Urothelial CarcinomaPhase 1/2NCT01234519
A Phase I/II Trial of AEZS-108 in Urothelial Cancer Patients Who Failed Platinum-chemotherapy
A Phase 1/2 study testing AEZS-108 and AEZS-108 at MTD in people with Urothelial Carcinoma, run by AEterna Zentaris at 2 sites in United States.
Trial summary
The purpose of this study is to determine the appropriate dose of AEZS-108 to treat patients with a tumor of the urinary system. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Urothelial Carcinoma
- What is being tested?
- AEZS-108 and AEZS-108 at MTD
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Nov 2010to Nov 2014
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologically or cytologically confirmed locally advanced or metastatic urothelial carcinoma
- Expression of LHRH receptors confirmed by immunohistochemistry on archival cancer tissue
- Measurable disease on radiological studies
- Patients with Locally advanced unresectable or metastatic urothelial carcinoma
- Documented progression on at least one prior chemotherapy regimen which must have incorporated platinum based therapy
- Left ventricular ejection fraction (EF) > 50%
- Eastern cooperative oncology group (ECOG) status of 0, 1 or 2
- Adequate bone marrow, renal and hepatic function
Who cannot join
- Prior treatment with or allergy to any components of AEZS-108
- Active second malignancies other than non-melanoma skin cancers
- Ongoing use of an LHRH agonist (or antagonist)
- Presence of an active infection or fever > 38.5 C, parenchymal brain metastases or uncontrolled intercurrent illness
- Prior exposure to anthracyclines or anthracenediones including doxorubicin, daunorubicin, and mitoxantrone
- Patients who received radiotherapy within 4 weeks of entry
- Major surgery within the last 4 weeks and minor surgery in last 7 days
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Miami Miller School of Medicine
Miami, Florida, 33136
Univerity of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Phase I/II Trial of AEZS-108 in Locally Advanced Unresectable or Metastatic Luteinizing Hormone-releasing Hormone(LHRH) Positive Urothelial Carcinoma Patients Who Failed Platinum Based Chemotherapy
The purpose of this study is to determine the appropriate dose of AEZS-108 to treat patients with a tumor of the urinary system.
Read the detailed description
AEZS-108 is an investigational drug, combining luteinizing hormone-releasing hormone (LHRH), an hormone and doxorubicin (a drug approved to treat different types of cancer).
Some tumors, such as those found in the urinary system (also called urothelial carcinomas), have LHRH hormone receptors to which the LHRH hormone part of AEZS-108 is attracted.
AEZS-108 is expected to work by accumulating mostly on the surface of cancer cells that have LHRH hormone receptors and by delivering doxorubicin more directly into the cells to kill them. This would allow the use doxorubicin at lower doses and thus would cause less toxicity.
In the first part of the study, the appropriate dose of AEZS-108 will be determined based on its side effects. The best dose will be the highest one without severe side effects.
In the second part of the study, this best dose of AEZS-108 will be given to determine its efficacy to stop the tumor from progressing.
- Conditions
- Urothelial Carcinoma
- Being tested
- AEZS-108, AEZS-108 at MTD
- Keywords
- ureter cancer, renal pelvis cancer, bladder cancer, urethra
- Medical subject headings
- Carcinoma, Transitional Cell, Ureteral Neoplasms, Urinary Bladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AEZS-108Drug
Also called ZEN-008, AN-152, D-81858
128, 160, 210 or 267 mg/m2, 2-hour intravenous (IV) infusion, Day 1 of 21-day cycles , until toxicity or progression, up to 6 cycles
AEZS-108 at MTDDrug
Also called ZEN-008, AN-152, D-81858
2-hour IV infusion, Day 1 of 21-day cycles , until toxicity or progression, up to 6 cycles
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 - Cohort 1 | Experimental | Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort).
Patients will be enrolled in cohorts of 3 at a specified AEZS-108 dose beginning with 160mg/m^2. Enrollment will be suspended until all members of a cohort have been observed for dose limiting toxicities (DLT) for a period of 3 weeks (1 cycle of AEZS-108) from initial treatment with AEZS-108. Dose escalation will proceed within each cohort according to a specific scheme where DLT is defined. Receives: Drug: AEZS-108 |
| Phase 1 - Cohort 2 | Experimental | Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). Receives: Drug: AEZS-108 at MTD |
| Phase 1 - Cohort 3 | Experimental | Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). Receives: Drug: AEZS-108 |
| Phase 1 - Cohort 4 | Experimental | Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). Receives: Drug: AEZS-108 |
| Phase 2 | Experimental | AEZS-108 at MTD to determine efficacy in up to 40 patients. Receives: Drug: AEZS-108 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD)
Toxicity per Common Terminology Criteria for Adverse Events (CTCAE).
Measured: Day 1 of each 21-day cycle
Secondary outcomes (5)
Objective tumor response
Response evaluation criteria in solid tumors (RECIST) criteria.
Measured: Within 5 days of cycle 4, then every 3 cycles
Progression-free survival (PFS)
Time elapsed from the start of treatment to the date of documented progression or death, whichever comes first.
Measured: last cycle
Pharmacokinetics (PK)
To evaluate PK parameters of a single dose AEZS-108 and explore whether the PK parameters are associated with cardiac effects as measured by electrocardiography.
Measured: cycle 1
Overall survival
Time elapsed from the start of treatment until death.
Measured: last cycle
Circulating tumor cell (CTC) levels
To quantify tumor cells and attempt to correlate their presence and response to the outcomes of this study.
Measured: last cycle
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionNov 2014
- Study completionNov 2014
Why it stopped: Poor recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urothelial CarcinomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Phase 1 : conventional 3+3 design. Phase 2: Simon's two-stage design will be utilized. In Stage I, 22 patients will be treated with the dose determined in Phase I. The study will be terminated for futility if no more than 2 patients out of the 22 patients in Stage I are responders. If 3 or more responders are observed, an additional 18 patients will be enrolled. If at least 8 out of the 40 patients are responders, the treatment will be considered worthy of further development.
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- AEterna Zentaris
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Miami, Miami VA Healthcare System, University of Miami Sylvester Comprehensive Cancer Center
- Responsible party
- Sponsor
- Investigators
- Gustavo Fernandez, MD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01234519
- Sponsor's protocol ID
- AEZS-108-046
- Other IDs
- EProst # 20091095
- Status last verified
- Mar 2013
- First submitted
- 2 Nov 2010
- Last updated
- 15 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Trial of AEZS-108 in Locally Advanced Unresectable or Metastatic Luteinizing Hormone-releasing Hormone(LHRH) Positive Urothelial Carcinoma Patients Who Failed Platinum Based Chemotherapy
- ClinicalTrials.gov ID
- NCT01234519
- Acronym / sponsor ID
- AEZS-108-046
- Conditions
- Urothelial Carcinoma
- Keywords
- ureter cancer, renal pelvis cancer, bladder cancer, urethra
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor recruitment
- Enrolment
- 12 participants (actual)
- Lead sponsor
- AEterna ZentarisIndustry (pharma, biotech, devices)
- Collaborators
- University of Miami, Miami VA Healthcare System, University of Miami Sylvester Comprehensive Cancer Center
- Study start
- Nov 2010
- Primary completion
- Nov 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 4 Nov 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the appropriate dose of AEZS-108 to treat patients with a tumor of the urinary system.
Detailed description
AEZS-108 is an investigational drug, combining luteinizing hormone-releasing hormone (LHRH), an hormone and doxorubicin (a drug approved to treat different types of cancer).
Some tumors, such as those found in the urinary system (also called urothelial carcinomas), have LHRH hormone receptors to which the LHRH hormone part of AEZS-108 is attracted.
AEZS-108 is expected to work by accumulating mostly on the surface of cancer cells that have LHRH hormone receptors and by delivering doxorubicin more directly into the cells to kill them. This would allow the use doxorubicin at lower doses and thus would cause less toxicity.
In the first part of the study, the appropriate dose of AEZS-108 will be determined based on its side effects. The best dose will be the highest one without severe side effects.
In the second part of the study, this best dose of AEZS-108 will be given to determine its efficacy to stop the tumor from progressing.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
- Model description
- Phase 1 : conventional 3+3 design. Phase 2: Simon's two-stage design will be utilized. In Stage I, 22 patients will be treated with the dose determined in Phase I. The study will be terminated for futility if no more than 2 patients out of the 22 patients in Stage I are responders. If 3 or more responders are observed, an additional 18 patients will be enrolled. If at least 8 out of the 40 patients are responders, the treatment will be considered worthy of further development.
| Arm | Type | Receives |
|---|---|---|
| Phase 1 - Cohort 1 | Experimental | Drug: AEZS-108Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). Patients will be enrolled in cohorts of 3 at a specified AEZS-108 dose beginning with 160mg/m^2. Enrollment will be suspended until all members of a cohort have been observed for dose limiting toxicities (DLT) for a period of 3 weeks (1 cycle of AEZS-108) from initial treatment with AEZS-108. Dose escalation will proceed within each cohort according to a specific scheme where DLT is defined. |
| Phase 1 - Cohort 2 | Experimental | Drug: AEZS-108 at MTDDetermination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). |
| Phase 1 - Cohort 3 | Experimental | Drug: AEZS-108Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). |
| Phase 1 - Cohort 4 | Experimental | Drug: AEZS-108Determination of maximum tolerated dose (MTD) and recommended parenteral administration dosing for AEZS-108 in 4 sequential cohorts of patients (3-6 patients/cohort). |
| Phase 2 | Experimental | Drug: AEZS-108AEZS-108 at MTD to determine efficacy in up to 40 patients. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Histologically or cytologically confirmed locally advanced or metastatic urothelial carcinoma
- Expression of LHRH receptors confirmed by immunohistochemistry on archival cancer tissue
- Measurable disease on radiological studies
- Patients with Locally advanced unresectable or metastatic urothelial carcinoma
- Documented progression on at least one prior chemotherapy regimen which must have incorporated platinum based therapy
Key exclusion criteria (7 listed)
- Prior treatment with or allergy to any components of AEZS-108
- Active second malignancies other than non-melanoma skin cancers
- Ongoing use of an LHRH agonist (or antagonist)
- Presence of an active infection or fever > 38.5 C, parenchymal brain metastases or uncontrolled intercurrent illness
- Prior exposure to anthracyclines or anthracenediones including doxorubicin, daunorubicin, and mitoxantrone
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AEZS-108Also: ZEN-008, AN-152, D-81858128, 160, 210 or 267 mg/m2, 2-hour intravenous (IV) infusion, Day 1 of 21-day cycles , until toxicity or progression, up to 6 cycles | Drug | Phase 1 - Cohort 1, Phase 1 - Cohort 3, Phase 1 - Cohort 4, Phase 2 |
| AEZS-108 at MTDAlso: ZEN-008, AN-152, D-818582-hour IV infusion, Day 1 of 21-day cycles , until toxicity or progression, up to 6 cycles | Drug | Phase 1 - Cohort 2 |
Primary outcome
Maximum tolerated dose (MTD)
Time frame: Day 1 of each 21-day cycle
Toxicity per Common Terminology Criteria for Adverse Events (CTCAE).
5 secondary outcomes
Objective tumor response
Time frame: Within 5 days of cycle 4, then every 3 cycles
Progression-free survival (PFS)
Time frame: last cycle
Pharmacokinetics (PK)
Time frame: cycle 1
Overall survival
Time frame: last cycle
Circulating tumor cell (CTC) levels
Time frame: last cycle
Results and safety
The study was stopped early (reason given: Poor recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gustavo Fernandez, MD principal investigator
University of Miami
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AEZS-108 and AEZS-108 at MTD in people with Urothelial Carcinoma, run by AEterna Zentaris at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Poor recruitment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose (MTD) (time frame: Day 1 of each 21-day cycle). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Mar 2018.