A Phase 2 interventional study of Alisertib in Small Cell Lung Cancer, sponsored by Puma Biotechnology, Inc.. Recruiting at 30 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-17.
Sponsored by Puma Biotechnology, Inc. · Phase 2, Interventional, and Treatment
PUMA-ALI-4201 is a Phase 2 study evaluating alisertib monotherapy in patients with pathologically-confirmed small cell lung cancer (SCLC) following progression on or after treatment with one platinum-based chemotherapy and anti-PD-L1/PD-1 immunotherapy agent. Up to one additional systemic anti-cancer therapy for SCLC is allowed, for a total of up to two prior treatment regimens. This study is intended to identify the biomarker-defined subgroup(s) that may benefit most from alisertib treatment and to evaluate the efficacy, safety, and pharmacokinetics of alisertib.
1,301 studies on the registry are indexed under Small Cell Lung Carcinoma; 367 are open to participants now.
This study's planned enrollment of 120 is above the median of 56 across 1,077 interventional studies indexed under Small Cell Lung Carcinoma.
Browse Small Cell Lung Carcinoma studies →Puma Biotechnology, Inc. is the lead sponsor of 38 studies on the registry; 3 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 17 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Note: There are additional inclusion and exclusion criteria. The study center will determine if you meet all of the criteria.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PD-1, PD-L1 and checkpoint inhibitors
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
50 mg (Prior to Amendment 2) or 60 mg (Amendment 2) or 70 mg (Amendment 3 or later) of alisertib PO BID on days 1-7 of each 21-day cycle
Drug: Alisertib
Alisertib enteric-coated tablets
Also known as: PB-8237, MLN8237
Objective response rate (ORR) within biomarker-defined subgroup
Objective response rate is defined as the percentage of participants demonstrating a confirmed objective response during the study
Time frame: From date of first dose to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 36 months
Duration of response (DOR) within biomarker-defined subgroup
Duration of response is measured from the time at which measurement criteria are first met for CR or PR (whichever status is recorded first) until the first date of recurrence or progressive disease (PD) or death is objectively documented.
Time frame: From start date of response (after date of first dose) to first PD, assessed up to 36 months
Disease Control Rate (DCR) within biomarker-defined subgroup
Disease control rate is the proportion of patients who achieve overall tumor response (confirmed CR or PR) or SD lasting for at least 8 weeks from first dose of investigational product.
Time frame: From date of first dose to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 36 months
Progression Free Survival (PFS) within biomarker-defined subgroup
Progression Free Survival (PFS) is measured in months and based on the local tumor assessment. The time interval from the date of first dose until the first date on which recurrence, progression, or death due to any cause, is documented.
Time frame: From date of first dose to date of recurrence, progression or death, assessed up to 36 months
Overall Survival (OS) within biomarker-defined subgroup
Overall survival (OS) is defined as the time from date of first dose to death due to any cause, censored at the last date known alive on or prior to the data cutoff employed for the analysis, whichever was earlier.
Time frame: From date of first dose to death, assessed up to 36 months
Objective response rate (ORR) in the enrolled patient population
Objective response rate is defined as the percentage of participants demonstrating a confirmed objective response during the study.
Time frame: From date of first dose to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 36 months
Duration of response (DOR) in the enrolled patient population
Duration of response is measured from the time at which measurement criteria are first met for CR or PR (whichever status is recorded first) until the first date of recurrence or progressive disease (PD) or death is objectively documented.
Time frame: From start date of response (after date of first dose) to first PD, assessed up to 36 months
Disease Control Rate (DCR) in the enrolled patient population
Disease control rate is the proportion of patients who achieve overall tumor response (confirmed CR or PR) or SD lasting for at least 8 weeks from first dose of investigational product.
Time frame: From date of first dose to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 36 months
Progression Free Survival (PFS) in the enrolled patient population
Progression Free Survival (PFS) is measured in months and based on the local tumor assessment. The time interval from the date of first dose until the first date on which recurrence, progression, or death due to any cause, is documented.
Time frame: From date of first dose to date of recurrence, progression or death, assessed up to 36 months
Overall Survival (OS) in the enrolled patient population
Overall survival (OS) is defined as the time from date of first dose to death due to any cause, censored at the last date known alive on or prior to the data cutoff employed for the analysis, whichever was earlier.
Time frame: From date of first dose to death, assessed up to 36 months
Percentage of Participants With Treatment-Emergent Adverse Events (Adverse Events and Serious Adverse Events) in the enrolled patient population
Treatment emergent adverse events are those events reported on or after the first dose of investigational product and up to 28 days after last dose.
Time frame: From date of first dose through last dose plus 28 days, assessed up to 36 months
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Plan to share: Yes — Puma Biotechnology is committed to sharing clinical trial data and information to help physicians and patients make informed treatment decisions, and to help qualified researchers advance scientific knowledge. In accordance with legal and regulatory requirements, Puma publishes study protocol information and clinical study results on clinical trial registries, including ClinicalTrials.gov and EU Clinical Trials Register. Puma also publishes information about clinical studies in peer-reviewed scientific journals and shares data in scientific meetings. Puma commits to safeguarding confidentiality and patient privacy throughout the clinical trial data and information sharing process. Any patient-level data will be anonymized to protect personally identifiable information. Qualified researchers and study participants may submit requests for other study documentation and clinical trial data to clinicaltrials@pumabiotechnology.com for consideration.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Time frame: Clinical study documents and clinical trial data may be requested by qualified researchers and study participants for studies that have been completed for at least 18 months, and for which the indication of the drug has been approved in the US and/or EU, as applicable. Requests will be accepted for up to 24 months after the criteria described in this section are met.
Access criteria: Requestors must provide organizational contact information; a detailed research plan, including outcomes; timeline for completion of the research; qualifications of the research team; funding source; and potential conflicts of interest. Puma will not provide access to patient-level data if there is a reasonable likelihood that individual patients could be identified, or in cases where confidentiality or consent provisions prohibit transfer of data or information to third parties. Additionally, Puma will not disclose information that jeopardizes intellectual property rights or divulges confidential commercial information.
Data sharing details ↗ · pumabiotechnology.com
No publications or documents are linked to this record.
Started Feb 2024 (actual). Primary completion is estimated for Oct 2027, 44 months after the start.
Of 53 industry-sponsored phase 2 interventional studies under Small Cell Lung Carcinoma, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 47. This study's planned 44 months is above that.
Counting every sponsor, half of 203 studies reached it within 47 months; a quarter within 29 and three quarters within 84.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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Studies of the drug tested here: MLN 8237