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NCT03190967

T-DM1 Alone Versus T-DM1 and Metronomic Temozolomide in Secondary Prevention of HER2-Positive Breast Cancer Brain Metastases Following Stereotactic Radiosurgery

Terminated Phase 1, PHASE2 Results posted Last updated 11 October 2023
What this trial tests

Phase 1, PHASE2 trial testing T-DM1 in Breast Cancer in 12 participants. Terminated before completion.

Timeline
18 April 2018
Primary endpoint
28 June 2021
13 June 2023

Quick facts

Lead sponsorNational Cancer Institute (NCI)
PhasePhase 1, PHASE2
StatusTerminated
Study typeINTERVENTIONAL
Allocationrandomized
Designsequential
Maskingnone
Primary purposetreatment
Enrollment12
Start date18 April 2018
Primary completion28 June 2021
Estimated completion13 June 2023
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

National Cancer Institute (NCI)

Who can join

18 and older, any sex, with Breast Cancer or Brain Metastasis. 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.

Phase I: Maximum Tolerated Dose (MTD) of Temozolomide (TMZ) When Used With T-DM1 (Ado-trastuzumab) Primary · first 21 days of treatment

Maximum tolerated dose of TMZ when used in combination with T-DM1. MTD is defined as the dose level at which 0 or 1 participant in 6 has a dose limiting toxicity (DLT). A DLT is defined as grade 3 or higher non-hematologic adverse events excluding grade 3 hypertension controlled with anti-hypertensive therapy; or grade 3 asymptomatic electrolytes imbalance; grade 3 endocrinopathy; grade 3 asymptomatic increase in aspartate aminotransferase or alanine aminotransferase; and transient (lasting less than \<48 hours) nausea, emesis, or diarrhea if corrected with conservative measures within 24-48 h

GroupValue95% CI
All Participants40
Phase I: Number of Participants With Grade 3 and/or Grade 4 Adverse Events Secondary · Evaluated at the beginning of every cycle while on study, for an average of 9.6 months (range 2.8-33.9 months).

Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 3 is severe. Grade 4 is life-threatening.

Grade 3 Anemia
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^20
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^21
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^20
Grade 3 Aspartate aminotransferase increased
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^20
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^20
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^21
Grade 3 CD4 lymphocytes decreased
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^21
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^21
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^25
Grade 3 Dysphasia
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^21
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^20
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^20
Grade 3 Hypokalemia
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^20
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^20
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^21
Grade 3 Lymphocyte count decreased
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^21
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^22
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^25
Grade 3 Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Malignant Neoplasm-CML
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^20
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^20
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^21
Grade 3 Neutrophil count decreased
GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^20
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^20
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^21
Phase I: Number of Participants With Dose Limiting Toxicity (DLTs) at Each Dose Level Secondary · After first cycle of treatment, up to 30 days

Number of participants with DLTs at each dose level 30 days after treatment. A DLT is defined as grade 3 or higher non-hematologic adverse events excluding grade 3 hypertension controlled with anti-hypertensive therapy; or grade 3 asymptomatic electrolytes imbalance; grade 3 endocrinopathy; grade 3 asymptomatic increase in aspartate aminotransferase or alanine aminotransferase; and transient (lasting less than \<48 hours) nausea, emesis, or diarrhea if corrected with conservative measures within 24-48 hours. A hematologic grade 4 neutropenia of ≥ 7 days duration, grade ≥ 3 thrombocytopenia, an

GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^20
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^20
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^20
Phase I: Median Survival Secondary · From date of first therapy until death, an average of 40.79 months

Median amount of time participants survives after therapy. Survival compared between the two arms and Kaplan-Meier curves constructed with a two-tailed log-rank test used to compare the arms.

GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^237.8± 22.6
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^248.4± 7.5
Level 3 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^238.5± 9.5
Phase I: Standard Time to Progression (TTP) Secondary · From first day of treatment to the day of disease progression, an average of 15 months.

TTP is the time between the first day of treatment to the day of disease progression. Progression was assessed using the Response Evaluation Criteria in Solid Tumors (RECIST). Progression is at least a 25% increase in the sum of products of all measurable lesions over smallest sum observed, clear worsening of any evaluable disease, appearance of any new lesion/site, or failure to return for evaluation due to death or deteriorating condition.

GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^26.95± 1.06
Level 2 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^216.26± 13.8
Level 3 Phase I:Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^217.46± 8.6
Phase I: Number of Participants With Serious and/or 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 44 months and 27 days for level 1, 28 months and 10 days for level 2, and 40 months and 8 days for level 3.

Here is the number of participants with serious and/or 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

GroupValue95% CI
Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^23
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^23
Level 3 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^26

Adverse events — posted to ClinicalTrials.gov

Time frame: Date treatment consent signed to date off study, approximately 44 months and 27 days for level 1, 28 months and 10 days for level 2, and 40 months and 8 days for level 3.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Level 1 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 30 mg/m^2
Serious: 1/3 (33%)
Deaths: 2/3
Level 2 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 40 mg/m^2
Serious: 0/3 (0%)
Deaths: 1/3
Level 3 Phase I: Ado-trastuzumab (T-DMI) 3.6 mg/kg + Temozolomide (TMZ) 50 mg/m^2
Serious: 1/6 (17%)
Deaths: 1/6

Serious adverse events (3 terms)

ReactionSystemLevel 1 Phase I: Ado-trast…Level 2 Phase I: Ado-trast…Level 3 Phase I: Ado-trast…
DysphasiaNervous system disorders
Muscle weakness right-sidedMusculoskeletal and connective tissue disorders
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Malignant Neoplasm -CMLNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Other adverse events (134 terms — click to expand)

ReactionSystemLevel 1 Phase I: Ado-trast…Level 2 Phase I: Ado-trast…Level 3 Phase I: Ado-trast…
NauseaGastrointestinal disorders
Platelet count decreasedInvestigations
Aspartate aminotransferase increasedInvestigations
CD4 lymphocytes decreasedInvestigations
ConstipationGastrointestinal disorders
Lymphocyte count decreasedInvestigations
VomitingGastrointestinal disorders
Alanine aminotransferase increasedInvestigations
Alkaline phosphatase increasedInvestigations
GGT increasedInvestigations
HypokalemiaMetabolism and nutrition disorders
HyponatremiaMetabolism and nutrition disorders
Peripheral sensory neuropathyNervous system disorders
Weight lossInvestigations
White blood cell decreasedInvestigations
AnemiaBlood and lymphatic system disorders
ArthralgiaMusculoskeletal and connective tissue disorders
Creatinine increasedInvestigations
FatigueGeneral disorders
HeadacheNervous system disorders
Nasal congestionRespiratory, thoracic and mediastinal disorders
Abdominal painGastrointestinal disorders
Activated partial thromboplastin time prolongedInvestigations
AnorexiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
CoughRespiratory, thoracic and mediastinal disorders
DiarrheaGastrointestinal disorders
DizzinessNervous system disorders
Dry mouthGastrointestinal disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
HypercalcemiaMetabolism and nutrition disorders
Infections and infestations - Other, ShinglesInfections and infestations
Mucositis oralGastrointestinal disorders
Neutrophil count decreasedInvestigations
PainGeneral disorders
Pain in extremityMusculoskeletal and connective tissue disorders
Sore throatRespiratory, thoracic and mediastinal disorders
Allergic reactionImmune system disorders
AlopeciaSkin and subcutaneous tissue disorders
AnxietyPsychiatric disorders

Most-reported serious reactions: Dysphasia, Muscle weakness right-sided, Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Malignant Neoplasm -CML.

Data from ClinicalTrials.gov NCT03190967 adverse events section.

Sponsor's own description

Background: Sometimes breast cancer spreads (metastasizes) to the brain. Researchers want to study new treatments for brain metastases. The drug Temozolomide is approved to treat brain tumors. Researchers want to see if combining it with the drug trastuzumab emtansine (T-DMI) prevents the formation of new metastases in the brain. Objective: To study if Temozolomide with T-DM1 lowers the chance of having new metastases in the brain. Eligibility: Adults at least 18 years old with a human epidermal growth factor receptor 2 (HER2)-positive breast cancer that has spread to the brain and was recently treated with stereotactic radiation or surgery. Design: Participants will be screened with * Medical history * Physical exam * Heart tests * A scan (computed tomography (CT) that makes a picture of the body using a small amount of radiation * A scan (magnetic resonance imaging (MRI) that uses a magnetic field to make an image of the brain * Blood tests. * Pregnancy test. The study will be done in 3-week cycles. All participants will get T-DM1 on Day 1 of every cycle through a small plastic tube inserted in an arm vein. Some participants will also take Temozolomide capsules by mouth every day. Participants will keep a medication diary. During the study, participants will also: * Repeat most of the screening tests. * Answer questions about their general well-being and functioning. Participants will have lumbar puncture at least 2 times. A needle is inserted into the spinal canal low in the back and cerebrospinal fluid is collected. This will be done with local anesthesia and with the help of images. Participants will be asked to provide tumor samples when available. Participants will have a follow-up visit about 1 month after stopping the study drug. They will be contacted by telephone or email every 3 months after that.

Publications & conference data

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

  1. A framework for the development of effective anti-metastatic agents.
    Anderson RL, Balasas T, Callaghan J, Coombes RC, et al · · 2019 · cited 249× · PMID 30514977 · DOI 10.1038/s41571-018-0134-8
  2. The promise and challenges of combination therapies with antibody-drug conjugates in solid tumors.
    Wei Q, Li P, Yang T, Zhu J, et al · · 2024 · cited 102× · PMID 38178200 · DOI 10.1186/s13045-023-01509-2
  3. Investigational chemotherapy and novel pharmacokinetic mechanisms for the treatment of breast cancer brain metastases.
    Shah N, Mohammad AS, Saralkar P, Sprowls SA, et al · · 2018 · cited 102× · PMID 29604436 · DOI 10.1016/j.phrs.2018.03.021
  4. Understanding patterns of brain metastasis in breast cancer and designing rational therapeutic strategies.
    Brosnan EM, Anders CK. · · 2018 · cited 98× · PMID 29911111 · DOI 10.21037/atm.2018.04.35
  5. Brain Metastases in HER2-Positive Breast Cancer: Current and Novel Treatment Strategies.
    Garcia-Alvarez A, Papakonstantinou A, Oliveira M. · · 2021 · cited 77× · PMID 34208287 · DOI 10.3390/cancers13122927
  6. The PI3K/Akt/mTOR pathway as a preventive target in melanoma brain metastasis.
    Tehranian C, Fankhauser L, Fankhauser L, Harter PN, et al · · 2022 · cited 57× · PMID 34216217 · DOI 10.1093/neuonc/noab159
  7. Antibody-Drug Conjugates for the Treatment of Breast Cancer.
    Corti C, Giugliano F, Nicolò E, Ascione L, et al · · 2021 · cited 51× · PMID 34207890 · DOI 10.3390/cancers13122898
  8. Breast cancer brain metastasis: Current evidence and future directions.
    Sun H, Xu J, Dai S, Ma Y, et al · · 2023 · cited 46× · PMID 35822637 · DOI 10.1002/cam4.5021

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