Overview
Sponsor-declared trial summary
multifocal spasticity of the upper and lower limb due to cerebral palsy
to investigate the efficacy of IncobotulinumtoxinA in the treatment of both BoNT-A naïve and pretreated children for upper and lower limb spasticity using a dose titration approach over three injection cycles, with a flexible observation period after injection of 12 to 20 weeks and a total duration of exposure up to 60…
Key facts
- Sponsor
- Azienda Ospedaliera Universitaria Integrata Verona
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 10 Feb 2023 → ongoing
- Decision date (initial)
- 2024-10-29
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- Merz Pharma Italia
External identifiers
- EU CT number
- 2024-518447-38-00
- EudraCT number
- 2019-003559-12
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Efficacy, Safety
to investigate the efficacy of IncobotulinumtoxinA in the treatment of both BoNT-A naïve and pretreated children for upper and lower limb spasticity using a dose titration approach over three injection cycles, with a flexible observation period after injection of 12 to 20 weeks and a total duration of exposure up to 60 weeks
Secondary objectives 1
- to investigate the safety of IncobotulinumtoxinA in the treatment of both BoNT-A naïve and pretreated children for upper and lower limb spasticity using a dose titration approach over three injection cycles, with a flexible observation period after injection of 12 to 20 weeks and a total duration of exposure up to 60 weeks
Conditions and MedDRA coding
multifocal spasticity of the upper and lower limb due to cerebral palsy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10028335 | Muscle spasticity | 100000004852 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 8
- Children (female or male) aged 2 to 17 years
- Patients with upper and lower limb spasticity due to cerebral palsy
- Gross Motor Function Classification System (GMFCS) from Level II to Level V
- Selected target clinical pattern diagnosed by a qualified health care professional
- Focal spasticity scored at least 2 points on the Ashworth scale (AS) in the joints associate with the selected target clinical pattern
- Patient deemed by the investigator to require a total body dose up to 22U/kg (maximum 550U) during the study period
- In the case of children pretreated with BoNT-A, time from last injection at least 5 months
- Informed consent signed by parents or legal guardian
Exclusion criteria 8
- Participation in other trials
- BoNT-A treatment contraindicated
- Therapy with anticoagulants or other substances that could have an anticoagulant effect
- Girls of childbearing potential (defined as females post menarche)
- Presence of fixed contractures, or bony deformities of the affected limbs
- Previous treatment of spastic muscles with nerve phenolization
- Other neurological or orthopedic conditions involving the affected limbs
- institutionalized patients
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Changes in Ashworth scale values between the injection cycle baseline visits and the control ones of respective injection cycle (single interventional effect)
Secondary endpoints 10
- Efficacy endpoint: Scala di Ashworth
- REsistance to PAssive movement Scale (REPAS)
- The GMFCS
- The visual analog scale (VAS)
- The Goal Attainment scale (GAS)
- The Global Assessment of Efficacy
- Safety endpoints: Values of blood pressure, heart rate, and respiratory rate
- Values of Body Weight
- adverse events
- The Global Assessment of Tolerability
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
XEOMIN, 100 unità, polvere per soluzione iniettabile
PRD1975292 · Product
- Active substance
- Clostridium Botulinum Neurotoxin Type a (150KD), Free of Complexing Proteins
- Substance synonyms
- IncobotulinumtoxinA, NT 201, Botulinum toxin type A (150 kD), free from complexing proteins
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAMUSCULAR USE
- Max daily dose
- 550 U unit(s)
- Max total dose
- 550 U unit(s)
- Max treatment duration
- 40 Week(s)
- Authorisation status
- Authorised
- ATC code
- M03AX01 — BOTULINUM TOXIN
- Marketing authorisation
- 038232017
- MA holder
- MERZ PHARMACEUTICALS GMBH
- MA country
- Italy
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Azienda Ospedaliera Universitaria Integrata Verona
- Sponsor organisation
- Azienda Ospedaliera Universitaria Integrata Verona
- Address
- Piazzale Ludovico Antonio Scuro 10
- City
- Verona
- Postcode
- 37134
- Country
- Italy
Scientific contact point
- Organisation
- Azienda Ospedaliera Universitaria Integrata Verona
- Contact name
- Nicola Smania
Public contact point
- Organisation
- Azienda Ospedaliera Universitaria Integrata Verona
- Contact name
- Nicola Smania
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Ongoing, recruiting | 30 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Italy | 2023-02-10 | 2023-02-10 |
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.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocollo_2019-003559-12_INCIPIT_v4_p | 4 |
| Recruitment arrangements (for publication) | BLANK DOCUMENT_new CTR | 1 |
| Subject information and informed consent form (for publication) | L1_Letter for GP_v2_p | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF minor_v1_p | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents_v2_p | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF privacy_v2_p | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Xeomin | 1 |
| Synopsis of the protocol (for publication) | BLANK DOCUMENT_RA or EC under CTD | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-02 | Italy | Acceptable 2024-10-24
|
2024-10-29 |