At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Etavopivat (drug)
- How long the study runs
- Study runs about 79 months (dates as stated)
- About the drug or intervention
- Etavopivat — drug: Participants will receive oral tablets or granules of etavopivat once daily.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 6 Months to 18 Years
- Who
- All
- Number of participants
- 95
- Started
- 2023-01-12
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for sickle cell disease
- • Phase2 - 95 participants
- • The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD)
Who can take part?
- • Ages 6 Months to 18 Years
- • Diagnosed with sickle cell disease
Where?
- • London - Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
- • London - King's College Hospital - Alex Mowat Research Hub
- • Manchester - Manchester Royal Infirmary_1
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 6 Months - 18 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Type of Participant and Disease Characteristics
- ✓Patient's parent, legal guardian, or legal representative has provided documented agreement to take part and patients have...
- ✓Age greater than or equal to (≥) 6 months and lesser than (\<) 18 years of age at time of enrollment, according to...
- ✓Cohort 1: age 12 to \< 18 years (adolescents)
- ✓Cohort 2: age 6 to \< 12 years
Who cannot take part
- ✗Medical Conditions
- ✗Female who is breastfeeding or pregnant
- ✗More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room...
- ✗Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study...
- ✗Abnormal TCD in the 12 months prior to starting study treatment
See the full criteria
Where Is This Study? (3 UK sites)
Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
London SE1 7EH, United Kingdom
King's College Hospital - Alex Mowat Research Hub
London SE5 9RS, United Kingdom
Manchester Royal Infirmary_1
Manchester M13 9WL, United Kingdom
How to Get in Touch
Novo Nordisk
Sponsor contactCONTACT
