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Study of GBT021601 for Adults and Children with Sickle Cell Disease

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What is this study about?

This clinical trial is focused on studying Sickle Cell Disease, a genetic condition that affects the shape and function of red blood cells. The study will evaluate a treatment called Osivelotor, which is taken orally in the form of a tablet. The purpose of the study is to understand how Osivelotor affects patients with sickle cell disease, particularly looking at changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen throughout the body.

The study is divided into different parts. In one part, adult participants will receive osivelotor to see how it changes their hemoglobin levels over a period of time. Another part of the study will compare the effects of osivelotor to a placebo in both adult and adolescent participants, focusing on hemoglobin response and the frequency of painful episodes known as vaso-occlusive crises. Additionally, there is an open-label part of the study that will look at how osivelotor is processed in the bodies of pediatric participants, which means the participants and researchers know what treatment is being given.

Participants in the study will take the medication for a maximum of 48 weeks, and the effects will be monitored through regular check-ups and tests. The study aims to provide valuable information on the safety and effectiveness of osivelotor for treating sickle cell disease, potentially leading to improved management of this condition in the future.

1 Joining the study

Upon joining the study, eligibility is confirmed based on specific criteria related to sickle cell disease (SCD).

Participants are grouped into different parts of the study based on age and health status.

2 Part A: Initial treatment phase

Participants receive the medication osivelotor orally.

The primary goal is to observe changes in hemoglobin (Hb) levels over 12 weeks.

3 Part B: Comparative treatment phase

Participants aged 12 and older receive either osivelotor at a dose of 150 mg once daily or a placebo.

The study measures Hb response and the rate of vaso-occlusive crisis (VOC) events over 48 weeks.

4 Part C: Pediatric study phase

Pediatric participants are divided into cohorts based on age, ranging from 6 months to 18 years.

The study assesses the pharmacokinetics (PK) of osivelotor, which involves understanding how the drug is absorbed, distributed, metabolized, and excreted in the body.

5 Monitoring and assessments

Throughout the study, participants undergo regular monitoring of Hb levels, reticulocyte counts, and other health indicators.

The study also tracks any adverse events and changes in laboratory assessments, electrocardiograms (ECGs), and vital signs.

6 Completion of study

The study is expected to conclude by April 2027.

Final assessments are conducted to evaluate the overall effects of the treatment on participants.

Who Can Join the Study?

  • The participant must have Sickle Cell Disease (SCD). This means they have a specific type of blood disorder.
  • The participant’s SCD genotype must be either HbSS or HbSB. This refers to the specific genetic type of SCD they have, which can be confirmed by a lab test.
  • For Part B, the participant must be 12 years or older. Those aged 12 to under 18 will be included only after safety is checked in younger groups.
  • The participant must have had 2 to 10 VOCs in the past year. VOCs are episodes of pain caused by blocked blood flow.
  • The participant’s hemoglobin (Hb) level must be between 5.5 and 10.5 g/dL during the screening. Hemoglobin is a protein in red blood cells that carries oxygen.
  • If the participant is taking hydroxyurea or L-glutamine, their dose must have been stable for at least 90 days before joining the study. These are medications used to treat SCD.
  • For Part C, the participant’s age must fit into specific groups:
    • Cohort C1: 12 to under 18 years
    • Cohort C2: 6 to under 12 years
    • Cohort C3: 2 to under 6 years
    • Cohort C4: 6 months to under 2 years

Who Cannot Join the Study?

  • Participants who do not have Sickle Cell Disease cannot join the study. Sickle Cell Disease is a condition where red blood cells, which carry oxygen, are shaped like a sickle or crescent, causing various health issues.
  • Participants who are not within the specified age range for the study cannot participate. The study includes certain age groups, so those outside these groups are excluded.
  • Participants who are not willing or able to follow the study procedures and requirements cannot take part in the study.
  • Participants who have other medical conditions that might interfere with the study or its results cannot participate.
  • Participants who are currently taking medications that might affect the study outcomes cannot join the study.
  • Participants who are pregnant or breastfeeding cannot participate in the study.
  • Participants who have participated in another clinical trial recently may not be eligible to join this study.
  • Participants who have a history of allergic reactions to the study medication or similar medications cannot take part in the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Oncopole Claudius Regaud Toulouse France

Other Sites

Site Name City Country Status
Robert Bosch Krankenhaus GmbH Stuttgart Germany
Hospital Edouard Herriot Lyon France
Hôpital Avicenne Bobigny France
Uzxtkfowpnyosjakojsqt Eigrv Axc Essen Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not yet recruiting
01.08.2024
Germany Germany
Not yet recruiting
01.08.2024

Trial locations

Investigated drugs:

Osivelotor is a medication being studied for its effects on individuals with Sickle Cell Disease (SCD). It is taken orally and is being evaluated for its ability to improve hemoglobin levels in adults and adolescents with SCD. The study also looks at how the medication affects the frequency of vaso-occlusive crisis (VOC) events, which are painful episodes common in SCD. Additionally, the trial includes a part that examines how the body processes the medication in pediatric participants.

Investigated diseases:

Sickle Cell Disease – Sickle Cell Disease is a genetic disorder that affects the shape and function of red blood cells. Normally, red blood cells are round and flexible, allowing them to move easily through blood vessels. In Sickle Cell Disease, the red blood cells become rigid and shaped like a crescent or sickle. These sickle-shaped cells can get stuck in small blood vessels, which can slow or block blood flow and oxygen to parts of the body. This can lead to episodes of pain, known as vaso-occlusive crises, and can cause damage to organs over time. The disease is chronic and can vary in severity among individuals.

Trial ID:
2023-508766-14-00
Protocol code:
C5351004
NCT ID:
NCT05431088
Trial Phase:
Therapeutic use (Phase IV)

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