ETAVOPIVAT for Sickle Cell Disease | Sickle cell anemia

Inclusion criteria

• {"criterion_text":"- 1. Patient’s parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent"}
• {"criterion_text":"- 2. Age > 6 months and < 18 years of age at time of enrollment, according to the enrolling cohort:  Cohort 1: age 12 to < 18 years (adolescents)  Cohort 2: age 6 to < 12 years  Cohort 3: age 2 to < 6 years  Cohort 4: age 6 months to < 2 years"}
• {"criterion_text":"- 3. Patient has confirmed diagnosis of SCD  Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, high-performance liquid chromatography (HPLC), or similar testing. Note that Hb electrophoresis or HPLC is performed by the local laboratory at Screening."}
• {"criterion_text":"- 4. Hemoglobin ≥ 5.5 and < 10.5 g/dL"}
• {"criterion_text":"- 5. Pediatric patients with severe SCD, as defined by at least 1 of the following:  2-15 episodes of documented VOC (defined in Section 8.4.2) within the 12 months prior to screening. Documentation must exist in the patient’s medical record prior to screening. Events based solely on patient recall without supporting documentation should not be counted towards eligibility.  Hospitalization for any SCD-related complication in the last 12 months prior to starting study treatment  Proteinuria, defined as an albumin:creatinine ratio (ACR) > 100 mg/g on 2 measures (separated by ≥ 1 month) as an indicator of early renal disease  History of a conditional TCD in the last 12 months prior to starting study treatment, but not currently being treated with chronic transfusion therapy (applicable to participants > 2 years of age). Conditional TCD is defined as a TAMMV of 170-199 cm/s by TCD or 155-184 cm/s by imaging TCD (TCDi)."}
• {"criterion_text":"- 6. For participants taking HU, the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the investigator"}
• {"criterion_text":"- 7. Patients on crizanlizumab or L-glutamine treatment at the time of consent may be eligible if they:  Have been on a stable dose for ≥ 12 months at the time of consent (ie, no changes to the dose except for changes to weight or for safety reasons)  For patients on crizanlizumab, have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent"}
• {"criterion_text":"- 8. Female patients of childbearing potential who are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and male patients who are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug."}

Exclusion criteria

• {"criterion_text":"- 1. Female who is breastfeeding or pregnant"}
• {"criterion_text":"- 10. Receipt of prior cellular based therapy (e.g., hematopoietic cell transplant, gene modification therapy)"}
• {"criterion_text":"- 2. More than 15 VOCs (as defined in Section 8.4.2) within the 12 months prior to starting study treatment that required a hospital, emergency room (ER), or clinic visit"}
• {"criterion_text":"- 3. Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study treatment"}
• {"criterion_text":"- 4. Abnormal TCD in the 12 months prior to starting study treatment"}
• {"criterion_text":"- 5. Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)"}
• {"criterion_text":"- 6. Received any blood products within 30 days of starting study treatment"}
• {"criterion_text":"- 7. Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of starting study treatment"}
• {"criterion_text":"- 8. Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study"}
• {"criterion_text":"- 9. Receipt of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study"}

Primary endpoints

• {"endpoint_text":"- • Single-dose: maximum concentration (Cmax), area under the concentration time curve (AUC)0-t, AUC0-inf","definition_or_measurement_approach":"Pharmacokinetic measures (Cmax, AUC0-t, AUC0-inf) following single dose"}
• {"endpoint_text":"- • Steady-state etavopivat plasma exposure (Cmax,ss, AUCtau,ss, Cavg,ss, Cmin,ss)","definition_or_measurement_approach":"Steady-state plasma exposure parameters (Cmax,ss, AUCtau,ss, Cavg,ss, Cmin,ss)"}
• {"endpoint_text":"- • Estimated using population PK","definition_or_measurement_approach":"Population pharmacokinetic modelling to estimate exposures"}
• {"endpoint_text":"- • Incidence of AEs, SAEs, and AEs related to etavopivat","definition_or_measurement_approach":"Safety monitoring via adverse event reporting; incidence counts of AEs and SAEs"}
• {"endpoint_text":"- • Number of premature discontinuations, dose interruptions, and dose reductions.","definition_or_measurement_approach":"Count of treatment discontinuations, dose interruptions and dose reductions during primary period"}

Secondary endpoints

• {"endpoint_text":"- • Incidence of AEs, SAEs, and AEs related to etavopivat (extension period)","definition_or_measurement_approach":"Safety monitoring via adverse event reporting during extension period"}
• {"endpoint_text":"- • Number of premature discontinuations, dose interruptions, and dose reductions (extension period)","definition_or_measurement_approach":"Count of treatment discontinuations, dose interruptions and dose reductions during extension period"}
• {"endpoint_text":"- • Hb response rate at Weeks 12 and 24 (increase of > 1 g/dL from baseline)","definition_or_measurement_approach":"Proportion of participants with Hb increase >1 g/dL at Weeks 12 and 24"}
• {"endpoint_text":"- • Change in Hb from baseline at Weeks 12 and 24","definition_or_measurement_approach":"Mean change in hemoglobin from baseline at Weeks 12 and 24"}
• {"endpoint_text":"- Incidence of VOCs during the 24-week primary treatment period","definition_or_measurement_approach":"Incidence of vaso-occlusive crises during 24-week primary period"}
• {"endpoint_text":"- o Number of VOCs","definition_or_measurement_approach":"Count of VOC events"}
• {"endpoint_text":"- o Annualized Rate of VOC","definition_or_measurement_approach":"Annualized VOC rate calculation"}
• {"endpoint_text":"- Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Scale at Weeks 12 and 24 (between 5 to 18 years of age)","definition_or_measurement_approach":"Change from baseline in PROMIS Fatigue score at Weeks 12 and 24 for participants aged 5–18"}
• {"endpoint_text":"- Change from baseline in time-averaged mean of the maximum velocity (TAMMV) by transcranial Doppler ultrasonography (TCD) (> 2 years of age)","definition_or_measurement_approach":"Change from baseline in TAMMV measured by TCD for participants >2 years"}

France

Earliest CTIS Part Ii Submission Date

12-01-2026

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

100

Number Of Sites

3

Number Of Participants

9

Primary sponsor

Full Name

Novo Nordisk A/S

Organisation Type

Pharmaceutical company

Country Of Registered Address

Denmark

Contract research organisations

Name

Icon Development Solutions LLC

Responsibilities

4

Name

Iqvia Biotech LLC

Responsibilities

1, 12, 2

Name

PPD Global Central Labs

Responsibilities

4

Name

Suvoda LLC

Responsibilities

3

Name

WCG Clinical Inc.

Responsibilities

10, 13

Third parties

• {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Charles River Laboratories Inc.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Prometrika LLC","duties_or_roles":"6","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Ascopharm GmbH","duties_or_roles":"15 (patient reimbursement support)","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Vivos Technology Limited","duties_or_roles":"10, 13","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Forma Therapeutics Inc.","duties_or_roles":"11, 13","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"1, 12, 2","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"International Drug Development Institute","duties_or_roles":"10, 13","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"10, 13","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"4","organisation_type":"Laboratory/Research/Testing facility"}