Clinical trial • Phase II • Neurology
CENEGERMIN for Spastic cerebral palsy
Phase II trial of CENEGERMIN for Spastic cerebral palsy.
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Spastic cerebral palsy
- Trial Stage
- Phase II
- Drug Modality
- Peptide/protein/enzyme
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 22-08-2025
- First CTIS Authorization Date
- 11-12-2025
Trial design
Randomised, placebo (vehicle). 'the placebo for investigational lyophilized cenegermin drug product is the same as the drug product with the exception that it does not contain cenegermin.' dose and schedule not specified in provided data.-controlled, crossover Phase II trial across 10 sites in Italy.
- Randomised
- Yes
- Comparator
- Placebo (vehicle). 'The placebo for investigational lyophilized cenegermin Drug Product is the same as the Drug Product with the exception that it does not contain cenegermin.' Dose and schedule not specified in provided data.
- Crossover
- Yes
- Target Sample Size
- 60
- Trial Duration For Participant
- 28
Eligibility
Recruits 60 paediatric patients.
- Vulnerable Population
- Participants are pediatric (ages ≥2 and ≤6). Vulnerable population selected. Informed consent required from parent/legal guardian (parent/guardian ICF available). Assent not specified in available documents. ICF documents available in Italian.
Inclusion criteria
- {"criterion_text":"- 1. Male and female participants ≥2 and ≤6 years of age.\n- 2. Confirmed diagnosis of predominantly spastic cerebral palsy with a spasticity score of ≥2 on the MAS in at least one joint\n- 3. Gross Motor Function Classification System – Expanded & Revised (GMFCS-E&R) levels I, II, III, IV, or V.\n- 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)\n- 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
- {"criterion_text":"- 1. Any progressive or unstable medical conditions.\n- 2. Recent changes in oral or injectable anti-spasticity treatments during Part 1 only.\n- 3. Use of intrathecal baclofen pump.\n- 4. Severe joint contractures or epilepsy.\n- 5. Behavioral barriers to participation.\n- 6. Systemic immunosuppressive therapy (excluding corticosteroids).\n- 7. Nasal abnormalities that may interfere with intranasal administration."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Incidence of treatment-emergent adverse events following the first administration of investigational product.","definition_or_measurement_approach":"Measured as incidence of treatment-emergent adverse events (TEAEs) following the first administration of investigational product."}
Secondary endpoints
- {"endpoint_text":"- 2. Change in the MAS score, PROM, GMFM-66 & GMFCS-E&R.\n- 4. NGF serum concentrations.\n- 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.\n- 3. Change in daily activities & Social/cognitive domains, as assessed by PEDI-CAT, & in communication function as assessed by the CFCS.","definition_or_measurement_approach":"Change in validated clinical scales and laboratory/physiological measures: MAS (Modified Ashworth Scale), PROM, GMFM-66, GMFCS-E&R for motor function; NGF serum concentrations measured by PK assays; safety endpoints include incidence of SAEs/AEs, vitals, weight/height, physical exam, labs, ECG, EEG; functional outcomes assessed by PEDI-CAT and CFCS."}
Recruitment
- Planned Sample Size
- 60
- Recruitment Window Months
- 35
- Consent Approach
- Informed consent to be obtained from parent/legal guardian (Parent/Guardian ICF available). Subject information and informed consent documents provided (files listed: L1_IT_SIS-ICF_Parent Guardian, L1_IT_SIS-ICF_SS, etc.). Assent procedures for children not specified in available documents. Documents available in Italian.
Geography
- Total Number Of Sites
- 10
- Total Number Of Participants
- 60
Italy
- Earliest CTIS Part Ii Submission Date
- 12-11-2025
- Latest Decision Or Authorization Date
- 01-04-2026
- Processing Time Days
- 140
- Number Of Sites
- 10
- Number Of Participants
- 60
Sites
- Site Name
- Azienda USL IRCCS Di Reggio Emilia
- Department Name
- Unità Riabilitazione Disabilità Gravi Età Evolutiva
- Contact Person Name
- Silvia Faccioli
- Contact Person Email
- Silvia.Faccioli@ausl.re.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- U.O.C. Neuroriabilitazione UDGEE Degenza
- Contact Person Name
- Susanna Staccioli
- Contact Person Email
- susanna.staccioli@opbg.net
- Site Name
- Associazione La Nostra Famiglia
- Department Name
- OC Riabilitazione Neuromotoria UGDE - URNA
- Contact Person Name
- Elena Carraro
- Contact Person Email
- elena.carraro@lanostrafamiglia.it
- Site Name
- IRCCS Istituto Giannina Gaslini
- Department Name
- Neuropsichiatria Infantile
- Contact Person Name
- Elisa De Grandis
- Contact Person Email
- elisadegrandis@gaslini.org
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- UOC Neuropsichiatria Infantile
- Contact Person Name
- Domenico Romeo
- Contact Person Email
- domenicomarco.romeo@policlinicogemelli.it
- Site Name
- Associazione La Nostra Famiglia
- Department Name
- Unità per le Disabilità gravi dell’età Evolutiva e Giovane Adulta
- Contact Person Name
- Antonio Trabacca
- Contact Person Email
- antonio.trabacca@lanostrafamiglia.it
- Site Name
- Associazione La Nostra Famiglia
- Department Name
- .O.C. Riabilitazione Specialistica (funzionale/infantile post-chirurgica)
- Contact Person Name
- Luigi Piccinini
- Contact Person Email
- luigi.piccinini@lanostrafamiglia.it
- Site Name
- IRCCS Fondazione Stella Maris
- Department Name
- UOC Neuropsichiatria Infantile 1
- Contact Person Name
- Andrea Guzzetta
- Contact Person Email
- a.guzzetta@fsm.unipi.it
- Site Name
- Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
- Department Name
- Neuropsichiatria dell'Infanzia e dell'Adolescenza
- Contact Person Name
- Jessica Galli
- Contact Person Email
- jessica.galli@unibs.it
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- Sezione Disordini dello Sviluppo Motorio U.O. Neurologia dello Sviluppo
- Contact Person Name
- Emanuela Pagliano
- Contact Person Email
- Emanuela.Pagliano@istituto-besta.it
Sponsor
Primary sponsor
- Full Name
- Dompe' Farmaceutici S.p.A.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Italy
Contract research organisations
- Name
- Icon Clinical Research Limited
- Responsibilities
- codes: 1,10,12,2,4,5,6,7,8
- Name
- Eresearchtechnology Inc.
- Responsibilities
- eCOA and additional duties.
- Name
- Medidata Solutions Inc.
- Responsibilities
- codes: 3, 7
Third parties
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"eCOA and additional duties.","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"codes: 3, 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Italy","full_name":"Depo-pack S.r.l.","duties_or_roles":"Secondary packaging, labelling, release and distribution.","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Istituto Biochimico Italiano Giovanni Lorenzini S.p.A.","duties_or_roles":"Production of diluent vial (WFI 0.5mL and \"methocel\" 1.0mL).","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"codes: 1,10,12,2,4,5,6,7,8","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Dompe' Farmaceutici S.p.A.","duties_or_roles":"Production of API","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Arisglobal Limited","duties_or_roles":"Vendor of the Global Safety Database","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Patheon Italia S.p.A.","duties_or_roles":"Production of lyophilized rhNGF 0.090 mg/vial and placebo.","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Cenegermin
- Active Substance
- CENEGERMIN
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRANASAL USE
- Route
- INTRANASAL USE
- Authorisation Status
- DE_BY_04_MIA_2024_0107 / aM174/2022 / aAMM126/2023
- Investigational Product Name
- The placebo for investigational lyophilized cenegermin Drug Product is the same as the Drug Product with the exception that it does not contain cenegermin.
- Modality
- Other
- Authorisation Status
- DE_BY_04_MIA_2024_0107 / aM174/2022 / aAMM126/2023
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