinicaltrials.gov repository reflects that this medical trial, initially published on May 18th 2016, is actively recruiting patients. A total of 155 individuals will be sought out from 6 separate research sites."

Answered by AI
~13 spots leftby Jan 2025