Proof-of-Concept Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy Study of Intranasal Cenegermin in Adult Participants with moderate to severe Traumatic Brain Injury (TBI).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dompe' Farmaceutici S.p.A.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-04-14

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: Safety, Efficacy, Others, Pharmacokinetic, Pharmacodynamic

To evaluate the safety and tolerability of intranasal cenegermin in participants with moderate to severe subacute TBI.

Secondary objectives 4

1. 1. To evaluate improvements in the global outcome of participants with moderate to severe subacute TBI following intranasal administration of cenegermin compared to vehicle.
2. 2. To evaluate the long-term safety and tolerability of intranasal cenegermin in participants with moderate to severe subacute TBI.
3. 3. To determine the systemic pharmacokinetic (PK) characteristics of intranasal cenegermin in participants with moderate to severe subacute TBI.
4. 4. To assess the immunogenicity of intranasal cenegermin in participants with moderate to severe subacute TBI.

Conditions and MedDRA coding

Subacute Moderate to Severe Traumatic Brain Injury (TBI)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1. Male and female adults 18 to 70 years of age.
2. 2. TBI resulting in hospital evaluation within the last 2-8 weeks prior to informed consent
3. 3. A clinical diagnosis of moderate to severe TBI (defined by Glasgow Coma Scale [GCS] score 3 to 12), considering the highest GCS score assessed within 24 hours following injury
4. 4. Confirmed TBI diagnosis by computed tomography (CT) assessed by the investigator
5. 5. Anticipated discharge to a rehabilitation setting within a facility or at home.

Exclusion criteria 6

1. 1. GOS-E score of 7 (lower good recovery) or 8 (upper good recovery) at time of informed consent.
2. 2. Penetrating TBI (eg, gunshot, stabs).
3. 3. Medically refractory brain edema at time of screening.
4. 4. Dependence on invasive mechanical ventilation without the ability to sustain spontaneous breathing at time of screening.
5. 5. Poor survival prognosis (investigator assessment).
6. 6. Pre-existing medical conditions which can continue to produce functional disability up to the time of injury.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Incidence of treatment-emergent serious and non-serious adverse events through 6 months following the first intranasal administration of cenegermin.

Secondary endpoints 5

1. 1. Sliding and good outcome in Glasgow Outcome Scale – Extended at month 6.
2. 2. Through 12 months following the first administration of investigational product.
3. 3. Incidence of treatment-emergent serious and non-serious adverse events, and adverse events of special interest.
4. 4. Participant incidence of study discontinuation for tolerability reasons.
5. 5. Change from baseline on vital signs, 12-lead ECG results, clinical laboratory assessments, physical examinations, brain MRI, C-SSRS and blood levels of ADA.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dompe' Farmaceutici S.p.A.

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

Francisco Martinez Torres

Public contact point

Organisation

Dompe' Farmaceutici S.p.A.

Contact name

Francisco Martinez Torres

Third parties 6

Locations

8 EU/EEA countries · 26 investigational sites

By country

Investigational sites

Results and documents

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

Documents 110 files

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

Application history

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