Endometrial AdenocarcinomasPhase 1NCT01970540

Study Escalating Doses of PM01183 in Combination With Fixed Doxorubicin in Patients With Specific Advanced Unresectable Solid Tumors

A Phase 1 study testing lurbinectedin (PM01183) and Doxorubicin in people with Endometrial Adenocarcinomas and Neuroendocrine Tumors, run by PharmaMar at 7 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Endometrial Adenocarcinomas, Neuroendocrine Tumors, Small-cell Lung Cancer With Less Than 2 Prior Cytotoxic-containing Lines of Therapy
What is being tested?: lurbinectedin (PM01183) and Doxorubicin
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 122 participants
Where: 7 sitesin 2 countries
Study dates: 25 May 2011to 9 Aug 2017
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Voluntarily written informed consent
Age: between 18 and 75 years (both inclusive).
Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 1. Cohort of patients with SCLC and endometrial cáncer ECOG PS ≤ 2.
Life expectancy ≥ 3 months.
Patients with a histologically/cytologically confirmed diagnosis of advanced disease of any of the following tumors:
Breast cancer

Show all 19Show fewer

Soft-tissue sarcoma
Primary bone sarcomas.
Gynecologic tumors (endometrial adenocarcinomas, epithelial ovarian cancer...)
Hepatocellular carcinoma
Gastroenteropancreatic neuroendocrine tumors
Small cell lung cancer (SCLC)
Gastric cancer
Bladder cancer
Adenocarcinoma of unknown primary site
At least three weeks since the last anticancer therapy, including radiotherapy
Adequate bone marrow, renal, hepatic, and metabolic function
Left ventricular ejection fraction (LVEF) by echocardiogram (ECHO) or multiple-gated acquisition (MUGA) scan within normal range (according to institutional standards).
Women of childbearing potential must have a negative serum pregnancy test before study entry. Both women and men must agree to use a medically acceptable method of contraception throughout the treatment period and for six weeks after discontinuation of treatment

Who cannot join

Concomitant diseases/conditions:
History or presence of unstable angina, myocardial infarction, congestive heart failure, or clinically significant valvular heart disease within last year.
Symptomatic or any uncontrolled arrhythmia
Ongoing chronic alcohol consumption, or cirrhosis
Active uncontrolled infection.
Known human immunodeficiency virus (HIV) infection.

Show all 11Show fewer

Any other major illness that, in the Investigator's judgment
Brain metastases or leptomeningeal disease involvement.
Men or women of childbearing potential who are not using an effective method of contraception
Patients who have had radiation therapy in more than 35% of the bone marrow. This criterion will not apply to cohort of patients with SCLC and endometrial cáncer.
History of previous bone marrow and/or stem cell transplantation.

Trial locations and contacts

7 sites in 2 countries.

Country

City, postcode or hospital

7 sites in 2 countries

Spain6 sites

Hospital Universitari Vall D'Hebron
Barcelona, 08035
Centro Oncológico Md Anderson International España
Madrid, 28033
Hospital Ramón Y Cajal
Madrid, 28034
Hospital Universitario Fundación Jiménez
Madrid, 28040
Hospital Universitario Madrid Sanchinarro
Madrid, 28050
Fundación Instituto Valenciano de Oncología
Valencia, 46009

Other trials in Spain

United Kingdom1 site

UCLH (University College London Hospitals)
London, WC1E 6DB

Other trials in United Kingdom

About this study

Official title: Phase I Multicenter, Open-label, Clinical and Pharmacokinetic Study of PM01183 in Combination With Doxorubicin in Non-heavily Pretreated Patients With Selected Advanced Solid Tumors

Phase I Multicenter, Open-label, Clinical and Pharmacokinetic Study of PM01183 in Combination with Fixed Doxorubicin in Non- Heavily Pretreated Patients with Selected Advanced Solid Tumors to determine the maximum tolerated dose (MTD) and the recommended dose (RD) of PM01183 in combination with doxorubicin, to characterize the safety profile and feasibility of this combination, to characterize the pharmacokinetics (PK) of this combination, to obtain preliminary information on the clinical antitumor activity,to explore the feasibility, safety and efficacy of a potential improvable dose of this combination in selected tumor types [i.e. small cell lung cancer (SCLC) and endometrial cáncer] and to evaluate the pharmacogenomics (PGx) in tumor samples of patients exposed to PM01183 and doxorubicin at the RD in order to assess potential markers of response and/or resistance.

Read the detailed description

The study has currently met its primary end point and is now recruiting patients to be treated at the RD expansion cohort of selected tumor types, specifically: endometrial adenocarcinomas, neuroendocrine tumors, and small-cell lung cancer (SCLC).

Conditions: Endometrial Adenocarcinomas, Neuroendocrine Tumors, Small-cell Lung Cancer With Less Than 2 Prior Cytotoxic-containing Lines of Therapy
Being tested: lurbinectedin (PM01183), Doxorubicin
Keywords: lurbinectedin, PM01183, tumors, cancer, Pharma Mar
Medical subject headings: Neuroendocrine Tumors, Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

lurbinectedin (PM01183)Drug
lurbinectedin (PM01183) is presented as powder for concentrate for solution for infusion with two strengths, 1-mg and 4-mg vials.
DoxorubicinDrug
Commercially available presentations of vials containing doxorubicin will be provided as appropriate.

Study groups in NCT01970540
Study group	Role	What they receive
lurbinectedin (PM01183) / doxorubicin	Experimental	Not described Receives: Drug: lurbinectedin (PM01183); Drug: Doxorubicin

What is being measured

The primary outcome is the main question the study is designed to answer.

Maximum Tolerated Dose (MTD)
A minimum of three patients will be included at each DL. If no patients experience a DLT during Cycle 1, the dose will be escalated. If one of three patients experiences a DLT, three additional patients will be included at that level. If >1 evaluable patient during dose escalation at a given DL experience a DLT during Cycle 1, that DL will be considered the MTD and dose escalation will be terminated, except if all DLTs are related to neutropenia exclusively, in which case dose escalation may be resumed as originally planned in a new cohort of patients but with compulsory primary G-CSF prophylaxis.
Measured: During the first cycle of treatment, up to 28 days
Recommended Dose (RD)
The DL immediately below the MTD, or DL4 if the MTD is not yet defined during dose escalation before the last DL (i.e., DL4) is reached, was to be initially expanded up to a minimum of nine evaluable patients. If less than three among the first nine evaluable patients treated within the expansion cohort experience a DLT during Cycle 1, this DL will be the RD.
Measured: During the first cycle of treatment, up to 28 days
Number of Participants With Dose-limiting Toxicities
DLT,dose-limiting toxicity Patients enrolled into this study were originally not allowed to receive primary G-CSF prophylaxis. However, the finding of dose-limiting febrile neutropenia in two patients treated at dose level I in the present study (see below) suggested that increasing doses of the DOX/PM01183 combination might require primary G-CSF prophylaxis to decrease the risk of febrile neutropenia. Therefore, a separate dose escalation was established to define the MTD and the RD of the DOX/PM01183 combination with compulsory primary G-CSF prophylaxis.
Measured: During the first cycle of treatment, up to 28 days

Secondary outcomes (4)

Best Overall Tumor Response
Tumor response was evaluated according to the RECIST v.1.1 for target lesions and assessed by computed tomography (CT) scans or magnetic resonance imagings (MRIs): Progressive disease (PD) is declared when there is an increase in sum of target disease ≥ 20%, stable diease (SD) when the change is > -30% and ≤ 20%, partial response (PR) when there is a decrease in sum of target disease ≥ 30%, and complete response (CR) when all lesions have disappeared or all lesions have disapeared and all nodal disease is < 10 mm each.
Measured: Tumor assessments were done every six weeks up to study completion
Duration of Response
The duration of overall response was measured from the time measurement criteria are met for complete response (CR)/ PR, whichever is first recorded, until the first date in which progressive disease is objectively documented (taking as reference for progressive disease the smallest measurements recorded since the treatment started) or the date of death. In absence of disease progression or death, the duration of response was censored on the date of last tumor evaluation.
Measured: Time from the time measurement criteria are met for complete response, whichever is first recorded, until the first date in which progressive disease is objectively documented or the date of death, assessed up to 72 months
Progression-free Survival
Progression-free survival (PFS) is defined as the time from the date of first infusion of study treatment to the date of progression or death (due to any cause). If progression or death has not occurred at the time of the analysis, the PFS was censored on the date of last tumor evaluation.
Measured: Time from the date of first administration to the date of PD or death (of any cause), whichever came first, assessed up to 72 months
Overall Survival
Overall survival (OS) is defined as the time from the date of first infusion of study treatment to the date of death (due to any cause). Patients with no documented death were censored at the last date they are known to be alive
Measured: Time from the date of first administration to the date of death (of any cause), assessed up to 72 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

25 May 20119 Aug 2017

First posted28 Oct 2013 (estimated)
Study start25 May 2011
Primary completion9 Aug 2017
Study completion9 Aug 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Endometrial AdenocarcinomasStudies 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
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 122 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01970540
Sponsor's protocol ID: PM1183-A-003-10
Status last verified: Jan 2020
First submitted: 22 Oct 2013
Last updated: 9 Mar 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Olmedo ME, Forster M, Moreno V, Lopez-Criado MP, Brana I, Flynn M, Doger B, de Miguel M, Lopez-Vilarino JA, Nunez R, Kahatt C, Cullell-Young M, Zeaiter A, Calvo E. Efficacy and safety of lurbinectedin and doxorubicin in relapsed small cell lung cancer. Results from an expansion cohort of a phase I study. Invest New Drugs. 2021 Oct;39(5):1275-1283. doi: 10.1007/s10637-020-01025-x. Epub 2021 Mar 11.
PubMed 33704620

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 9 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase I Multicenter, Open-label, Clinical and Pharmacokinetic Study of PM01183 in Combination With Doxorubicin in Non-heavily Pretreated Patients With Selected Advanced Solid Tumors
ClinicalTrials.gov ID: NCT01970540
Acronym / sponsor ID: PM1183-A-003-10
Conditions: Endometrial Adenocarcinomas, Neuroendocrine Tumors, Small-cell Lung Cancer With Less Than 2 Prior Cytotoxic-containing Lines of Therapy
Keywords: lurbinectedin, PM01183, tumors, cancer, Pharma Mar
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 122 participants (actual)
Lead sponsor: PharmaMarIndustry (pharma, biotech, devices)
Study start: 25 May 2011 (actual)
Primary completion: 9 Aug 2017 (actual)
Study completion: 9 Aug 2017 (actual)
First posted: 28 Oct 2013 (estimated)
Results: Posted 9 Mar 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
lurbinectedin (PM01183) / doxorubicin	Experimental	Drug: lurbinectedin (PM01183), Drug: Doxorubicin

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (19 listed)

Voluntarily written informed consent
Age: between 18 and 75 years (both inclusive).
Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 1. Cohort of patients with SCLC and endometrial cáncer ECOG PS ≤ 2.
Life expectancy ≥ 3 months.
Patients with a histologically/cytologically confirmed diagnosis of advanced disease of any of the following tumors:

Key exclusion criteria (11 listed)

Concomitant diseases/conditions:
Brain metastases or leptomeningeal disease involvement.
Men or women of childbearing potential who are not using an effective method of contraception
Patients who have had radiation therapy in more than 35% of the bone marrow. This criterion will not apply to cohort of patients with SCLC and endometrial cáncer.
History of previous bone marrow and/or stem cell transplantation.

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
lurbinectedin (PM01183)lurbinectedin (PM01183) is presented as powder for concentrate for solution for infusion with two strengths, 1-mg and 4-mg vials.	Drug	lurbinectedin (PM01183) / doxorubicin
DoxorubicinCommercially available presentations of vials containing doxorubicin will be provided as appropriate.	Drug	lurbinectedin (PM01183) / doxorubicin

Primary outcome

Maximum Tolerated Dose (MTD)
Time frame: During the first cycle of treatment, up to 28 days
A minimum of three patients will be included at each DL. If no patients experience a DLT during Cycle 1, the dose will be escalated. If one of three patients experiences a DLT, three additional patients will be included at that level. If >1 evaluable patient during dose escalation at a given DL experience a DLT during Cycle 1, that DL will be considered the MTD and dose escalation will be terminated, except if all DLTs are related to neutropenia exclusively, in which case dose escalation may be resumed as originally planned in a new cohort of patients but with compulsory primary G-CSF prophylaxis.
Recommended Dose (RD)
Time frame: During the first cycle of treatment, up to 28 days
The DL immediately below the MTD, or DL4 if the MTD is not yet defined during dose escalation before the last DL (i.e., DL4) is reached, was to be initially expanded up to a minimum of nine evaluable patients. If less than three among the first nine evaluable patients treated within the expansion cohort experience a DLT during Cycle 1, this DL will be the RD.
Number of Participants With Dose-limiting Toxicities
Time frame: During the first cycle of treatment, up to 28 days
DLT,dose-limiting toxicity Patients enrolled into this study were originally not allowed to receive primary G-CSF prophylaxis. However, the finding of dose-limiting febrile neutropenia in two patients treated at dose level I in the present study (see below) suggested that increasing doses of the DOX/PM01183 combination might require primary G-CSF prophylaxis to decrease the risk of febrile neutropenia. Therefore, a separate dose escalation was established to define the MTD and the RD of the DOX/PM01183 combination with compulsory primary G-CSF prophylaxis.

4 secondary outcomes

Best Overall Tumor Response
Time frame: Tumor assessments were done every six weeks up to study completion
Duration of Response
Time frame: Time from the time measurement criteria are met for complete response, whichever is first recorded, until the first date in which progressive disease is objectively documented or the date of death, assessed up to 72 months
Progression-free Survival
Time frame: Time from the date of first administration to the date of PD or death (of any cause), whichever came first, assessed up to 72 months
Overall Survival
Time frame: Time from the date of first administration to the date of death (of any cause), assessed up to 72 months

Results and safety

Results posted to the registry on 9 Mar 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort A	73	0
Cohort B: SCLC 2nd Line	28	0
Cohort B: Endometrial	19	0

Primary outcome: Maximum Tolerated Dose (MTD)

Time frame: During the first cycle of treatment, up to 28 days. Number. Unit: DOX mg/m2 PM01183 mg FD

Measure	Patients Without Primary G-CSF Prophylaxis n = 57	Patients With Primary G-CSF Prophylaxis n = 11
Doxorubicin	50	50
PM01183	5.0	5.0

Population analysed: Fifty-seven of the 61 patients treated in this cohort without primary G-CSF prophylaxis were evaluable for DLTs Eleven of the 12 patients treated with primary G-CSF prophylaxis in this cohort were evaluable for DLTs.

Primary outcome: Recommended Dose (RD)

Time frame: During the first cycle of treatment, up to 28 days. Number. Unit: DOX mg/m2 PM01183 mg FD

Measure	Patients Without Primary G-CSF Prophylaxis n = 57
Doxorubicin	50
PM01183	4.0

Population analysed: Fifty-seven of the 61 patients treated in this cohort without primary G-CSF prophylaxis were evaluable for DLTs

Primary outcome: Number of Participants With Dose-limiting Toxicities

Time frame: During the first cycle of treatment, up to 28 days. Count of participants. Unit: Participants

Measure	Cohort A: Dose I Without Primary G-CSF Prophylaxis n = 5	Cohort A: Dose II Without Primary G-CSF Prophylaxis n = 7	Cohort A: Dose I Without Primary G-CSF Proph and Age Limit n = 3	Cohort A: Dose III Without Primary G-CSF Prophylaxis n = 37	Cohort A: Dose IV Without Primary G-CSF Prophylaxis n = 5	Cohort A: Dose I With Primary G-CSF Prophylaxis n = 3	Cohort A: Dose III With Primary G-CSF Prophylaxis n = 3	Cohort A: Dose IV With Primary G-CSF Prophylaxis n = 5	Cohort B: DOX [mg/m2] Plus PM01183 [mg/m2] n = 46
Value	2	0	0	8	2	0	0	2	4

Population analysed: Patients evaluable for the primary endpoint (evaluable for DLT): 5 patients were not evaluable in Cohort A and 1 patient in Cohort B.

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Participants were assessed through study completion, approximately 6 years. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort A	47 of 73 (64.4%)	73 of 73 (100%)	15 of 73
Cohort B: SCLC 2nd Line	19 of 28 (67.9%)	28 of 28 (100%)	22 of 28
Cohort B: Endometrial	14 of 19 (73.7%)	18 of 19 (94.7%)	11 of 19

Limitations and caveats (from the sponsor): None reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33704620Olmedo ME, Forster M, Moreno V, Lopez-Criado MP, Brana I, Flynn M, Doger B, de Miguel M, Lopez-Vilarino JA, Nunez R, Kahatt C, Cullell-Young M, Zeaiter A, Calvo E. Efficacy and safety of lurbinectedin and doxorubicin in relapsed small cell lung cancer. Results from an expansion cohort of a phase I study. Invest New Drugs. 2021 Oct;39(5):1275-1283. doi: 10.1007/s10637-020-01025-x. Epub 2021 Mar 11.

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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.

The record lists no contact. Use the official record to reach the sponsor.

7 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing lurbinectedin (PM01183) and Doxorubicin in people with Endometrial Adenocarcinomas and Neuroendocrine Tumors, run by PharmaMar at 7 sites in 2 countries.

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 19 inclusion and 11 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: finished, as of the sponsor's last update on 9 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in Spain, United Kingdom. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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: During the first cycle of treatment, up to 28 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01970540, which also lists 1 linked publication.

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 9 Mar 2020.