Intranasal Cenegermin in Pediatric Participants with Spastic Cerebral Palsy.

2025-522786-29-00 Protocol NGF-CP-201 Therapeutic exploratory (Phase II) Ongoing, recruiting

Start 7 Jan 2026 · Status Ongoing, recruiting · 1 EU/EEA countries · 10 sites · Protocol NGF-CP-201

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)
Status Ongoing, recruiting
Participants planned 60
Countries 1
Sites 10

Spastic Cerebral Palsy

To evaluate the safety and tolerability of intranasal cenegermin in pediatric participants with spastic CP.

Key facts

Sponsor
Dompe' Farmaceutici S.p.A.
Participant type
Pediatric, Patients
Age range
0-17 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10]
Trial duration
7 Jan 2026 → ongoing
Decision date (initial)
2025-12-11
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes
Funding sources
Dompé Farmaceutici S.p.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Safety, Others

To evaluate the safety and tolerability of intranasal cenegermin in pediatric participants with spastic CP.

Secondary objectives 4

  1. 1. To evaluate the long-term safety and tolerability of intranasal cenegermin in pediatric participants with spastic CP.
  2. 2. To evaluate motor outcomes of pediatric participants with spastic CP treated with intranasal cenegermin compared to vehicle.
  3. 3. To evaluate neurodevelopmental function in pediatric participants with spastic CP treated with intranasal cenegermin compared to vehicle.
  4. 4. To determine the systemic pharmacokinetic (PK) characteristics of intranasal cenegermin in pediatric participants with spastic CP treated with intranasal cenegermin compared to vehicle.

Conditions and MedDRA coding

Spastic Cerebral Palsy

VersionLevelCodeTermSystem organ class
20.1 PT 10008129 Cerebral palsy 100000004850

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

  1. 1. Male and female participants ≥2 and ≤6 years of age.
  2. 2. Confirmed diagnosis of predominantly spastic cerebral palsy with a spasticity score of ≥2 on the MAS in at least one joint
  3. 3. Gross Motor Function Classification System – Expanded & Revised (GMFCS-E&R) levels I, II, III, IV, or V.
  4. 4. Body weight ≥8 kg (participants >30 kg may be included at the investigator’s judgement and upon medical monitor review with dosing modifications as needed)
  5. 5. Currently receiving physical and/or occupational therapy for at least 3 months prior to informed consent and for the duration of the study.

Exclusion criteria 7

  1. 1. Any progressive or unstable medical conditions.
  2. 2. Recent changes in oral or injectable anti-spasticity treatments during Part 1 only.
  3. 3. Use of intrathecal baclofen pump.
  4. 4. Severe joint contractures or epilepsy.
  5. 5. Behavioral barriers to participation.
  6. 6. Systemic immunosuppressive therapy (excluding corticosteroids).
  7. 7. Nasal abnormalities that may interfere with intranasal administration.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Incidence of treatment-emergent adverse events following the first administration of investigational product.

Secondary endpoints 4

  1. 2. Change in the MAS score, PROM, GMFM-66 & GMFCS-E&R.
  2. 4. NGF serum concentrations.
  3. 1.Incidence of treatment-emergent SAEs, treatment-emergent nonserious AEs, study discontinuation for tolerability reasons. Change on Vital signs, Weight and height, Physical examinations, Clinical laboratory assessments, 12-lead ECG results, EEG results.
  4. 3. Change in daily activities & Social/cognitive domains, as assessed by PEDI-CAT, & in communication function as assessed by the CFCS.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Cenegermin

PRD12642067 · Product

Active substance
Cenegermin
Pharmaceutical form
NASAL SPRAY, POWDER FOR SOLUTION
Route of administration
INTRANASAL USE
Max daily dose
0.00 µg/Kg microgram(s)/kilogram
Max total dose
0.00 µg/Kg microgram(s)/kilogram
Max treatment duration
28 Day(s)
Authorisation status
Not Authorised
MA holder
DOMPÉ FARMACEUTICI SPA
Paediatric formulation
No
Orphan designation
No

Placebo 1

The placebo for investigational lyophilized cenegermin Drug Product is the same as the Drug Product with the exception that it does not contain cenegermin.

N/A · Product

Other product name
N/A
Pharmaceutical form
N/A
ATC code
N/A — N/A
Marketing authorisation
N/A
MA holder
N/A
MA country
Iceland
Paediatric formulation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dompe' Farmaceutici S.p.A.

11 Total trials 3 Recruiting 5 Ended
Commercial
Sponsor organisation
Dompe' Farmaceutici S.p.A.
Address
Via Santa Lucia 6
City
Milan
Postcode
20122
Country
Italy

Scientific contact point

Organisation
Dompe' Farmaceutici S.p.A.
Contact name
Kristi Aleksi

Public contact point

Organisation
Dompe' Farmaceutici S.p.A.
Contact name
Kristi Aleksi

Third parties 8

OrganisationCity, countryDuties
Eresearchtechnology Inc.
ORG-100013039
 Philadelphia, United States Other
Medidata Solutions Inc.
ORG-100016256
 New York, United States Interactive response technologies (IRT), E-data capture
Depo-pack S.r.l.
ORG-100013780
 Saronno, Italy Other
Istituto Biochimico Italiano Giovanni Lorenzini S.p.A.
ORG-100001469
 Aprilia, Italy Other
Icon Clinical Research Limited
ORG-100008322
 Dublin 18, Ireland On site monitoring, Code 10, Code 12, Code 2, Laboratory analysis, Code 5, Data management, E-data capture, Code 8
Dompe' Farmaceutici S.p.A.
ORG-100001464
 L'Aquila, Italy Other
Arisglobal Limited
ORG-100051285
 Dublin, Ireland Other
Patheon Italia S.p.A.
ORG-100011736
 Ferentino, Italy Other

Locations

1 EU/EEA country · 10 investigational sites

By country

CountryMS statusPlanned subjectsSites
Italy Ongoing, recruiting 60 10
Rest of world — 0 —

Investigational sites

Italy

10 sites · Ongoing, recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
UOC Neuropsichiatria Infantile, Largo Francesco Vito 1, 00168, Rome
Ospedale Pediatrico Bambino Gesu
U.O.C. Neuroriabilitazione UDGEE Degenza, Piazza Di Sant'onofrio 4, 00165, Rome
Associazione La Nostra Famiglia
.O.C. Riabilitazione Specialistica (funzionale/infantile post-chirurgica), Via Don Luigi Monza 20, 23842, Bosisio Parini
Associazione La Nostra Famiglia
Unità per le Disabilità gravi dell’età Evolutiva e Giovane Adulta, Piazza Antonino Di Summa 13, 72100, Brindisi
Associazione La Nostra Famiglia
OC Riabilitazione Neuromotoria UGDE - URNA, Via Costa Alta 37, 31015, Conegliano
IRCCS Foundation Istituto Neurologico Carlo Besta
Sezione Disordini dello Sviluppo Motorio U.O. Neurologia dello Sviluppo, Via Giovanni Celoria 11, 20133, Milan
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Neuropsichiatria dell'Infanzia e dell'Adolescenza, Piazzale Spedali Civili 1, 25123, Brescia
Azienda USL IRCCS Di Reggio Emilia
Unità Riabilitazione Disabilità Gravi Età Evolutiva, Viale Risorgimento 80, 42123, Reggio Emilia
IRCCS Istituto Giannina Gaslini
Neuropsichiatria Infantile, Via Gerolamo Gaslini 5, 16147, Genoa
IRCCS Fondazione Stella Maris
UOC Neuropsichiatria Infantile 1, Viale Del Tirreno 341, 56128, Pisa

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Italy 2026-01-07 2026-02-23

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol_2025-522786-29-00_redacted 3.0
Recruitment arrangements (for publication) K1_IT_Recruitment Procedure N/A
Subject information and informed consent form (for publication) L1_IT_SIS-ICF_Parent Guardian_Italian_redacted 1.2
Subject information and informed consent form (for publication) L1_IT_SIS-ICF_Personal Data_Italian 1.2
Subject information and informed consent form (for publication) L1_IT_SIS-ICF_SS_Italian_redacted 1.1
Synopsis of the protocol (for publication) D1_Lay Protocol Summary_2025-522786-29-00_Italian_redacted 1.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis_2025-522786-29-00_Italian_redacted 3.0
Synopsis of the protocol (for publication) D1_Protocol Synopsis_2025-522786-29-00_redacted 3.0

Application history

3 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-08-22 Italy Acceptable
2025-12-09
 2025-12-11
2 NON SUBSTANTIAL MODIFICATION NSM-1 2025-12-17 Italy Acceptable
2025-12-09
 2025-12-17
3 SUBSTANTIAL MODIFICATION SM-3 2026-01-30 Italy Acceptable 2026-04-01