Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - Other
Status
Ended
Participants planned
76
Countries
1
Sites
1
Non-muscle invasive bladder cancer awaiting transurethral surgery
The main objective is to evaluate the safety and assess the efficacy of intravesical instillation of Alpha1H in subjects with non-muscle invasive bladder cancer
Key facts
- Sponsor
- Hamlet BioPharma AB
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- 21 May 2018 → 30 Apr 2025
- Decision date (initial)
- 2025-01-09
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-514628-17-00
- EudraCT number
- 2016-004269-14
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety
The main objective is to evaluate the safety and assess the efficacy of intravesical instillation of Alpha1H in subjects with non-muscle invasive bladder cancer
Secondary objectives 1
- The secondary objective is to further assess the efficacy of Alpha1H in subjects with non-muscle invasive bladder cancer
Conditions and MedDRA coding
Non-muscle invasive bladder cancer awaiting transurethral surgery
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- Signed and dated informed consent
- Patient with non-muscle invasive papillary bladder cancer (NMIBC) based on cystoscopy appearance, on the waiting list for TURB
- Male and female subjects, 18 years or older
- Negative pregnancy test in women of childbearing potential
- Appropriate methods of contraception in women of childbearing potential during study
- Patients should be able to keep the content of the bladder for at least one hour
Exclusion criteria 10
- Patient with a previous history of muscle invasive bladder cancer
- Patient with a history of NMIBC with an interval shorter than 6 months after previous TURB
- Previous intravesical BCG immunotherapy in the last 12 months
- Previous intravesical chemotherapy in the last 12 months
- Participants with any other cancer diagnosis within the last 5 years (except of skin basaliomas)
- Acute urinary tract infection
- Participants with prior radiotherapy or systemic chemotherapy
- Participants receiving any other investigational agent or non-marketed product one month prior to Visit 1 and during the trial
- Any concurrent illness that may render a participant ineligible or limit compliance with study requirements
- Previously enrolled in this trial
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- Adverse events (AEs); local and systemic Occurrence, intensity and relationship to the active treatment of AEs will be collected during the trial and 30 days after the last active treatment/placebo administration. Safety will also be evaluated by assessment of Vital Signs, ECG and Laboratory parameters
- Quantification of cell shedding in urine - Cell shedding into the urine will be used as a biomarker of the tumor response to the Alpha1H peptide-oleate complex. Alpha1H is expected to trigger tumor cell shedding into the urine, based on studies of HAMLET treatment, where intravesical instillations triggered tumor cell shedding in subjects with bladder cancer
- The response of the papillary tumors to Alpha1H will be characterized by in vivo imaging during examination by cystoscopy. The resected tumor will be characterized by histopathology. The Alpha1H peptideoleate complex is expected to trigger a reduction in tumor size and a change in shape, based on studies of HAMLET in patients with bladder cancer and results from the murine bladder cancer model
Secondary endpoints 6
- Histopathology scoring - Tissue changes will be quantified by histopathology, using established parameters for scoring of Grade and Stage/Invasiveness. Biopsies will be analysed by a designated study pathologist
- - Changes in tumor tissue will be characterized by immunohistochemistry for tumor markers, cell death, proliferation, and inflammation
- Tissue accumulation of Alpha1H, defined by staining with specific antibodies - Uptake of Alpha1H by tumor tissue will be quantified by staining of tissue sections with specific antibodies. Uptake will be compared to healthy tissue in individual hosts
- Tumor response to Alpha1H by gene expression analysis - Changes in cancer-associated pathways will be analysed in tumor biopsies by whole genome transcriptional profiling. Changes in cancerassociated signalling pathways will be compared between the treatment and placebo groups
- Urine cytology and apoptosis - Cells in urine will be quantified before and after Alpha1H instillation, using standard cytology techniques and parameters defining tumor grade and stage. - Apoptotic changes in the shed cells will be quantified, as well as other markers of cell death
- Proteomic analysis of markers in urine - Changes in protein markers will be quantified by comparing samples obtained before and after the instillation of Alpha1H
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11726361 · Product
- Active substance
- ALPHA1H
- Pharmaceutical form
- SOLUTION FOR INSTILLATION
- Route of administration
- INTRAVESICAL USE
- Authorisation status
- Not Authorised
- MA holder
- HAMLET PHARMA AB
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Hamlet BioPharma AB
- Sponsor organisation
- Hamlet BioPharma AB
- Address
- S Allhelgonafors., Klinikgatan 32, Lunds Allhelgonafors. Klinikgatan 32 Lunds Allhelgonafors.
- City
- Lund
- Postcode
- 222 42
- Country
- Sweden
Scientific contact point
- Organisation
- Hamlet BioPharma AB
- Contact name
- Catharina Svanborg
Public contact point
- Organisation
- Hamlet BioPharma AB
- Contact name
- Catharina Svanborg
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| Emmes Biopharma Global s.r.o. ORG-100027596
|
Prague 1, Czechia | On site monitoring, Code 10, Code 11, Code 12, Code 5, Data management, E-data capture, Code 8, Code 9 |
| Fakultni Nemocnice V Motole ORG-100012719
|
Prague, Czechia | Code 2 |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Ended | 76 | 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 |
|---|---|---|---|---|---|
| Czechia | 2018-05-21 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| Summary of Clinical Trial Results_2024-514628-17-00 SUM-131523
|
2026-04-29T16:49:02 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| Shrnuti vysledku klinickeho hodnoceni pro laickou verejnost_2024-514628-17-00 | 2026-04-29T16:50:40 | Submitted | Laypersons Summary of Results |
| Summary of Clinical Trials Results for Laypersons_2024-514628-17-00 | 2026-04-29T16:49:56 | Submitted | Laypersons Summary of Results |
Documents 9 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | Shrnuti vysledku klinickeho hodnoceni_Laicka verejnost_2024-514628-17-00 | 1 |
| Laypersons summary of results (for publication) | Summary of Clinical Trials Results_Layperson_2024-514628-17-00 | 1 |
| Protocol (for publication) | D1_Protocol_2024-514628-17-00_Redacted | 6.1 |
| Recruitment arrangements (for publication) | blank document | 1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_Extended_treatment_Redacted | 1.4 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_Follow-up_monitoring | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_GDPR_CZ_redacted | 1.0 |
| Summary of results (for publication) | Summary of Clinical Trial Results_Technical_2024-514628-17-00 | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis CZ_2024-514628-17-00 | 1.0 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-11-19 | Czechia | Acceptable 2025-01-08
|
2025-01-09 |