Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
25
Countries
1
Sites
2
papillary craniopharyngioma
To evaluate tumour response measured as reduction in tumour volume on MRI for patients with papillary craniopharyngioma on treatment with dabrafenib and trametinib.
Key facts
- Sponsor
- Region Skane
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04], Diseases [C] - Hormonal diseases [C19]
- Trial duration
- 1 Nov 2024 → ongoing
- Decision date (initial)
- 2024-10-18
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- Novartis
External identifiers
- EU CT number
- 2024-517611-67-00
- EudraCT number
- 2021-000087-30
- ClinicalTrials.gov
- NCT05525273
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Therapy
To evaluate tumour response measured as reduction in tumour volume on MRI for patients with papillary craniopharyngioma on treatment with dabrafenib and trametinib.
Secondary objectives 1
- To evaluate treatment with dabrafenib and trametinib for the following aspects: - response according to RECIST - response duration for patients who are treated without subsequent surgery - number of patients that become operable after neoadjuvant treatment - progression free survival after 1 and 2 years - quality of life during and after treatment - the effect of treatment on vision, cognition and hypothalamic function
Conditions and MedDRA coding
papillary craniopharyngioma
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 23.1 | LLT | 10084813 | Papillary craniopharyngioma | 100000004848 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- 1. Histologically verified papillary craniopharyngioma 2. BRAFmut V600E, verified immunohistochemically and with sequencing. 3. Newly diagnosed tumour or recurrence after previous surgery, where additional surgery would give serious or permanent sequelae. 4. Age over 18 years. 5. Performance status acccording to ECOG 0-2 6. Adequate organ function
Exclusion criteria 1
- 1. Ongoing treatment with prohibited drugs (strong inducers of CYP2C8 or CYP3A4). 2. Known cardiovascular disease where MEK-inhibitor is contraindicated, e g serious heart failure, increased QTc time, uncontrolled arrythmia, recent myocardial infarction, uncontrolled hypertension 3. Active bleeding 4. Thromboembolic event last 6 months 5- Known serious ocular disease 6. Surgery last 3 weeks 7. Cohort b: radiotherapy last 3 months
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- To evaluate tumour response measured as reduction in tumour volume on MRI for patients with papillary craniopharyngioma on treatment with dabrafenib and trametinib.
Secondary endpoints 1
- To evaluate treatment with dabrafenib and trametinib for the following aspects: - response according to RECIST - response duration for patients who are treated without subsequent surgery - number of patients that become operable after neoadjuvant treatment - progression free survival after 1 and 2 years - quality of life during and after treatment - the effect of treatment on vision, cognition and hypothalamic function
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
SUB45696 · Substance
- Active substance
- Dabrafenib
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 300 mg milligram(s)
- Max total dose
- 109500 mg milligram(s)
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB119776 · Substance
- Active substance
- Trametinib
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 2 mg milligram(s)
- Max total dose
- 730 mg milligram(s)
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Region Skane
- Sponsor organisation
- Region Skane
- Address
- Dockplatsen 26, Malmo S:t Petri Malmo S:t Petri
- City
- Malmo
- Postcode
- 211 74
- Country
- Sweden
Scientific contact point
- Organisation
- Region Skane
- Contact name
- Eva-Marie Erfurth
Public contact point
- Organisation
- Region Skane
- Contact name
- Eva-Marie Erfurth
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Sweden | Ongoing, recruiting | 25 | 2 |
| 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 |
|---|---|---|---|---|---|
| Sweden | 2024-11-01 | 2024-11-01 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 9 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | Studieprotokoll kraniofaryngeom 210730 | 1 |
| Protocol (for publication) | Studyprotocol craniopharyngioma 210730 | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Subject information and informed consent form (for publication) | Patientinformation 210318 | 1 |
| Subject information and informed consent form (for publication) | Samtycke till deltagande i Studien | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | CP_NPI_Mekinist_07-Aug-24_Swedish_Clean | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | CP_NPI_Tafinlar_07-Aug-24_Swedish_Clean | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis SE EU CT number | 1 |
| Synopsis of the protocol (for publication) | Synopsis svenska 220117 | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-03 | Sweden | Acceptable 2024-10-16
|
2024-10-18 |