NCT01931098 is a Phase 2 clinical trial of topotecan in Glioblastoma, sponsored by National Cancer Institute (NCI).

Who is sponsoring NCT01931098?

NCT01931098 is sponsored by National Cancer Institute (NCI).

What drugs are being tested in NCT01931098?

Interventions in NCT01931098: topotecan, pazopanib.

What condition does NCT01931098 study?

NCT01931098 studies Glioblastoma, Glioblastoma Multiforme, Gliosarcoma, Brain Neoplasms, Central Nervous System Neoplasms.

Is NCT01931098 still recruiting?

NCT01931098 is currently completed. Last updated 11 August 2025.

Are results published for NCT01931098?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2025-08-11 · How we verify

NCT01931098

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Oral Pazopanib Plus Oral Topotecan Metronomic Antiangiogenic Therapy for Recurrent Glioblastoma Multiforme (A) Without Prior Bevacizumab Exposure and (B) After Failing Prior Bevacizumab

Completed Phase 2 Results posted Last updated 11 August 2025

What this trial tests

Phase 2 trial testing topotecan in Glioblastoma in 35 participants. Completed in 12 September 2019.

Timeline

10 December 2015

Primary endpoint
12 September 2019

12 September 2019

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	35
Start date	10 December 2015
Primary completion	12 September 2019
Estimated completion	12 September 2019
Sites	1 location across United States

Drugs / interventions tested

topotecan (topotecan) — full drug profile →
pazopanib — full drug profile →

Conditions studied

Glioblastoma — all drugs for Glioblastoma →
Glioblastoma Multiforme — all drugs for Glioblastoma Multiforme →
Gliosarcoma — all drugs for Gliosarcoma →
Brain Neoplasms — all drugs for Brain Neoplasms →

Sponsor

National Cancer Institute (NCI)

Who can join

18 and older, any sex, with Glioblastoma or Glioblastoma Multiforme. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Proportion of Patients That Have Progressive Survival (PFS) at Six Months From Patient Registration for Bevacizumab Naive Patients and PFS at 3 Months for Patients With Prior Bevacizumab Treatment Primary · six months from patient registration for bevacizumab naive patients, and 3 months from patient registration for patients with prior bevacizumab treatment

PFS was evaluated using the Kaplan-Meier product-limit survival curve methodology. For participants with no prior bevacizumab exposure, PFS is defined as 2 or more participants who are progression free at 6 months. For participants with prior bevacizumab exposure, PFS is defined as 1 or more participants who are progression free at 3 months. Progression is a 25% increase in the sum of products of all measurable lesions over smallest sum observed (over baseline if no decrease) or clear worsening of evaluable disease or the appearance of any new lesions.

3 months

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	0.44	0.14 – 0.72
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	0.18	0.06 – 0.36

6 months

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	0.11	0.01 – 0.39
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	0	0 – 0

Overall Survival Secondary · From date of registration to date of death due to any cause, up to 5 years.

OS was evaluated using the Kaplan-Meier product-limit survival curve methodology and is defined as the time from date of registration to date of death due to any cause.

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	7.1	2.9 – 8.2
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	6.3	2.8 – 9.1

Mean Symptom Severity Score Between Responders and Non-responders at Baseline and by Cycle Secondary · Baseline, and Cycles 1-6

Responders and Non-Responders symptom presence and severity were assessed by the M.D. Anderson Symptom Inventory-Brain Tumor Module (MDASI-BT) questionnaire using an scale of (0-10); 0 being not present and 10 being the worst, and Responders were defined as patients with stable disease (SD) and partial response (PR) as their best response, and Non-responders were defined as patients with disease progression (PD) as their best response. Partial response is greater than or equal to 50% decrease under baseline in the sum of products of perpendicular diameters of all measurable lesion. Progressive

Responders - baseline

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	1.1	± 0.4
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	1.9	± 1.3

Responders - Cycle 1

Group	Value	95% CI
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	2.3	± 1.4

Responders - Cycle 2

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	0.8	± 0.4
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	3.5	± 2.5

Responders - Cycle 3

Group	Value	95% CI
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	2.9	± 3.4

Responders - Cycle 4

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	2.0	± 1.7

Responders - Cycle 5

Group	Value	95% CI
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	1.5

Responders - Cycle 6

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	2.1	± 2.0

Non-Responders - baseline

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	1.6	± 0.7
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	2.8	± 2.1

Mean Symptom Interference Score Between Responders and Non-responders at Baseline and by Cycle Secondary · Baseline, and Cycles 1-6

Responders and Non-Responders symptom interference was assessed by the M.D. Anderson Symptom Inventory-Brain Tumor Module (MDASI-BT) questionnaire that determined how much general activity, mood, work inside and outside the home, relations with other people, walking and enjoying life interfered with a participant's life rated on an scale (0-10); 0 being not present and 10 being the worst. Responders were defined as patients with stable disease (SD) and partial response (PR) as their best response, and Non-responders were defined as patients with disease progression (PD) as their best response.

Responders - baseline

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	2.1	± 1.2
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	3.6	± 2.9

Responders - Cycle 1

Group	Value	95% CI
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	3.7	± 2.5

Responders - Cycle 2

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	1.4	± 0.4
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	6.0	± 3.2

Responders - Cycle 3

Group	Value	95% CI
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	4.2	± 4.0

Responders - Cycle 4

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	3.2	± 2.0

Responders - Cycle 5

Group	Value	95% CI
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	0.2

Responders - Cycle 6

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	2.7	± 0.9

Non-Responders - baseline

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	1.7	± 1.2
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	3.9	± 3.2

Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0) Secondary · Date treatment consent signed to date off study, approximately 20 months and 18 days for the first Arm/Group, and 28 months and 23 days for the second Arm/Group.

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one

Group	Value	95% CI
Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure	9
Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure	21

Adverse events — posted to ClinicalTrials.gov

Time frame: Date treatment consent signed to date off study, approximately 20 months and 18 days for the first Arm/Group, and 28 months and 23 days for the second Arm/Group.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Glioblastoma or Gliosarcoma With No Prior Bevacizumab Exposure

Serious: 2/9 (22%)

Deaths: 9/9

Glioblastoma or Gliosarcoma With Prior Bevacizumab Exposure

Serious: 7/26 (27%)

Deaths: 20/26

Serious adverse events (12 terms)

Reaction	System	Glioblastoma or Gliosarcom…	Glioblastoma or Gliosarcom…
Muscle weakness left-sided	Musculoskeletal and connective tissue disorders	—	—
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Glioblastoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Alanine aminotransferase increased	Investigations	—	—
Cholecystitis	Hepatobiliary disorders	—	—
Generalized muscle weakness	Musculoskeletal and connective tissue disorders	—	—
Headache	Nervous system disorders	—	—
Meningitis	Infections and infestations	—	—
Musculoskeletal and connective tissue disorder - Other, specify	Musculoskeletal and connective tissue disorders	—	—
Seizure	Nervous system disorders	—	—
Thromboembolic event	Vascular disorders	—	—
Upper gastrointestinal hemorrhage	Gastrointestinal disorders	—	—
Hyperglycemia	Metabolism and nutrition disorders	—	—

Other adverse events (103 terms — click to expand)

Reaction	System	Glioblastoma or Gliosarcom…	Glioblastoma or Gliosarcom…
Fatigue	General disorders	—	—
Hypertension	Vascular disorders	—	—
Diarrhea	Gastrointestinal disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Anorexia	Metabolism and nutrition disorders	—	—
Headache	Nervous system disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Constipation	Gastrointestinal disorders	—	—
Platelet count decreased	Investigations	—	—
Blurred vision	Eye disorders	—	—
Cognitive disturbance	Nervous system disorders	—	—
Dysgeusia	Nervous system disorders	—	—
Dysphasia	Nervous system disorders	—	—
Hypoalbuminemia	Metabolism and nutrition disorders	—	—
Memory impairment	Nervous system disorders	—	—
Neutrophil count decreased	Investigations	—	—
Seizure	Nervous system disorders	—	—
Weight loss	Investigations	—	—
White blood cell decreased	Investigations	—	—
Abdominal pain	Gastrointestinal disorders	—	—
Alanine aminotransferase increased	Investigations	—	—
Anemia	Blood and lymphatic system disorders	—	—
Bronchial infection	Infections and infestations	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—
Confusion	Psychiatric disorders	—	—
Depression	Psychiatric disorders	—	—
Dizziness	Nervous system disorders	—	—
Gait disturbance	General disorders	—	—
Hypokalemia	Metabolism and nutrition disorders	—	—
Muscle weakness left-sided	Musculoskeletal and connective tissue disorders	—	—
Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Pain	General disorders	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—
Skin and subcutaneous tissue disorders - Other, specify	Skin and subcutaneous tissue disorders	—	—
Anxiety	Psychiatric disorders	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—
Aspartate aminotransferase increased	Investigations	—	—
Ataxia	Nervous system disorders	—	—
Bladder infection	Infections and infestations	—	—
Blood and lymphatic system disorders - Other, Blood in stool	Blood and lymphatic system disorders	—	—

Most-reported serious reactions: Muscle weakness left-sided, Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Glioblastoma, Alanine aminotransferase increased, Cholecystitis, Generalized muscle weakness, Headache, Meningitis, Musculoskeletal and connective tissue disorder - Other, specify.

Data from ClinicalTrials.gov NCT01931098 adverse events section.

Sponsor's own description

Background: Glioblastoma is the most common and most aggressive type of malignant brain tumor. The drug pazopanib is used to treat people with a type of kidney cancer. Topotecan is used to treat lung cancer. Both topotecan and pazopanib have individually been used to treat patients with glioblastoma and some anti-tumor activity has been found. Researchers want to see if these two drugs together may be able to help people with glioblastoma. Objectives: To learn if pazopanib with topotecan can help control glioblastoma. Also, to study the safety of this drug combination. Eligibility: Adults at least 18 years old whose glioblastoma has returned after treatment. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Brain computed tomography (CT) or magnetic resonance imaging (MRI) For these, participants lay in a machine that takes pictures. Chest CT scan or x-ray Heart electrocardiogram (EKG) A questionnaire about quality of life Participants will be assigned to a study group. Participants will take the study drugs for 28-day cycles for up to 1 year. They will take capsules of topotecan by mouth once every day. They will take tablets of pazopanib by mouth once every day. Participants will write in a diary the times they take the study drugs. Participants will have several study visits during each cycle. These may include Blood pressure measurement Blood and urine tests EKG Physical exam and/or neurological exam Brain MRI or CT scan to check the status of the disease A symptom questionnaire At the end of treatment, participants will have a physical exam. They may have blood drawn. Participants will have follow-up calls once every 3 months to check.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Hypoxia signaling in human health and diseases: implications and prospects for therapeutics.
Luo Z, Tian M, Yang G, Tan Q, et al · · 2022 · cited 326× · PMID 35798726 · DOI 10.1038/s41392-022-01080-1
Understanding the immunosuppressive microenvironment of glioma: mechanistic insights and clinical perspectives.
Lin H, Liu C, Hu A, Zhang D, et al · · 2024 · cited 232× · PMID 38720342 · DOI 10.1186/s13045-024-01544-7
An update on the molecular biology of glioblastoma, with clinical implications and progress in its treatment.
Verdugo E, Puerto I, Medina MÁ. · · 2022 · cited 152× · PMID 36129048 · DOI 10.1002/cac2.12361
Lessons from anti-vascular endothelial growth factor and anti-vascular endothelial growth factor receptor trials in patients with glioblastoma.
Lu-Emerson C, Duda DG, Emblem KE, Taylor JW, et al · · 2015 · cited 140× · PMID 25713439 · DOI 10.1200/jco.2014.55.9575
Advances in the Knowledge of the Molecular Biology of Glioblastoma and Its Impact in Patient Diagnosis, Stratification, and Treatment.
Delgado-Martín B, Medina MÁ. · · 2020 · cited 129× · PMID 32382477 · DOI 10.1002/advs.201902971
Cancer combination therapies by angiogenesis inhibitors; a comprehensive review.
Ansari MJ, Bokov D, Markov A, Jalil AT, et al · · 2022 · cited 117× · PMID 35392964 · DOI 10.1186/s12964-022-00838-y
A Systematic Review of Glioblastoma-Targeted Therapies in Phases II, III, IV Clinical Trials.
Cruz Da Silva E, Mercier MC, Etienne-Selloum N, Dontenwill M, et al · · 2021 · cited 99× · PMID 33918704 · DOI 10.3390/cancers13081795
Glioblastoma at the crossroads: current understanding and future therapeutic horizons.
Singh S, Dey D, Barik D, Mohapatra I, et al · · 2025 · cited 91× · PMID 40628732 · DOI 10.1038/s41392-025-02299-4

Verify or expand the search:

Other trials of topotecan

Trials testing the same drug.

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NCT02500459 — Topotecan in Glioma Undergoing A Clinically-Indicated Surgical Resection · EARLY_PHASE1 · terminated
NCT01857934 — Therapy for Children With Advanced Stage Neuroblastoma · Phase 2 · completed
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NCT00732420 — Open-label Study of Topotecan and Pazopanib in Advanced Solid Tumors · Phase 1 · completed

Other recruiting trials for Glioblastoma

Currently open trials in the same condition.

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NCT05653635 — Contribution From PET-DOPA in Glioblastoma Re-irradiation - A Randomized Phase II Study · Phase 2 · recruiting
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NCT07448480 — Comprehensive Analysis of Chemotherapy and Targeted Therapy Outcomes in Recurrent Malignant Gliomas · active not recruiting
NCT07410676 — EBNK-001 Allogeneic NK Cells With Low-Dose IL-15 ± Pembrolizumab in Advanced Solid Tumors · Phase 1, PHASE2 · recruiting

Other National Cancer Institute (NCI) trials

Trials by the same sponsor.

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NCT07012044 — A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After · Phase 1 · not yet recruiting
NCT07437950 — Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A Myel · Phase 2 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01931098 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 11 August 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01931098.

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