Your session is about to expire
← Back to Search
Voxelotor for Sickle Cell Disease (HOPE Trial)
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.
HOPE Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowHOPE Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2022 Phase 4 trial • 25 Patients • NCT04400487HOPE Trial Design
Find a Location
Who is running the clinical trial?
Media Library
- 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.I have either HbSS or HbS β0thal.I have been on a stable dose of hydroxyurea for the required time without side effects.I've had a serious sickle cell complication or needed a blood transfusion in the last month.I weigh more than 5 kg.I am within the age range specified for a part of the study.
- Group 1: Voxelotor
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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."
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."
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."
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)."
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."
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."
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."
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."
Share this study with friends
Copy Link
Messenger