Overview
Sponsor-declared trial summary
Myclonic-astatic epilepsy
The aim of this study is to monitor the individual long-term safety and efficacy of low dose Fenfluramine (minimum 0.2 mg/kg/day BID up to a maximum of 0.8 mg/kg/day BID, max 30.0 mg/day) as add-on therapy. At the discretion of the investigator, an individual, if necessary repeated, gradual adaptation of the dose to th…
Key facts
- Sponsor
- Universitaetsklinikum Schleswig-Holstein AöR, Universitaetsklinikum Schleswig-Holstein AöR
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 24 Nov 2021 → ongoing
- Decision date (initial)
- 2024-10-24
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- UCB Biopharma SRL, Belgium
External identifiers
- EU CT number
- 2024-518520-77-00
- EudraCT number
- 2020-002238-34
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
The aim of this study is to monitor the individual long-term safety and efficacy of low dose Fenfluramine (minimum 0.2 mg/kg/day BID up to a maximum of 0.8 mg/kg/day BID, max 30.0 mg/day) as add-on therapy. At the discretion of the investigator, an individual, if necessary repeated, gradual adaptation of the dose to the respective situation of the seizures is possible in this extension study.
Conditions and MedDRA coding
Myclonic-astatic epilepsy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | LLT | 10081183 | Myoclonic-astatic epilepsy | 10010331 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- Subjects is currently enrolled in FFA-MAE-study
- Subject and parents/caregiver have been informed of the nature of the study and written informed consent has been obtained from the patient and the legally responsible parents/caregiver
- Subject is between 1 (12 months) and 17 years
- In the medical opinion of the Investigator, subject must be a candidate for continued treatment for an extended period of time with Fenfluramine (i.e. subject has demonstrated a clinically meaningful benefit with Fenfluramine in the prior trial (FFA-MAE), and benefits of continued treatment outweigh potential risks)
- Clinically meaningful benefit is defined as follows: at least 50% reduction of total number of seizures (sum of GTKA, TS, AS, AB, MS) compared to baseline in FFA-MAE-study
- Subjects receives >= 1 AED in addition to Fenfluramine
Exclusion criteria 5
- Subject has a known hypersensitivity to Fenfluramine hydrochloride or other components in the study formulation
- Weight loss of 10 percent or more compared to visit 2 (first intake of IMP) of the FFA-MAE study
- Certain drugs, as listed in the prohibited food & medication section in the protocol
- Intake of any investigational medicinal product (IMP) other than Fenfluramine
- Any cardiovascular abnormality
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Efficacy Endpoint. Change of individual (per subject) number and frequency of countable seizures compared to the baseline visit of the previous FFA-MAE study (before onset of Fenfluramine treatment)
- Safety: (Serious) Adverse Events; Laboratory measurements; Vital signs; Physical examination; 12-lead electrocardiogram (ECGs); Doppler echocardiogram (ECHOs); Body weight
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Fintepla 2.2 mg/ml oral solution
PRD8612210 · Product
- Active substance
- Fenfluramine Hydrochloride
- Pharmaceutical form
- ORAL SOLUTION
- Route of administration
- ORAL
- Max daily dose
- 0.8 mg/kg milligram(s)/kilogram
- Max total dose
- 17520 mg/kg milligram(s)/kilogram
- Max treatment duration
- 60 Month(s)
- Authorisation status
- Authorised
- ATC code
- N03AX26 — -
- Marketing authorisation
- EU/1/20/1491/004
- MA holder
- UCB PHARMA S.A.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/13/1219
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- An additional label has been added to the secondary packaging
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Universitaetsklinikum Schleswig-Holstein AöR
- Sponsor organisation
- Universitaetsklinikum Schleswig-Holstein AöR
- Address
- Arnold-Heller-Strasse 3, Brunswik Brunswik
- City
- Kiel
- Postcode
- 24105
- Country
- Germany
Scientific contact point
- Organisation
- Universitaetsklinikum Schleswig-Holstein AöR
- Contact name
- Department of Children's and Adolescence Medicine II
Public contact point
- Organisation
- Universitaetsklinikum Schleswig-Holstein AöR
- Contact name
- Department of Children's and Adolescence Medicine II
Universitaetsklinikum Schleswig-Holstein AöR
- Sponsor organisation
- Universitaetsklinikum Schleswig-Holstein AöR
- Address
- Arnold-Heller-Strasse 3, Brunswik Brunswik
- City
- Kiel
- Postcode
- 24105
- Country
- Germany
Scientific contact point
- Organisation
- Universitaetsklinikum Schleswig-Holstein AöR
- Contact name
- Department of Children and Adolescent Medicine II
Public contact point
- Organisation
- Universitaetsklinikum Schleswig-Holstein AöR
- Contact name
- Department of Children and Adolescent Medicine II
Sponsor responsibilities
- Article 77 compliance
- Universitaetsklinikum Schleswig-Holstein AöR
- Contact point sponsor
- Universitaetsklinikum Schleswig-Holstein AöR
- Article 77 implementation
- Universitaetsklinikum Schleswig-Holstein AöR
Locations
1 EU/EEA country · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Authorised, recruiting | 10 | 3 |
| 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 |
|---|---|---|---|---|---|
| Germany | 2021-11-24 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 10 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_2024-518520-77-00_Protocol_P | 2.1 |
| Protocol (for publication) | D2_Protocol_2024-518520-77-00_Note | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment_arrangements_2024-518520-77-00 | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material description 2024-518520-77-00 | 1 |
| Recruitment arrangements (for publication) | Placeholder_Transition | 1 |
| Subject information and informed consent form - Extract (for publication) | L2_Other Subject info Material 2024-518520-77-00 | 1 |
| Subject information and informed consent form (for publication) | L1_2024-518520-77-00_SIS_ICF_children_7_11_P | 1.1 |
| Subject information and informed consent form (for publication) | L1_2024-518520-77-00_SIS_ICF_children_8_17_P | 1.2 |
| Subject information and informed consent form (for publication) | L1_2024-518520-77-00_SIS_ICF_parents_P | 3.0 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis_2024-518520-77-00 | 2 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-14 | Germany | Acceptable 2024-10-16
|
2024-10-24 |
| 2 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-04-15 | Germany | Acceptable 2025-05-26
|
2025-06-02 |
| 3 | SUBSTANTIAL MODIFICATION | SM-4 | 2026-04-01 | Germany | Acceptable 2026-04-14
|
2026-04-14 |