Trial Outcomes & Findings for ATX-101 in Advanced Dedifferentiated Liposarcoma and Leiomyosarcoma (NCT NCT05116683)

Last Updated: 2023-12-06

Results Overview

The study will evaluate the preliminary efficacy of ATX-101 in advanced L-sarcomas (LMS, LPS) by measuring the PFR (progression free rate) at 12 weeks (PFR12). Progression evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 and defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm (the appearance of one or more new lesions is also considered progression).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

12 weeks

Results posted on

2023-12-06

Participant Flow

Participant milestones

Participant milestones
Measure	ATX-101 Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	ATX-101 Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Overall Study Physician Decision	4

Baseline Characteristics

ATX-101 in Advanced Dedifferentiated Liposarcoma and Leiomyosarcoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ATX-101 n=4 Participants Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants
Age, Customized 18-49 years	0 Participants n=5 Participants
Age, Customized 50-59 years	2 Participants n=5 Participants
Age, Customized 60-69 years	0 Participants n=5 Participants
Age, Customized 70-79 years	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Three of four participants analyzed due to n=1 participant who expired prior to week 12 (stable disease at time of death).

Outcome measures

Outcome measures
Measure	ATX-101 n=3 Participants Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Progression Free Rate (PFR)	1 Participants

SECONDARY outcome

Timeframe: Up to approximately 7 months

Counted per adverse event basis by grade as evaluated by Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. The higher the grade, the more severe the event.

Outcome measures

Outcome measures
Measure	ATX-101 n=4 Participants Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Number of Adverse Events Grade 1	13 Adverse Events
Number of Adverse Events Grade 2	16 Adverse Events
Number of Adverse Events Grade 3	4 Adverse Events
Number of Adverse Events Grade 4	0 Adverse Events
Number of Adverse Events Grade 5	0 Adverse Events

SECONDARY outcome

Timeframe: Up to approximately 7 months

The percentage of patients whose cancer shrinks or disappears after treatment. This will be measured by the percentage of patients having a complete or partial response per RECIST Version 1.1. ORR = Complete Response (CR) + Partial Response (PR); CR defined as disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 mm and PR defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.

Outcome measures

Outcome measures
Measure	ATX-101 n=4 Participants Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Objective Response Rate (ORR)	0 Participants

SECONDARY outcome

Timeframe: Up to approximately 7 months

Population: Zero analyzed as zero participants demonstrated objective response.

Duration of response is measured from the time the measurement criteria for an objective response is recorded until the first date that recurrent or progressive disease is objectively documented.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to approximately 5 months

Median time from start treatment until the time of progression or death.

Outcome measures

Outcome measures
Measure	ATX-101 n=4 Participants Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Median Progression Free Survival (PFS)	2.46 months Interval 1.28 to 5.19

SECONDARY outcome

Timeframe: Up to approximately 7 months

Median time from start of treatment until the time of death from any cause.

Outcome measures

Outcome measures
Measure	ATX-101 n=4 Participants Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Median Overall Survival (OS)	4.40 Months Interval 2.53 to 6.64

Adverse Events

ATX-101

Serious events: 1 serious events

Other events: 4 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	ATX-101 n=4 participants at risk Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Renal and urinary disorders Acute kidney injury	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Cardiac disorders Atrial fibrillation	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Respiratory, thoracic and mediastinal disorders Dyspnea	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
General disorders Edema limbs	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).

Other adverse events

Other adverse events
Measure	ATX-101 n=4 participants at risk Patients will be treated with ATX-101 60 mg/m2 IV weekly in continuous 21 day cycles. Patients will receive premedication prior to the ATX-101 infusion to reduce the risk of infusion-related reactions. ATX-101: Patients will be given ATX-101 at 60 mg/m2 IV weekly in continuous 21 day cycles.
Blood and lymphatic system disorders Anemia	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Metabolism and nutrition disorders Anorexia	50.0% 2/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Investigations Blood bilirubin increased	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Respiratory, thoracic and mediastinal disorders Cough	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Gastrointestinal disorders Dry mouth	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Nervous system disorders Dysgeusia	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
General disorders Fatigue	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
General disorders Generalized edema	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
General disorders Infusion related reaction	75.0% 3/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Gastrointestinal disorders Nausea	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Skin and subcutaneous tissue disorders Periorbital edema	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Respiratory, thoracic and mediastinal disorders Pleural effusion	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Skin and subcutaneous tissue disorders Pruritus	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Skin and subcutaneous tissue disorders Rash maculo-papular	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Infections and infestations Upper respiratory infection	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Renal and urinary disorders Urinary tract obstruction	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).
Gastrointestinal disorders Vomiting	25.0% 1/4 • Up to approximately 7 months Adverse event assessment conducted at each follow up visit (day 1, 8, and 15 of each cycle).

Additional Information

Benjamin Izar, MD

Columbia University

Phone: 212-304-5871

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place