What is the mechanism of action of this treatment?

Hydroxyurea is a cornerstone treatment for Sickle Cell Disease (SCD) that works by increasing the production of fetal hemoglobin (Hb F), which reduces the sickling of red blood cells and decreases vaso-occlusive events. This is crucial for SCD patients as it helps to prevent painful crises and other complications like acute chest syndrome. Osivelotor, a hemoglobin modulator, functions by increasing hemoglobin levels and reducing hemolysis, thereby improving oxygen delivery and reducing the frequency of vaso-occlusive crises. These treatments are vital as they address the underlying pathophysiology of SCD, improving quality of life and reducing morbidity.

What side effects are associated with this type of intervention?

Common treatments for Sickle Cell Disease (SCD) include hemoglobin modulators like osivelotor, hydroxyurea, and red blood cell transfusions. Osivelotor, being a newer treatment, has its safety and tolerability still under evaluation. Hydroxyurea, a well-established treatment, can cause side effects such as bone marrow suppression, leading to neutropenia, thrombocytopenia, and anemia. It may also cause gastrointestinal disturbances and skin and nail changes. Red blood cell transfusions, while effective, carry risks such as iron overload, alloimmunization, and transfusion reactions. Each treatment's risks and benefits should be carefully considered in consultation with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved several treatments for Sickle Cell Disease (SCD) that focus on modifying the disease's pathogenesis. Hydroxyurea, the first FDA-approved drug for SCD, increases fetal hemoglobin production, reducing the frequency of pain crises and other complications. More recently, voxelotor, a hemoglobin modulator like osivelotor, was approved. Voxelotor works by increasing hemoglobin's affinity for oxygen, thereby reducing sickling of red blood cells. These treatments aim to alleviate symptoms and prevent complications associated with SCD.

What other types of research exist?

Promising alternatives to Osivelotor for Sickle Cell Disease include Hydroxyurea and Voxelotor. Hydroxyurea is an FDA-approved disease-modifying agent that increases fetal hemoglobin levels and reduces complications. Voxelotor, approved in 2019, inhibits hemoglobin S polymerization, improving hemoglobin levels and reducing hemolysis. Both have shown efficacy in clinical trials and are recommended based on patient-specific factors.

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GBT021601 for Sickle Cell Disease

Phase 2 & 3

Recruiting

Research Sponsored by Pfizer

Eligibility Criteria Checklist

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

Must have

Participants with SCD ages 12 to 65 years, inclusive

Male or female with SCD

Must not have

Female participant who is breastfeeding or pregnant

Participants who had more than 10 VOC within 12 months of screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through week 48

Summary

This trial is testing an oral medication called osivelotor to see if it can safely help people with sickle cell disease feel better. It will also study how the medication works in the body.

Who is the study for?

This trial is for individuals aged 12-65 with Sickle Cell Disease (SCD) who have had 2 to 10 pain episodes related to the disease in the past year. They must not be pregnant or breastfeeding, and should not need regular blood transfusions or have received one within the last 90 days.

What is being tested?

The study is testing GBT021601's safety and effectiveness in treating SCD. It will look at how the body processes the drug and its impact on health outcomes for both adults and children with stable hemoglobin levels.

What are the potential side effects?

While specific side effects of GBT021601 are not listed, common ones may include reactions at injection sites, potential liver issues, headaches, nausea, fatigue, and possible changes in blood counts.

Eligibility Criteria

Inclusion Criteria

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

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I am between 12 and 65 years old and have sickle cell disease.

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I have sickle cell disease.

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I've had 2 to 10 severe pain crises in the last year.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently pregnant or breastfeeding.

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I have had more than 10 pain crises in the last year.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and through 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

Part B

Part C

Trial Design

3Treatment groups

Experimental Treatment

Active Control

Placebo Group

Group I: Part CExperimental Treatment1 Intervention

100 mg dose in cohort C1, dose level for cohorts C2 to C4 to be determined based on emerging data

Group II: Part AActive Control1 Intervention

Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg. Participants will then receive maintenance once daily doses through Week 12.

Group III: Part BPlacebo Group1 Intervention

Following the selection of the optimal safe and effective dose from Part A of the study, Part B of the study will assess the efficacy and safety of 48 weeks of the optimal dose, compared to placebo

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Hydroxyurea is a cornerstone treatment for Sickle Cell Disease (SCD) that works by increasing the production of fetal hemoglobin (Hb F), which reduces the sickling of red blood cells and decreases vaso-occlusive events. This is crucial for SCD patients as it helps to prevent painful crises and other complications like acute chest syndrome. Osivelotor, a hemoglobin modulator, functions by increasing hemoglobin levels and reducing hemolysis, thereby improving oxygen delivery and reducing the frequency of vaso-occlusive crises. These treatments are vital as they address the underlying pathophysiology of SCD, improving quality of life and reducing morbidity.

Pharmacological interventions for painful sickle cell vaso-occlusive crises in adults.