GBT021601 for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called GBT021601 for people with sickle cell disease, a condition where red blood cells are sickle-shaped, causing pain and other health issues. The trial aims to assess the treatment's long-term effectiveness and safety. Participants should have previously taken part in a study with this drug or a placebo and completed the treatment visit. As a Phase 2, Phase 3 trial, this study evaluates the treatment's effectiveness in a larger group and represents the final step before FDA approval, offering participants a chance to contribute to advancing care for sickle cell disease.
Will I have to stop taking my current medications?
The trial requires that you stop taking voxelotor at least 10 days before starting and crizanlizumab at least 90 days before starting. The protocol does not specify other medications, so it's best to discuss your current medications with the study team.
Is there any evidence suggesting that GBT021601 is likely to be safe for humans?
Research has shown that GBT021601 is generally well-tolerated by both healthy individuals and those with sickle cell disease. Studies indicate that patients taking GBT021601 experienced improved hemoglobin levels and healthier red blood cells. Importantly, these studies also suggest that the treatment is safe, as it did not cause many serious side effects.
In one study, participants received multiple doses of GBT021601 and tolerated it well without major issues. Another study found that even at lower doses, the drug appeared to reduce the treatment burden compared to other similar medications. This suggests that the drug might be effective without requiring large amounts.
Overall, the available evidence suggests that GBT021601 is safe for individuals with sickle cell disease. However, as with any treatment, discussing all potential risks and benefits with a healthcare provider is important.12345Why do researchers think this study treatment might be promising?
GBT021601 is unique because it targets sickle cell disease with a novel mechanism of action. Unlike standard treatments such as hydroxyurea, which increases fetal hemoglobin to reduce sickling, GBT021601 works by directly inhibiting hemoglobin polymerization, the root cause of red blood cell sickling. This targeted approach could potentially offer more effective management of the disease with fewer side effects. Researchers are excited about the possibility of GBT021601 providing a new pathway to alleviate symptoms and improve the quality of life for those living with sickle cell disease.
What evidence suggests that GBT021601 might be an effective treatment for Sickle Cell Disease?
Research has shown that GBT021601, also known as osivelotor, is a promising treatment for sickle cell disease. Studies have found that it prevents sickle hemoglobin from sticking together, a major issue in this disease. Early tests suggest that GBT021601 can improve red blood cell health and help hemoglobin levels return to normal, potentially reducing problems caused by sickle cell disease. Additionally, it was well tolerated by both healthy volunteers and people with sickle cell disease, indicating good safety.12567
Who Is on the Research Team?
Pfizer CT.gov Call Center
Principal Investigator
Pfizer
Are You a Good Fit for This Trial?
This trial is for males and females aged 6 months or older with Sickle Cell Disease who were part of a previous GBT021601 study. They must use effective contraception if sexually active, have provided consent, and not have withdrawn from the earlier study. Those who recently used voxelotor or were noncompliant are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive GBT021601 to evaluate long-term safety and efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants continue to receive GBT021601 to assess long-term safety
What Are the Treatments Tested in This Trial?
Interventions
- GBT021601
Find a Clinic Near You
Who Is Running the Clinical Trial?
Pfizer
Lead Sponsor
Albert Bourla
Pfizer
Chief Executive Officer since 2019
PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki
Patrizia Cavazzoni
Pfizer
Chief Medical Officer
MD from McGill University
Global Blood Therapeutics
Lead Sponsor