First Progression of MGMT Promoter-methylated GlioblastomaPhase 2RIGOLETTONCT04933942
Phase II Trial of Romiplostim for Thrombocytopenia Induced by Lomustine at First Progression of MGMT Promoter-meth Glioblastoma
A Phase 2 study testing Lomustine and Romiplostim in people with First Progression of MGMT Promoter-methylated Glioblastoma, run by European Organisation for Research and Treatment of Cancer - EORTC at 5 sites in 2 countries.
Trial summary
Romiplostim for low platelets caused by lomustine chemotherapy in patients with first recurrence (growing back) of a brain tumor, glioblastoma that is MGMT methylated. Lomustine is an anticancer drug often used to treat glioblastoma that grows back after initial treatment. This anticancer drug can cause side effects. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- First Progression of MGMT Promoter-methylated Glioblastoma
- What is being tested?
- Lomustine and Romiplostim
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 13 Sep 2022to 19 Dec 2022
- Phase
- Phase 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
- Age 18 years or more on day of signing informed consent
- Karnofsky Performance Score (KPS) of 60-100
- Life expectancy > 8 weeks
- Stable or decreasing dose of steroids for at least 1 week prior to enrolment
- Glioblastoma isocitrate dehydrogenase (IDH1) R132H wild-type, per cIMPACT NOW recommendations (Brat et al., 2018; Brat et al., 2020)
- MGMT promoter methylation determined by methylation-specific PCR or pyrosequencing or methylation profiling per local assessment.
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- Treatment with lomustine alone for first progression after any treatment comprising intent to treat with standard TMZ /RT →TMZ for newly diagnosed glioblastoma, with at least one dose of maintenance TMZ received. Hypofractionated regimens of RT are allowed. Patients should have received at least 75% of the RT dose. Patients enrolled in a clinical study for newly diagnosed glioblastoma and treated with standard of care and an experimental agent can participate. Patients who had RT alone or TMZ alone for newly diagnosed glioblastoma are not eligible. Patients must have received at least one dose of lomustine
- Clinically relevant thrombocytopenia defined as thrombocytopenia requiring dose delay of lomustine for at least one week (more than 7 days) (for any grade of toxicity) or requiring a dose reduction of lomustine because of grade 3 or 4 thrombocytopenia
- Diagnosis of first progression according to RANO criteria (Wen et al., 2010) more than 3 months after the end of radiotherapy for first-line treatment
- Patients may have been operated for recurrence. If operated, patients should have fully recovered from surgery as assessed by the investigator. Criteria for full recovery include absence of post-operative infection, recovery from medical complications (CTCAE grade 0 and 1 acceptable). Residual and measurable disease after surgery is not required, but surgery must have confirmed the recurrence. The post-surgery MRI (performed within 72 h) can be used for enrolment if dated within 6 weeks of enrolment.
- For non-operated patients: recurrent disease must correspond to at least one bi-dimensionally measurable contrast-enhancing lesion with clearly defined margins by MRI scan, with minimal diameters of 10 mm (10x10 mm), visible on 2 or more axial slices 5 mm apart (measurable disease according to RANO criteria). The MRI can be used for enrolment if dated within 6 weeks prior to enrolment.
- In case of clinical deterioration or increase of steroids since the last MRI, a new MRI should be done prior to enrolment and should be dated within 6 weeks prior to enrolment.
- Patients diagnosed with a venous thromboembolism or thrombotic events within the last 3 months can be enrolled if they have been on a stable regimen of anticoagulants for at least 14 days.
- Capacity for adequate fluid and oral intake
- Adequate bone marrow (except for platelet count, which can be <100 x 109/L at enrolment), renal and hepatic function within 7 days before enrolment
- Hemoglobin ≥ 8 g/dl
- Leukocytes count NOT requiring CGSF per local Standard of Care
- Total bilirubin ≤ 1.5 ULN
- Alanine transaminase (ALT), Aspartate Transaminase (AST), alkaline phosphatase (ALP) ≤ 2.5 × ULN
- Serum creatinine < 1.5 x ULN or creatinine clearance (CrCl) > 30 mL/min (using the Cockcroft-Gault formula)
- Women of childbearing potential (WOCBP) must have a negative urine or serum pregnancy test within 7 days prior to enrolment.
- Patients of childbearing / reproductive potential must agree to use adequate birth control measures, as defined by the investigator, during the study treatment period and for at least 6 months after the last study treatment. A highly effective method of birth control is defined as those which result in low failure rate (i.e. less than 1% per year) when used consistently and correctly (see Appendix G)
- Patients must also agree not to donate sperm during the study and for 6 months after receiving the last dose of study treatment
- Women who are breast feeding must agree to discontinue nursing prior to the first dose of study treatment and until 6 months after the last study treatment
- Ability to understand the requirements of the study, provide written informed consent and authorization of use and disclosure of protected health information, and agree to abide by the study restrictions and return for the required assessments
- Before patient enrolment, written informed consent must be given according to ICH/GCP, and national/local regulations
Who cannot join
- Radiotherapy or stereotactic radiosurgery for the treatment of first progression prior to enrolment in this study
- Known further progression after initiation of lomustine at the time of enrolment. Any suspicion of progression should be explored by a new MRI prior to enrolment
- Prior exposure to romiplostim or other thrombopoietin (TPO) mimetics
- Other hematological toxicity (anemia, neutropenia) requiring erythropoietin or GCSF
- Contraindications for MRI, including intolerance of gadolinium as a contrast agent
- Known coagulation disease or known hematological disease even if resolved.
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- Known hypercoagulative state (e.g., factor V Leiden, ATIII deficiency, protein C deficiency, protein S deficiency, antiphospholipid antibody syndrome)
- Clinically significant cardiac comorbidities, including: history of arterial thrombotic events (e.g., myocardial ischemia, transient ischemic attack, or stroke) within 6 months prior to screening, any history of active congestive heart failure (NYHA class II to IV), symptomatic ischaemia, including myocardial infarction, uncontrolled cardiac arrythmias, clinically significant ECG abnormalities, including screening ECG with QTc interval > 470 msec in women, > 450 msec in men, known pericardial disorder.
- History or present acute lymphoblastic leukaemia, acute myeloid leukaemia, any myeloid malignancy, myelodysplastic syndrome, myeloproliferative disease, multiple myeloma
- Evidence of active infection within 2 weeks prior to enrolment
- Known hypersensitivity to any E-coli derived product
- Known hypersensitivity to the active substances or to any of the excipients of the study drugs
- Any live attenuated vaccines, such as yellow fever vaccine, within the last 3 months before lomustine initiation.
- Known coeliac disease or wheat allergy
- Concurrent severe or uncontrolled medical disease (e.g., active systemic infection, diabetes, hypertension, coronary artery disease) that, in the opinion of the Investigator, would compromise the safety of the patient or compromise the ability of the patient to complete the study
- Known human immunodeficiency virus infection or acquired immune deficiency syndrome
- Known chronic active HBV or HCV within 4 months prior to enrolment
- Previous other malignancies, except for any previous malignancy which was treated with curative intent more than 2 years prior to enrolment, and except for adequately controlled limited basal cell carcinoma of the skin, adequately treated and without evidence of disease non-melanoma of the skin, squamous carcinoma of the skin and carcinoma in situ of the cervix, adequately treated breast ductal carcinoma in situ without evidence of disease, prostatic intraepithelial neoplasia without evidence of prostate cancer. Low risk prostate cancer (cT1-2a N0 and Gleason score ≤ 6 and PSA < 10 ng/mL), either totally resected or irradiated with curative intent (with PSA of less than or equal to 0.1 ng/mL) or under active surveillance as per ESMO guidelines
- Pregnant women
- Any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
Spain3 sites
Hospital Clinic Universitari de Barcelona
Barcelona, 08036
Hospital Universitario Ramon y Cajal
Madrid, 28034
Hospital Universitario 12 De Octubre
Madrid, 28041
Switzerland2 sites
Oncology Institute of Southern Switzerland
Bellinzona
UniversitaetsSpital
Zurich
About this study
Official title: Romiplostim for Thrombocytopenia Induced by Lomustine at First Progression of MGMT Promoter-methylated Glioblastoma: a Randomized Phase II Open Label Multicenter Study
Romiplostim for low platelets caused by lomustine chemotherapy in patients with first recurrence (growing back) of a brain tumor, glioblastoma that is MGMT methylated.
Lomustine is an anticancer drug often used to treat glioblastoma that grows back after initial treatment. This anticancer drug can cause side effects. The most frequent and potentially serious side effect of all is lowering of the blood platelets. Low platelets can cause bleedings in the the stomach and intestines, the skin, the brain and other systems and tissues.
Read more
Low platelets are also the main cause of delaying or prematurely (ending treatment before the planned end) stopping chemotherapy. There is no treatment for low platelets except platelet transfusions. Romiplostim is a drug that stimulates the production of platelets in the bone marrow. It is an approved drug in USA, Europe, Australia and Switzerland for a special type of blood disease in which the body breaks down its own blood platelets.
The purpose of the study is to start the treatment with romiplostim once low platelets are diagnosed in order to restore the platelet count and to prevent the platelet count from dropping again during the lomustine treatment.
- Being tested
- Lomustine, Romiplostim
- Keywords
- First progression, MGMT promoter-methylated glioblastoma, Phase II, Romiplostim, Lomustine
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LomustineDrug
Oral administration of Lomustine
RomiplostimDrug
Subcutaneous administration of Romiplostim
| Study group | Role | What they receive |
|---|---|---|
| Control group | Active comparator | Lomustine alone Receives: Drug: Lomustine |
| Experimental group | Experimental | Lomustine plus Romiplostim Receives: Drug: Lomustine; Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression Free Survival (PFS)
Measured: 5 months after the last patient in
Secondary outcomes (3)
Overall Survival (OS)
Measured: 5 months after the last patient in
the Health-related Quality of Life (HRQoL)
HRQoL will be assessed with the EORTC Quality of Life Questionnaire (QLQ-C30) version 3.
Measured: 5 months after the last patient in
Frequency of worst Adverse Events (AEs)
This study will use the International Common Terminology Criteria for Adverse Events (CTCAE), version 5.0, for adverse event reporting.
Measured: 5 months after the last patient in
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2021
- Study start13 Sep 2022
- Primary completion19 Dec 2022
- Study completion19 Dec 2022
Why it stopped: Amgen withdrew interest in providing further financial support
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for First Progression of MGMT Promoter-methylated GlioblastomaStudies of the same condition, open and under way.
- More trials run by European Organisation for Research and Treatment of Cancer - EORTCThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Sponsor type
- Research network
- Collaborators
- Amgen
- Responsible party
- Sponsor
- Investigators
- Emilie Le Rhun, Principal investigator, EORTC Study Coordinator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04933942
- Sponsor's protocol ID
- EORTC-1926-BTG
- Acronym
- RIGOLETTO
- Status last verified
- May 2023
- First submitted
- 14 Jun 2021
- Last updated
- 24 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Romiplostim for Thrombocytopenia Induced by Lomustine at First Progression of MGMT Promoter-methylated Glioblastoma: a Randomized Phase II Open Label Multicenter Study
- ClinicalTrials.gov ID
- NCT04933942
- Acronym / sponsor ID
- RIGOLETTO / EORTC-1926-BTG
- Keywords
- First progression, MGMT promoter-methylated glioblastoma, Phase II, Romiplostim, Lomustine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Amgen withdrew interest in providing further financial support
- Enrolment
- 0 participants (actual)
- Lead sponsor
- European Organisation for Research and Treatment of Cancer - EORTCResearch network
- Collaborators
- Amgen
- Study start
- 13 Sep 2022 (actual)
- Primary completion
- 19 Dec 2022 (actual)
- Study completion
- 19 Dec 2022 (actual)
- First posted
- 22 Jun 2021 (actual)
- 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.
Romiplostim for low platelets caused by lomustine chemotherapy in patients with first recurrence (growing back) of a brain tumor, glioblastoma that is MGMT methylated.
Lomustine is an anticancer drug often used to treat glioblastoma that grows back after initial treatment. This anticancer drug can cause side effects. The most frequent and potentially serious side effect of all is lowering of the blood platelets. Low platelets can cause bleedings in the the stomach and intestines, the skin, the brain and other systems and tissues.
Low platelets are also the main cause of delaying or prematurely (ending treatment before the planned end) stopping chemotherapy. There is no treatment for low platelets except platelet transfusions. Romiplostim is a drug that stimulates the production of platelets in the bone marrow. It is an approved drug in USA, Europe, Australia and Switzerland for a special type of blood disease in which the body breaks down its own blood platelets.
The purpose of the study is to start the treatment with romiplostim once low platelets are diagnosed in order to restore the platelet count and to prevent the platelet count from dropping again during the lomustine treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control group | Active comparator | Drug: LomustineLomustine alone |
| Experimental group | Experimental | Drug: Lomustine, Drug: RomiplostimLomustine plus Romiplostim |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (26 listed)
- Age 18 years or more on day of signing informed consent
- Karnofsky Performance Score (KPS) of 60-100
- Life expectancy > 8 weeks
- Stable or decreasing dose of steroids for at least 1 week prior to enrolment
- Glioblastoma isocitrate dehydrogenase (IDH1) R132H wild-type, per cIMPACT NOW recommendations (Brat et al., 2018; Brat et al., 2020)
Key exclusion criteria (20 listed)
- Radiotherapy or stereotactic radiosurgery for the treatment of first progression prior to enrolment in this study
- Known further progression after initiation of lomustine at the time of enrolment. Any suspicion of progression should be explored by a new MRI prior to enrolment
- Prior exposure to romiplostim or other thrombopoietin (TPO) mimetics
- Other hematological toxicity (anemia, neutropenia) requiring erythropoietin or GCSF
- Contraindications for MRI, including intolerance of gadolinium as a contrast agent
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 |
|---|---|---|
| LomustineOral administration of Lomustine | Drug | Control group, Experimental group |
| RomiplostimSubcutaneous administration of Romiplostim | Drug | Experimental group |
Primary outcome
Progression Free Survival (PFS)
Time frame: 5 months after the last patient in
3 secondary outcomes
Overall Survival (OS)
Time frame: 5 months after the last patient in
the Health-related Quality of Life (HRQoL)
Time frame: 5 months after the last patient in
Frequency of worst Adverse Events (AEs)
Time frame: 5 months after the last patient in
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2023.
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.
Emilie Le Rhun principal investigator
EORTC Study Coordinator
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lomustine and Romiplostim in people with First Progression of MGMT Promoter-methylated Glioblastoma, run by European Organisation for Research and Treatment of Cancer - EORTC at 5 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 26 inclusion and 20 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: withdrawn, as of the sponsor's last update on 24 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Spain, Switzerland. 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 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 withdrawn before enrolling participants, so there are no results.
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?
Progression Free Survival (PFS) (time frame: 5 months after the last patient in). 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 24 May 2023.