This is a study that will evaluate the safety and efficacy of TOOKAD® (padeliporfin) Vascular Targeted Photodynamic Therapy (VTP) in the Treatment of Low-Grade Upper Tract Urothelial Cancer (UTUC).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Steba Biotech S.A.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

25 Oct 2022 → ongoing

Decision date (initial)

2024-05-21

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Steba biotech, S.A.

External identifiers

EU CT number

2024-512322-28-00

EudraCT number

2020-004494-41

ClinicalTrials.gov

NCT04620239

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Others, Pharmacokinetic, Efficacy

To demonstrate the efficacy and durability of effect following TOOKAD (padeliporfin) VTP on low grade UTUC tumors in the calyces, renal pelvis and ureter

Secondary objectives 2

1. To evaluate TOOKAD (padeliporfin) VTP related safety and tolerability in the treatment of low grade UTUC tumors in the calyces, renal pelvis and ureter
2. To explore a potential link between UTUC cancer genomic markers and treatment related clinical outcomes and disease progression

Conditions and MedDRA coding

Low Grade Upper Tract Urothelial Cancer

Study design 3 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Male and female patients 18 years or older
2. Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study
3. New or recurrent low-grade, non-invasive UTUC disease
4. Biopsy-proven disease. A concurrence of the central pathology reader will be required for eligibility
5. Up to 2 biopsy-proven sites of low-grade involvement with the largest tumor (index tumor) between 5 mm and 15 mm in diameter (as measured by endoscopy), both located in the calyces, renal pelvis or the ureter of ipsilateral kidney, with an absence of high-grade cells on cytology. (Ureter involvement should be in one anatomical location with no more than 20 mm of contiguous ureteral length)
6. Karnofsky Performance Status ≥ 50%
7. Adequate organ function defined at baseline as: ▪ANC ≥1,000/ μl, ▪Platelets ≥75,000/ μl, Hb ≥9 g/dl, ▪INR ≤2, ▪Estimated glomerular filtration rate eGFR ≥30 ml/min using CKD-EPI Method, ▪Total serum bilirubin <3 mg/dL, AST/ALT ≤5× upper limit of normal

Exclusion criteria 11

1. Current high-grade or muscle invasive (>pT1) urothelial carcinoma of the bladder
2. Carcinoma in situ (CIS) current or previous in the upper urinary tract
3. History of invasive T2 or higher urothelial cancer in past 2 years
4. Participation in another clinical study involving an investigational product within 1 month before study entry
5. BCG or local chemotherapy treatment (including VEGF-targeted therapy) in the upper urinary tract within 2 months prior to inclusion
6. Systemic chemotherapy treatment (including VEGF-targeted therapy) within 2 months prior to enrollment
7. Prohibited medication that could not be adjusted or discontinued prior to study treatment
8. Patients with photosensitive skin diseases or porphyria
9. Any other medical or psychiatric co-morbidities, including decompensated heart failure, unstable angina or coronary artery disease, severe pulmonary or liver disease or current heavy smoker that, in the opinion of the study investigator, would make the patient a poor candidate for the study
10. Pregnant or breast-feeding women; Women of childbearing potential (WOCBP) must undergo a negative serum pregnancy test prior to study entry
11. Men and women of reproductive potential not willing to observe conventional and effective birth control for the duration of treatment and for 90 days following the last TOOKAD VTP treatment

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. The primary efficacy outcome is the absence of UTUC tumors in the entire ipsilateral calyces, renal pelvis and ureter on endoscopic evaluation. This outcome will be determined dichotomously as either failure or success in achieving complete response.
2. Complete Response will be defined as absence of disease based on: •absence of visual tumor on endoscopy •no evidence of tumor on biopsy (if feasible) •negative urinary cytology by instrumented collection
3. Additional information are part of the protocol (section Primary Endpoint/Estimand)

Secondary endpoints 7

1. The duration of response at the entire ipsilateral kidney will be defined as absence of disease in the entire ipsilateral calyces, renal pelvis and ureter
2. The duration of response at the Treatment Area of the ipsilateral kidney will be defined as absence of disease in the ipsilateral Treatment Area
3. Overall renal functional outcome
4. Kidney organ loss or preservation
5. Pathological evaluation of response performed in kidney tissue of patient that will undergo kidney surgical removal following at least one TOOKAD (padeliporfin) VTP treatment
6. Safety follow up based and recording of adverse events
7. Additional information are part of the protocol (section Secondary Endpoint/Estimand)

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Steba Biotech S.A.

Sponsor organisation

Steba Biotech S.A.

Address

14a Rue Des Bains

City

Luxemburg

Postcode

1212

Country

Luxembourg

Scientific contact point

Organisation

Steba Biotech S.A.

Contact name

Global Head RA&PV

Public contact point

Organisation

Steba Biotech S.A.

Contact name

Global Head RA&PV

Third parties 8

Locations

5 EU/EEA countries · 14 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 94 files

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

Application history

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