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Trial record 6 of 20 for:    Steba

ENdoluminal LIGHT ActivatED Treatment of Upper Tract Urothelial Cancer (ENLIGHTED) Study (UCM301)

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ClinicalTrials.gov Identifier: NCT04620239
Recruitment Status : Recruiting
First Posted : November 6, 2020
Last Update Posted : March 29, 2021
Sponsor:
Collaborators:
Medpace, Inc.
PrimeVigilance
ICON plc
Information provided by (Responsible Party):
Steba Biotech S.A.

Brief Summary:
This is a phase 3, open label, single arm study of TOOKAD in the treatment of Upper Tract Urothelial Carcinoma (UTUC). The ENLIGHTED study will recruit patients with low-grade upper tract urothelial carcinoma in either the kidney or the ureter. The patients will be treated with TOOKAD (padeliporfin) VTP in two phases: an Induction Treatment Phase and a Maintenance Treatment Phase and will be followed up for additional 12 months in the long term (non intervention) follow up phase.

Condition or disease Intervention/treatment Phase
Transitional Cell Cancer of Renal Pelvis and Ureter Drug: TOOKAD VTP Phase 3

Detailed Description:

Patients entered in the study will undergo an induction treatment phase consisting of 1-3 TOOKAD VTP treatments provided 4 weeks (28 +/-3 days) apart. The goal of this induction treatment phase will be to achieve Complete Response (CR) in the involved ipsilateral tract system. During this phase, patients will be treated with TOOKAD (padeliporfin) VTP to visually identified tumor sites in the calyces,in the renal pelvis and/or ureter and subsequently examined endoscopically at 28 +/- 3 days post treatment to determine whether the treatment was successful. If CR was not achieved, additional two treatments of TOOKAD (padeliporfin) VTP are permitted 28 +/- 3 days apart for a total of up to 3 treatments during the induction treatment phase. The Primary Response Evaluation (PRE) will be performed 28 +/- 3 days after the last VTP treatment, to determine if the treatment was successful at achieving CR defined as: absence of visible tumor on endoscopy, negative urinary cytology by instrumented collection, and no evidence of tumor on biopsy (if feasible). If CR was not achieved after 3 treatments with TOOKAD (padeliporfin) VTP the treatment will be considered unsuccessful and the patient will be discontinued from the treatment phases. Patients achieving CR at the induction treatment phase will be allowed into the maintenance treatment phase of the study. The patients will then be followed over a period of 12 months post PRE, to assess the duration of response and its safety, and to provide planned maintenance treatment.

Repeated maintenance VTP treatments during this period will be provided for patients who show evidence of tumor recurrence that is deemed treatable as defined by these criteria: low-grade tumors with the largest tumor (index tumor) betwen 5 mm and 15 mm in diameter, in up to 2 anatomical locations in the calyces, renal pelvis or the ureter (ureter involvement should be in one anatomical location with no more than 20 mm of contiguous ureteral length). Patients with treatable tumor recurrence post Induction Treatment Phase would be considered as no longer having 'complete response in the entire ipsilateral kidney' and time to recurrence will be recorded.

Patients completing the 12 months of the maintenance treatment phase of the study, or patients discontinued from the treatment phases after at least one VTP treatment will be followed for an additional 12 months non intervention to monitor for disease related outcomes and VTP treatment related adverse events.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Phase III, single arm, non randomized, multicenter trial
Masking: None (Open Label)
Masking Description: Open label
Primary Purpose: Treatment
Official Title: Multicenter Phase 3 Pivotal Study to Evaluate the Safety and Efficacy of TOOKAD (Padeliporfin) Vascular Targeted Photodynamic Therapy in the Treatment of Low Grade Upper Tract Urothelial Cancer
Actual Study Start Date : March 22, 2021
Estimated Primary Completion Date : July 7, 2022
Estimated Study Completion Date : July 4, 2024

Arm Intervention/treatment
Experimental: TOOKAD VTP

Patients entered in the study will undergo an induction treatment phase consisting of 1-3 TOOKAD VTP treatments provided 4 weeks (28 +/-3 days) apart.

Patients achieving CR at the induction treatment phase will be allowed into the maintenance treatment phase. The patients will be followed over a period of 12 months post PRE, to assess the duration of response and its safety, and to provide planned maintenance treatment. Repeated maintenance VTP treatments during this period will be provided for patients who show evidence of tumor recurrence that is deemed treatable as defined: Up to 2 low-grade tumors with the largest tumor (index tumor) betwenn 5 mm and 15 mm in diameter, in up to 2 anatomical locations in the calyces, renal pelvis or the ureter (with ureter involvement should be in one anatomical location with no more than 20 mm of contiguous ureteral length).

Drug: TOOKAD VTP
During treatment, placement at the target area of an optical light fiber, through the working channel of the ureteroscope. Intravenous administration of TOOKAD (padeliporfin) at the dose of 3.66 mg/kg infused over 10 minutes. Each target area will be illuminated for 10 minutes.
Other Name: WST11




Primary Outcome Measures :
  1. Number of patients with absence of UTUC tumors in the entire ipsilateral calyces, renal pelvis and ureter [ Time Frame: 28 +/- 3 days post last treatment ]

    Primary efficacy outcome is the absence of UTUC tumors in the entire ipsilateral calyces renal pelvis and ureter on endoscopic evaluation at the time of Primary Response Evaluation (PRE) (28 +/- 3 days post last treatment) during TOOKAD (padeliporfin) VTP induction treatment phase. This outcome will be determined dichotomously as either failure or success in achieving complete response.

    · 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


Secondary Outcome Measures :
  1. Duration of response at the entire ipsilateral kidney [ Time Frame: 12 months post PRE ]

    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, based on:

    • instrumented cytology
    • visually on endoscopy
    • biopsy pathology (if feasible) as will be measured at the 12 months maintenance treatment visit post PRE

  2. Duration of response at the entire ipsilateral kidney [ Time Frame: 3, 6, 9 months post PRE ]

    Duration of response at the entire ipsilateral kidney will be defined as absence of disease in the entire ipsilateral calyces, renal pelvis and ureter, based on:

    • instrumented cytology
    • visually on endoscopy
    • biopsy pathology (if feasible) as will be measured at the 3, 6, 9 months maintenance treatment visits post PRE

  3. Duration of response at the Treatment Area of the ipsilateral kidney [ Time Frame: 3, 6, 9, and 12 months post PRE ]

    Duration of response at the Treatment Area of the ipsilateral kidney will be defined as absence of disease in the ipsilateral Treatment Area, based on:

    • instrumented cytology
    • visually on endoscopy
    • biopsy pathology (if feasible) as will be measured at the 3, 6, 9, and 12 months maintenance treatment visits post PRE

  4. Overall renal function [ Time Frame: 6 and 12 months post PRE ]

    Overall renal functional outcome will be measured at the 6 and 12 months maintenance treatment visits post PRE, and will be evaluated by comparing pre-treatment and 12-month estimated glomerular filtration rate (eGFR), calculated from serum creatinine levels, using the CKD-Epi method including:

    • Absolute change in eGFR as well as categories of CKD will be utilized based on KDIGO 2012 criteria
    • Description will include change in eGFR, change in CKD stage/risk category

  5. Kidney organ loss or preservation [ Time Frame: 3, 6, 9, and 12 months post PRE ]
    Kidney organ loss or preservation will be recorded at each maintenance treatment visit post PRE at 3, 6, 9, and 12 months Maintenance Treatment visits, and will describe the reasons for organ preservation or loss. A radical nephroureterectomy, nephron-sparing surgery for UTUC or ureterectomy will be considered as organ loss.

  6. Patients with ureteral obstruction [ Time Frame: 12 months ]
    Patients with existing ureteral obstruction and/or existing ureteral stent will be permitted with radiographic evidence of pre-existing obstruction documented, to demonstrate the site and degree of obstruction with retrograde pyelography at baseline (prior to treatment) and will be repeated and recorded at 12 months maintenance treatment visit (post treatment) which will further be supported with CT Urogram results at 12 months to demonstrate the site and degree of obstruction using standard nomenclature.

  7. Long Term follow-up endpoint Duration of the response [ Time Frame: 18 and 24 months post PRE or 6 and 12 months post PRE ]

    The duration of response in the ipsilateral kidney and treatment area will be defined as absence of disease, based on:

    • instrumented cytology
    • visually on endoscopy
    • biopsy pathology (if present and performed)

  8. Long Term follow-up endpoint Kidney organ loss or preservation [ Time Frame: 18 and 24 months post PRE or 6 and 12 months post PRE ]
    Kidney organ loss or preservation will be recorded and will describe the reasons for organ preservation or loss. A radical nephroureterectomy, nephron-sparing surgery for UTUC or ureterectomy will be considered as organ loss

  9. Long Term follow-up endpoint Overall renal functional [ Time Frame: 18 and 24 months post PRE or 6 and 12 months post PRE ]
    Overall renal functional outcome will be evaluated by estimated glomerular filtration rate(eGFR) using CKD method

  10. Long Term follow-up endpoint Safety Follow up [ Time Frame: 18 and 24 months post PRE or 6 and 12 months post PRE ]
    Safety follow up based and recording of adverse events

  11. Exploratory Endpoint [ Time Frame: Baseline ]
    Tumor and cytology samples will be collected, centrally stored and later submitted for tumor genomic sequencing studies to explore tumor genomic alteration, and mutation status that may be used as biomarkers with association to treatment response, recurrence and progression events such as, grade transformation, increased stage and metastases.

  12. Pharmacokinetic Endpoint Cmax [ Time Frame: 0, 20, 40, 60, 90, 120 and 360 mn after end of injection ]
    Patients with moderate hepatic impairment, will undergo a pharmacokinetic evaluation, blood samples will be collected to evaluate Cmax

  13. Pharmacokinetic Endpoint Tmax [ Time Frame: 0, 20, 40, 60, 90, 120 and 360 mn after end of injection ]
    Patients with moderate hepatic impairment, will undergo a pharmacokinetic evaluation, blood samples will be collected to evaluate Tmax

  14. Pharmacokinetic Endpoint T1/2 [ Time Frame: 0, 20, 40, 60, 90, 120 and 360 mn after end of injection ]
    Patients with moderate hepatic impairment, will undergo a pharmacokinetic evaluation, blood samples will be collected to evaluate T1/2

  15. Pharmacokinetic Endpoint AUC [ Time Frame: 0, 20, 40, 60, 90, 120 and 360 mn after end of injection ]
    Patients with moderate hepatic impairment, will undergo a pharmacokinetic evaluation, blood samples will be collected to evaluate AUC

  16. Pharmacokinetic Endpoint CL [ Time Frame: 0, 20, 40, 60, 90, 120 and 360 mn after end of injection ]
    Patients with moderate hepatic impairment, will undergo a pharmacokinetic evaluation, blood samples will be collected to evaluate CL



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female patients 18 years or older
  • Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study
  • New or recurrent low-grade, non-invasive UTUC disease
  • Biopsy-proven disease . A concurrence of the central pathology reader will be required for eligibility.
  • Up to 2 biopsy-proven sites of low-grade involvement with the largest tumor (index tumor) between 5 mm and 15 mm in diameter (measured by endoscopy), both located in the calyces,renal pelvis or in the ureter of the 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)
  • Karnofsky Performance Status ≥ 50%
  • Adequate organ function defined at baseline as:

    • ANC ≥1,000/ μl,
    • Platelets ≥75,000/ μl, Hb ≥9 g/dl,
    • INR ≤ 2
    • Estimated glomerular giltration rate (eGFR) ≥30 ml/min (using CKD-EPI Method
    • Total serum bilirubin <3 mg/dL, AST/ALT ≤5× upper limit of normal

Exclusion Criteria:

  • Current high-grade or muscle invasive (>pT1) urothelial carcinoma of the bladder
  • Carcinoma in situ (CIS) current or previous in the upper urinary tract
  • History of invasive T2 or higher urothelial cancer in past 2 years
  • Participation in another clinical study involving an investigational product within 1 month before study entry
  • BCG or local chemotherapy treatment in the upper urinary tract within 2 months prior to inclusion
  • Systemic chemotherapy treatment within 2 months prior to enrollment
  • Prohibited medication that could not be adjusted or discontinued prior to study treatment
  • Any other medical or psychiatric co-morbidities, including decompensated heart failure, unstable angina or coronary artery disease or 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
  • Pregnant or breast-feeding women.Women of childbearing potential (WOCBP) must undergo a negative serum pregnancy test prior to study entry.
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04620239


Contacts
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Contact: Jonathan Coleman, Professor (646) 422-4432 colemanj@mskcc.org
Contact: Neal Chore, MD (843) 449-1010 nchore@gsuro.com

Locations
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United States, New York
Department of Urology, Memorial Sloan Kettering Cancer Center Not yet recruiting
New York, New York, United States, 10065
Contact: Jonathan Coleman, Professor    646-422-4432    colemanj@mskcc.org   
United States, South Carolina
Carolina Urologic Research Center Recruiting
Myrtle Beach, South Carolina, United States, 29572
Contact: Neal Shore, MD    843-449-1010    nshore@gsuro.com   
Sponsors and Collaborators
Steba Biotech S.A.
Medpace, Inc.
PrimeVigilance
ICON plc
Investigators
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Principal Investigator: Jonathan Coleman, Professor Memorial Sloan Kettering Cancer Center
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Responsible Party: Steba Biotech S.A.
ClinicalTrials.gov Identifier: NCT04620239    
Other Study ID Numbers: CLIN2001 UCM301
First Posted: November 6, 2020    Key Record Dates
Last Update Posted: March 29, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Steba Biotech S.A.:
UTUC