A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease (NCT06198712)
A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: The Hospital for Sick Children — Toronto, Ontario
Age range: 6 Months to 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (18 Sites)
- The Hospital for Sick Children — Toronto, Ontario
- HCL-HOPITAL LYON SUD_Institut d'Hématologie et d'Oncologie Pédiatrique (IHOPe) — Lyon,
- APHP - Centre de Référence des Syndromes — Paris,
- Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle — Roeun,
- KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital — Siaya, Siaya County
- KEMRI-Walter-Reed Kericho — Kericho,
- Kombewa Clinical Research Centre — Kisumu,
- Ahero Clinical Trials Unit — Kisumu,
- American University of Beirut Medical center — Beirut,
- Hospital Nini — Tripoli,
- University of Nigeria Teaching Hospital (UNTH) — Ituku-Ozalla, Enugu State
- Lagos University Teaching Hospital, Lagos — Lagos,
- Aminu Kano Teaching Hospital (AKTH) — Tarauni,
- Hacettepe University pediatric hematology — Ankara,
- Acıbadem Adana Hastanesi — Seyhan,
- Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital — London,
- King's College Hospital - Alex Mowat Research Hub — London,
- Manchester Royal Infirmary_1 — Manchester,
📞 Contact the Study Coordinator
Phone: (866) 867-7178
Email: clinicaltrials@novonordisk.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Sickle Cell Disease
- Kidney Function in Sickle Cell Anemia — Chapel Hill, North Carolina
- BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease — Birmingham, Alabama
- Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease — Bethesda, Maryland
- Acupressure in Patients With Sickle Cell Disease — Irvine, California
- Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) — Chicago, Illinois
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) — Charlotte, North Carolina
Explore by condition: All Sickle Cell Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.