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A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease
EU CTIS ID: 2024-519746-70-01
What this study is testing
1. To compare the effect of tebapivat versus placebo on anemia and to detect a dose response for hemoglobin (Hb) response in participants with sickle cell disease (SCD).
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- ≥16 years of age at the time of providing informed assent/consent. For France ≥18 years of age at the time of providing informed consent.
- Documented diagnosis of SCD (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants).
- Hemoglobin ≥5.5 and ≤10.5 g/dL. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.
- If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before randomization. Discontinuation of hydroxyurea requires a 90-day washout before providing informed consent
- Women of childbearing potential (WOCBP) must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a highly effective method of contraception from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug; if the highly effective method of contraception is hormonal contraception, then an acceptable barrier method must also be used (see Appendix 1 for the definition of WOCBP and acceptable contraception methods). Men with partners who are WOCBP must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a condom from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug.
- Written informed assent/consent from the participant before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study
You likely can't join if
- Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or VOC is permitted. Additionally, a participant who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed consent or during the Screening Period.
- Current enrollment or past participation (within 4 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any other clinical study involving an investigational treatment or device.
- Receiving inhibitors of P-glycoprotein (P-gp) that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer) before administration of the first dose of study drug.
- >10 Sickle Cell Pain Crises (SCPC) in the 12 months before providing informed consent.
- Known allergy to tebapivat or its excipients (silicified microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, and the Opadry® II Blue film coat [polyvinyl alcohol, titanium dioxide, macrogol/polyethylene glycol, talc, FD&C blue #2/indigo carmine aluminum lake/E132]).
- Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥10 weeks before randomization.
See the full eligibility criteria
Who can join
- ≥16 years of age at the time of providing informed assent/consent. For France ≥18 years of age at the time of providing informed consent.
- Documented diagnosis of SCD (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants).
- Hemoglobin ≥5.5 and ≤10.5 g/dL. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.
- If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before randomization. Discontinuation of hydroxyurea requires a 90-day washout before providing informed consent
- Women of childbearing potential (WOCBP) must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a highly effective method of contraception from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug; if the highly effective method of contraception is hormonal contraception, then an acceptable barrier method must also be used (see Appendix 1 for the definition of WOCBP and acceptable contraception methods). Men with partners who are WOCBP must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use a condom from the time of providing informed consent throughout the study and for 28 days after the last dose of study drug.
- Written informed assent/consent from the participant before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study
- For participants under 18 years of age or under the age at which a participant is considered legally an adult per local regulations: Legally acceptable representative/parent(s) or legal guardian provides supplementary informed consent on behalf of the participant in addition to the participant’s assent. After the participant reaches the age of legal consent, if the participant is still in the clinical study, notification will be required, and a new consent form may need to be signed by participant.
What rules you out
- Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or VOC is permitted. Additionally, a participant who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed consent or during the Screening Period.
- Current enrollment or past participation (within 4 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any other clinical study involving an investigational treatment or device.
- Receiving inhibitors of P-glycoprotein (P-gp) that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer) before administration of the first dose of study drug.
- >10 Sickle Cell Pain Crises (SCPC) in the 12 months before providing informed consent.
- Known allergy to tebapivat or its excipients (silicified microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, and the Opadry® II Blue film coat [polyvinyl alcohol, titanium dioxide, macrogol/polyethylene glycol, talc, FD&C blue #2/indigo carmine aluminum lake/E132]).
- Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥10 weeks before randomization.
- Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days before providing informed consent or within 14 days before randomization. If an SCPC occurs during the Screening Period, the Screening Period may be extended with Medical Monitor approval. A hospitalization is defined as an in-patient admission to a hospital that may or may not be preceded by an emergency room or outpatient clinic visit. A visit to an emergency room that does not result in an in-patient admission does not meet the definition of hospitalization
- Receiving treatment with voxelotor, crizanlizumab, or L-glutamine within 90 days before randomization.
- Platelet count
- Receiving treatment with hematopoietic stimulating agents within 90 days before randomization.
- Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any conditioning regimen.
- History of active and uncontrolled cardiac or pulmonary disease within 6 months before randomization, including but not limited to: a. New York Heart Association Class III or IV heart failure or clinically significant dysrhythmia b. Myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism c. Heart rate–corrected QT interval using Fridericia’s method of ≥470 milliseconds for female participants and ≥450 milliseconds for male participants, except for right or left bundle branch block d. Severe pulmonary fibrosis as defined by severe hypoxia, evidence of right-sided heart failure, and radiographic pulmonary fibrosis >50% e. Severe pulmonary hypertension as defined by severe symptoms associated with hypoxia, right-sided heart failure, and oxygen indicated.
- History of any malignancy, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ. Participants must not have active disease or have received anticancer treatment ≤5 years before providing informed consent.
- Pregnant or breastfeeding.
- Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are: a. Participants deprived of liberty by court or administrative decision (eg, participants accommodated in an institution by order of an authority or court) b. Participants undergoing psychiatric care without their consent c. Participants admitted to a health or social establishment for purposes other than research d. Adult participants subject to a legal protection measure (guardian, curatorship, legal protection) e. Participants unable to express their consent.
- Hepatobiliary disorders as defined by: a. Serum aspartate aminotransferase >2.5×upper limit of normal (ULN) (unless due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase >2.5×ULN (unless due to hepatic iron deposition) b. Serum bilirubin >ULN, if the elevation is associated with clinically symptomatic choledocholithiasis, cholecystitis, biliary obstruction, or hepatocellular disease.
- Renal dysfunction as defined by an estimated glomerular filtration rate <30 mL/min/1.73 m2 by the Chronic Kidney Disease Epidemiology Collaboration creatinine equation.
- Nonfasting triglycerides >500 mg/dL (5 mmol/L).
- Active uncontrolled infection requiring systemic antimicrobial therapy.
- Positive test for hepatitis C virus (HCV) antibody (Ab) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg).
- Positive test for HIV-1 Ab or HIV-2 Ab.
- History of major surgery (including splenectomy) ≤16 weeks before providing informed consent and/or planning to undergo a major surgical procedure during the study.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- Canada
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; Canada; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.