Haematological Disorders
Conditions
Interventions
Placebo
Sponsors
Sanofi Aventis Recherche and Development
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Participants who have been diagnosed with SCD. - Participants who have had between =2 and =10 episodes of documented clinical acute clinical VOC within 12 months of the screening events. - Participants who are either not on hydroxyurea and/or L-glutamine at the Screening Visit and does not plan to receive them during the course of the study or has received HU and/or L-glutamine for a minimum of 6 months. Participants on hydroxyurea and/or L-glutamine must have been on a stable weight-based dose level (mg/kg) for at least 3 months prior to the Screening Visit, with the intent to continue at the same weight-based dose level for the duration of the study, except for safety reasons. - Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. - For participants =10 to <18 years of age: the parent(s)/legal guardian(s) must provide written informed consent prior to any study-related procedures being performed.
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: - Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years. - Clinically relevant cardiac abnormality, in the opinion of the Investigator or electrocardiogram (ECG) findings. - Participants with history of stroke, or history of abnormal transcranial doppler. - Participants with uncontrolled or active HBV infection. - HIV infection. - A history of active or latent tuberculosis (TB) - Positive COVID-19 molecular test. - Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days and/or voxelotor (OXBRYTA®) within 30 days prior to the Screening visit. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized rate of clinical VOC [Calculated from total number of clinical VOC incidents and total number of days during the observation period] | — |
Secondary
| Measure | Time frame |
|---|---|
| Time to first clinical VOC incidence;Annualized rate of visits due to SCD-related complications as assessed by the Investigator;Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary;Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults);Change in Hb levels;Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants);Incidence of treatment emergent adverseevents (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation.;Incidence of potentially clinically significant laboratory, vital signs, and ECG abnormalities. ;Absolute number of simple and exchange blood transfusion;Number of days requiring acetaminophen, NSAID and/or short-acting opoid usage | — |
Countries
Ghana, Kenya, Tanzania
Contacts
Public ContactHeindrich Pienaar
Regional Manager MCT AFRICA
Outcome results
None listed