RightFit
RecruitingPhase 2 / Phase 3

A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD

A PHASE 2/3 RANDOMIZED, MULTICENTER STUDY OF OSIVELOTOR ADMINISTERED ORALLY TO ADULT AND ADOLESCENT PARTICIPANTS WITH SICKLE CELL DISEASE

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests osivelotor in adults and teens with sickle cell disease (SCD). It looks at how safe the drug is, how well it works, and how the body handles and responds to it.

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • For Parts A and B: Have sickle cell disease of the HbSS or HbS beta-zero type.
  • For Parts A and B: Have a hemoglobin level of 5.5 to 10.5 g/dL at screening and be stable, as judged by the study doctor. Hemoglobin is the protein in red blood cells that carries oxygen.
  • If taking hydroxyurea or L-glutamine, have taken a steady dose for at least 90 days before agreeing to join. The study doctor must not expect dose changes during the study.
  • For Part B: Be age 12 or older at screening.
  • For Part B: Have had 2 to 10 vaso-occlusive crises (VOCs) in the 12 months before screening. These are sickle cell attacks that block blood flow and can cause pain.
  • For the open-label extension (OLE), the follow-up part of the study: Have successfully completed Part B.

Who can't join

  • For Parts A and B: Have had more than 10 VOCs in the 12 months before screening.
  • For Parts A and B: Be pregnant or breastfeeding.
  • For Parts A and B: Get regular red blood cell transfusions, or have had any red blood cell transfusion within 90 days before Day 1.
  • For Parts A and B: Have been hospitalized for a sickle cell crisis or another blocked-blood-flow event within 14 days before agreeing to join, or anytime during screening.
  • For Parts A and B: Live in Sub-Saharan Africa or have moved from there within the past 6 months.
  • For the OLE: Have an ongoing serious health problem, abnormal lab result, or other safety concern from Part B that the study doctor thinks makes taking the drug riskier.
  • For the OLE: Have met the rules for permanently stopping treatment during Part B.
  • For the OLE: Have taken back permission to participate or be unable to follow study steps.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: Part A and Part B: * Male or female with SCD, HbSS and HbSB-zero * Participants with Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator. * For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator. Part B: * Participants with SCD ages 12 years and older, inclusive at screening. * Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening. OLE: \- Participants who have completed the Part B successfully will be eligible. Exclusion Criteria: Part A and Part B: * Participants who had more than 10 VOC within 12 months of screening * Female participant who is breastfeeding or pregnant * Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1 * Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or anytime during the screening period. * Participants in Sub-Saharan Africa or who have relocated from Sub-Saharan Africa within 6 months. OLE: * Have any unresolved clinically significant adverse event, laboratory abnormality, or safety finding from Part B that, in the opinion of the Investigator, increases the risk of study drug administration. * Met permanent treatment discontinuation criteria during Part B. * Have withdrawn consent or are unable to comply with study procedure

Study sites (46)

AL

CT

FL

GA

IL

  • University of Illinois at Chicago Clinical Research Center · Chicago, IL 60612More trials in Chicago
  • University of Illinois Hospital and Health Sciences System - Investigational Drug Services (IDS) · Chicago, IL 60612More trials in Chicago
  • University of Illinois Hospital and Health Sciences System · Chicago, IL 60612More trials in Chicago

LA

MI

MO

MS

NC

NJ

NY

SC

TX

VA