Neurofibromatosis (NF) Platform Trial

GCAR and the Children’s Tumor Foundation (CTF) are collaborating to launch a platform trial that will accelerate the development of treatments for patients with neurofibromatosis (NF) – a group of rare and often debilitating diseases with high unmet medical need.

This first-of-its-kind clinical trial for patients with neurofibromatosis type 1 (NF1) was initially designed through the EU-PEARL (EU Patient-cEntric clinical tRial pLatforms) initiative. The trial will evaluate treatments for four different manifestations of NF1 including plexiform neurofibroma, cutaneous neurofibroma, optic pathway glioma, and low-grade glioma.


Annette Bakker, PhD (CTF)
Brigid Garelik, MD, MPH (CTF)
Marco Nievo, PhD (CTF)
Meredith Buxton, PhD, MPH (GCAR)


  • Randomized, open label, Phase 1/ Phase 2, multi-arm platform-basket study
  • Investigate the efficacy, safety, and pharmacodynamic activity of multiple investigational interventions in participants suffering from at least one of four different manifestations of NF1 (plexiform neurofibroma, cutaneous neurofibroma, optic pathway glioma, and low-grade glioma)
  • The primary endpoint is binary (response/non-response) — Response criteria and the time point when response/non-response is determined is defined per manifestations


  • Participants must meet all conditions outlined in the Master Protocol inclusion/exclusion criteria to be included
  • If a participant meets the eligibility criteria for a specific treatment, he/she are eligible to receive an investigational intervention for a specific manifestation
  • If a participant does not meet the eligibility criteria for a specific treatment, he/she remains in the observational pool
  • Note: If no investigational intervention is available for a specific manifestation, participants can be rescreened at a later time when investigational interventions are available

A Coalition by Collaboration: EU-PEARL, CTF, and GCAR

The strategic alliance of GCAR and CTF follows a 3-year, 26-million-euro investment from the Innovative Health Initiative (IHI), formerly called the Innovative Medicines Initiative (IMI). EU-PEARL was a public-private partnership dedicated to transforming clinical trials by making them more efficient and patient-centered. Through this partnership, leading organizations representing patients, clinicians, researchers, health authorities and industry collaborated to design the first-ever Phase 1 / Phase 2 global platform basket trials for patients with neurofibromatosis type 1 (NF1) or schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN).

CTF, one of the original collaborators on EU-PEARL, enlisted GCAR’s involvement in the NF platform trial initiatives in mid 2022 given our expertise in developing and operationalizing innovative clinical trials including platform trials and master protocols. GCAR and CTF are working together to build a patient-centric clinical trial that will rapidly and efficiently evaluate multiple investigational therapies for the treatment of NF. The NF platform trials will be built to complement CTF’s NF Preclinical Hub, a comprehensive infrastructure to accelerate the availability of clinical trial-ready treatments. The preclinical hub will provide access to a pipeline from concept to clinic, pre-negotiated master service agreements, protocols for compound testing, as well as access to testing models, data tools, libraries, and biomarkers. Additionally, the Foundation’s NF Patient Registry provides dependable access to NF patients available to participate in NF clinical trials and research studies.

With the establishment of the NF platform trials (an NF1 trial followed by a SWN trial), pharma companies are invited to explore testing of their therapies, as well as investors interested in the opportunity to fuel the progress to approved treatments.

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