Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN (NCT05309668)
A Phase I/II, Single-Arm, Open Label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE)
🩺 Plain-English Summary (8th-Grade Level)
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to \< 7 years with NF1 related symptomatic, inoperable PN.
Who can join: Review the eligibility criteria below.
Location: Research Site — Akron, Ohio
Age range: 1 Year to 6 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 (16 Sites)
- Research Site — Akron, Ohio
- Research Site — Philadelphia, Pennsylvania
- Research Site — Houston, Texas
- Research Site — Richmond, Virginia
- Research Site — Hamburg,
- Research Site — München,
- Research Site — Tübingen,
- Research Site — Milan,
- Research Site — Rome,
- Research Site — Nagoya,
- Research Site — Setagaya City,
- Research Site — Rotterdam,
- Research Site — Moscow,
- Research Site — Moscow,
- Research Site — Barcelona,
- Research Site — Madrid,
💰 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 Neurofibromatosis Type 1
- Natural History Study of Patients With Neurofibromatosis Type I — Bethesda, Maryland
- Natural History Study of Cutaneous Neurofibromas in People With NF1 — Baltimore, Maryland
- Neurofibromatosis Type 1 Tumor Early Detection Study — Boston, Massachusetts
- Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas — Rochester, Minnesota
- Interventions for Reading Disabilities in NF1 — Nashville, Tennessee
Explore by condition: All Neurofibromatosis Type 1 clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.