Dompé Begins Phase 2 Trial Testing Intranasal Nerve Growth Factor for Cerebral Palsy

Dompé has enrolled the first patient in a phase 2 trial testing whether intranasal nerve growth factor (NGF) can safely be given to young children with spastic cerebral palsy (CP), a condition for which no treatments currently address the underlying brain injury. The trial, known as Galassia, is testing the company's investigational cenegermin nasal spray, the same recombinant NGF molecule already approved as an eye drop for a rare corneal disease.

“Cerebral palsy is a devastating disease, and for patients and families there are still no proven treatments beyond supportive care,” Marcello Allegretti, chief scientific officer of Dompé, said in a statement.² "With our emerging intranasal NGF platform, we hope to deliver a treatment that goes beyond managing symptoms and changes the course of disease, opening the door to a better future for patients and their families.”

Investigators presented the trial's design at the 2026 Child Neurology Society (CNS) Annual Meeting in Montreal, Quebec, Canada. For context, Galassia (EU-CTR 2025-522786-29-00; protocol NGF-CP-201) is an ongoing phase 2, randomized, double-blind, vehicle-controlled, 2-part crossover study enrolling approximately 60 children ages 2 to 6 with spastic CP across 12 sites in Italy, including centers in Rome, Milan, Pisa, Brescia, and Genoa. Eligible participants span the full range of the Gross Motor Function Classification System-Expanded and Revised (GMFCS-E&R), levels I through V, and have a Modified Ashworth Scale spasticity score of 2 or higher.^1,3

Participants are randomized 1:1 to intranasal cenegermin or vehicle in the first part of the study, then cross over to the alternate treatment in the second part. Each part consists of four 28-day treatment cycles followed by 36 weeks of follow-up, for a total study duration of about 52 weeks. The primary endpoint is the incidence of treatment-emergent adverse events through week 52. Secondary endpoints include safety through week 104, along with motor function, neurodevelopmental, and clinician- and caregiver-reported outcome measures, pharmacokinetics, and immunogenicity.

Secondary outcome measures listed in the trial's registry include the Gross Motor Function Measure-66, passive range of motion, the Pediatric Evaluation of Disability Inventory Computer Adaptive Test for social and cognitive function, and the Communication Function Classification System, alongside serum NGF concentrations and standard safety monitoring including vital signs, growth, laboratory tests, electrocardiograms, and electroencephalograms.³

Galassia is actively recruiting, with its first patient enrolled at Agostino Gemelli IRCCS University Hospital Foundation in Rome and additional activation planned at IRCCS Bambino Gesù Pediatric Hospital. The trial's primary analysis is planned for week 52, once all participants complete both treatment periods, with a final analysis of longer-term safety and exploratory outcomes at week 104.^1,3

Why intranasal NGF

NGF is a neurotrophin naturally involved in the growth, survival, and repair of developing nerve cells, a role for which its discoverers, Rita Levi-Montalcini and Stanley Cohen, received the 1986 Nobel Prize in Physiology or Medicine. Dompé's intranasal formulation is designed to deliver NGF to the central nervous system non-invasively by targeting olfactory neurons and NGF-TrkA receptor signaling, bypassing the blood-brain barrier.^1,2

“The cause of CP-related disability is directly connected to NGF's natural role in the body, supporting the growth, survival, and repair of developing nerve cells,” Ahmed Enayetallah, MD, PhD, chief development officer of Dompé, said in a statement when the trial was first launched.² “With intranasal NGF, Dompé aims to address CP at its source, fundamentally reshape the disease's trajectory, and improve the life-long outlook for patients.”

The company's rationale draws in part on previously published case reports describing neurodevelopmental functional improvements with favorable tolerability after intranasal NGF administration in children with other forms of pediatric brain injury, though Galassia is the first randomized, controlled study of the approach in CP specifically.¹

Cenegermin, as cenegermin-bkbj (Oxervate), was already approved by the FDA as an ophthalmic solution for neurotrophic keratitis, a rare corneal disease, where Dompé has described it as the first recombinant human NGF therapy to reach approval. Galassia marks an early-stage effort to extend that same molecule, reformulated for intranasal delivery, into a neurodevelopmental indication with a substantially different route of administration and patient population.²

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REFERENCES
1. Romeo D, Guzzetta A, Galli J, et al. Clinical trial in progress: Galassia, CP-201, a phase 2, randomized, double-blind, vehicle-controlled study of intranasal cenegermin in pediatric spastic cerebral palsy. Abstract 19. Presented at: 2026 Child Neurology Society Annual Meeting; October 14-17, 2026; Montreal, Quebec, Canada.
2. Dompé announces first patient enrolled in new study of intranasal NGF in cerebral palsy. News release. Published March 26, 2026. Accessed October 13, 2026. https://www.businesswire.com/news/home/20260326265178/en/Domp-Announces-First-Patient-Enrolled-in-New-Study-of-Intranasal-NGF-in-Cerebral-Palsy
3. EU Clinical Trials Information System. Intranasal cenegermin in pediatric participants with spastic cerebral palsy. EU-CTR 2025-522786-29-00. Accessed October 13, 2026. https://ctis.eu/trial/2025-522786-29-00