Trial Outcomes & Findings for A Phase 2 Study of Lamivudine in Patients With p53 Mutant Metastatic Colorectal Cancer (NCT NCT03144804)

Last Updated: 2024-06-06

Results Overview

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT scan or MRI, complete response (CR) is the disappearance of all target lesions and partial response (PR) is a \>/=30% decrease in the sum of the longest diameter of target lesions. Overall Response (OR) = CR + PR. The best overall response is the best response recorded from the start of the treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started). The patient's best response assignment will depend on the achievement of both measurement and confirmation criteria.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

36 participants

Primary outcome timeframe

2 years

Results posted on

2024-06-06

Participant Flow

Participant milestones

Participant milestones
Measure	Lamivudine * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Overall Study STARTED	36
Overall Study COMPLETED	32
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Lamivudine * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Overall Study 4 participants had disease progression within 1 month of study treatment and were deemed unevaluable	4

Baseline Characteristics

A Phase 2 Study of Lamivudine in Patients With p53 Mutant Metastatic Colorectal Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lamivudine n=32 Participants * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	24 Participants n=93 Participants
Age, Categorical >=65 years	8 Participants n=93 Participants
Age, Continuous	59 years n=93 Participants
Sex: Female, Male Female	14 Participants n=93 Participants
Sex: Female, Male Male	18 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	29 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	29 Participants n=93 Participants
Race (NIH/OMB) More than one race	1 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=93 Participants
Region of Enrollment United States	32 participants n=93 Participants

PRIMARY outcome

Timeframe: 2 years

Outcome measures

Outcome measures
Measure	Lamivudine n=32 Participants * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Overall Response Rate	0 participants

SECONDARY outcome

Timeframe: 2 years

Population: Of the 32 analyzed participants, 5 participants were censored in PFS analysis because they were taken off treatment before their first radiographic progression.

Progression-Free Survival (PFS) is defined as the time from registration to the earlier of progression or death due to any cause. Participants alive without disease progression are censored at date of last disease evaluation.

Outcome measures

Outcome measures
Measure	Lamivudine n=27 Participants * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Progression Free Survival	56 days Interval 55.0 to 92.0

SECONDARY outcome

Timeframe: up to 2 years

Overall Survival (OS) is defined as the time from registration to death due to any cause, or censored at date last known alive.

Outcome measures

Outcome measures
Measure	Lamivudine n=36 Participants * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Overall Survival	186 days Interval 129.0 to 276.0

SECONDARY outcome

Timeframe: up to 2 years

Disease control rate (DCR) describes the percentage of patients with advanced cancer whose therapeutic intervention has led to a complete response (CR), partial response (PR), or stable disease (SD) as assessed by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria: * CR is the disappearance of all target lesions * PR is at least a 30% decrease in the sum of the longest diameters of target lesions * SD is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD) * PD is at least a 20% increase in the sum of the longest diameters of target lesions, and the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progressions.

Outcome measures

Outcome measures
Measure	Lamivudine n=36 Participants * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Overall Disease Control Rate	7 Participants

Adverse Events

Lamivudine

Serious events: 9 serious events

Other events: 31 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Lamivudine n=36 participants at risk * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Respiratory, thoracic and mediastinal disorders Dyspnea	11.1% 4/36 • Number of events 5 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Death NOS	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Vaginal bleeding	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Fever	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Cardiac disorders Atrial fibrillation	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Chest pain	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Fatigue	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Blood and lymphatic system disorders Leukocytosis	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Osteomyelitis	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Abdominal pain	5.6% 2/36 • Number of events 5 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Dehydration	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Small intestinal obstruction	8.3% 3/36 • Number of events 3 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Diarrhea	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Vomiting	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Nausea	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.

Other adverse events

Other adverse events
Measure	Lamivudine n=36 participants at risk * Lamivudine administered orally every 4 weeks * Treatment cycles will last 28 consecutive days * The dosage will be determine by the PI Lamivudine: This drug may help prevent the growth and spread of the cancer cells to other parts of the body.
Gastrointestinal disorders Abdominal distension	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Abdominal pain	38.9% 14/36 • Number of events 19 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Alanine aminotransferase increased	13.9% 5/36 • Number of events 10 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Alkaline phosphatase increased	47.2% 17/36 • Number of events 24 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Respiratory, thoracic and mediastinal disorders Allergic rhinitis	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Anal hemorrhage	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Blood and lymphatic system disorders Anemia	52.8% 19/36 • Number of events 50 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Anorexia	25.0% 9/36 • Number of events 11 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Psychiatric disorders Anxiety	11.1% 4/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Aspartate aminotransferase increased	36.1% 13/36 • Number of events 20 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Cardiac disorders Atrial fibrillation	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Musculoskeletal and connective tissue disorders Back pain	16.7% 6/36 • Number of events 6 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Bloating	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Blood bilirubin increased	19.4% 7/36 • Number of events 11 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Injury, poisoning and procedural complications Bruising	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Chills	8.3% 3/36 • Number of events 5 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Psychiatric disorders Confusion	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Constipation	27.8% 10/36 • Number of events 11 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Respiratory, thoracic and mediastinal disorders Cough	19.4% 7/36 • Number of events 9 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Creatinine increased	8.3% 3/36 • Number of events 5 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Dehydration	5.6% 2/36 • Number of events 3 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Psychiatric disorders Depression	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Diarrhea	22.2% 8/36 • Number of events 10 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Nervous system disorders Dizziness	11.1% 4/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Dry mouth	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Skin and subcutaneous tissue disorders Dry skin	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Nervous system disorders Dysgeusia	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Dyspepsia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Nervous system disorders Dysphasia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Respiratory, thoracic and mediastinal disorders Dyspnea	25.0% 9/36 • Number of events 10 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Edema face	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Edema limbs	5.6% 2/36 • Number of events 3 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Skin and subcutaneous tissue disorders Erythema multiforme	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Fatigue	55.6% 20/36 • Number of events 22 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Fever	16.7% 6/36 • Number of events 6 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Musculoskeletal and connective tissue disorders Flank pain	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Gait disturbance	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Gastroesophageal reflux disease	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Gastrointestinal disorders - Other	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Gastrointestinal pain	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	8.3% 3/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Nervous system disorders Headache	11.1% 4/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hyperglycemia	47.2% 17/36 • Number of events 53 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hyperkalemia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypermagnesemia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypernatremia	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Vascular disorders Hypertension	11.1% 4/36 • Number of events 6 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypoalbuminemia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Small intestinal obstruction	8.3% 3/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypocalcemia	13.9% 5/36 • Number of events 11 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypokalemia	2.8% 1/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypomagnesemia	11.1% 4/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hyponatremia	38.9% 14/36 • Number of events 23 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Metabolism and nutrition disorders Hypophosphatemia	22.2% 8/36 • Number of events 9 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Hypothermia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Psychiatric disorders Insomnia	8.3% 3/36 • Number of events 3 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Investigations - Other	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Irritability	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Infections and infestations Lip infection	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Infections and infestations Lung infection	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Lymphocyte count decreased	50.0% 18/36 • Number of events 54 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Malaise	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Musculoskeletal and connective tissue disorders Myalgia	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Nausea	41.7% 15/36 • Number of events 19 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Neutrophil count decreased	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Non-cardiac chest pain	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders Pain	41.7% 15/36 • Number of events 18 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Musculoskeletal and connective tissue disorders Pain in extremity	13.9% 5/36 • Number of events 6 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Cardiac disorders Palpitations	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Nervous system disorders Paresthesia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Reproductive system and breast disorders Perineal pain	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Nervous system disorders Peripheral sensory neuropathy	8.3% 3/36 • Number of events 3 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Platelet count decreased	13.9% 5/36 • Number of events 9 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Hepatobiliary disorders Portal vein thrombosis	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Respiratory, thoracic and mediastinal disorders Postnasal drip	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Renal and urinary disorders Proteinuria	5.6% 2/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Skin and subcutaneous tissue disorders Rash maculo-papular	8.3% 3/36 • Number of events 4 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Rectal fistula	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Rectal hemorrhage	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Reproductive system and breast disorders Scrotal pain	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Infections and infestations Sinusitis	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Infections and infestations Skin infection	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Skin and subcutaneous tissue disorders Skin/subcutaneous tissue disorders - Other	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Infections and infestations Urinary tract infection	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Renal and urinary disorders Urine discoloration	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Cardiac disorders Ventricular tachycardia	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Ear and labyrinth disorders Vertigo	5.6% 2/36 • Number of events 2 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Gastrointestinal disorders Vomiting	22.2% 8/36 • Number of events 10 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations Weight loss	5.6% 2/36 • Number of events 3 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Investigations White blood cell decreased	19.4% 7/36 • Number of events 13 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Injury, poisoning and procedural complications Wound complication	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Injury, poisoning and procedural complications Wound dehiscence	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
General disorders General disorders and administration site conditions - Other	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.
Skin and subcutaneous tissue disorders Pruritus	2.8% 1/36 • Number of events 1 • Adverse event data was collected from the start of protocol therapy up until participants were taken off study, a median of approximately 7 months. Adverse events were evaluated through regular investigator assessment and regular laboratory testing.

Additional Information

Aparna Parikh, MD

Massachusetts General Hospital

Phone: 617-339-8452

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place