An Open-Label Study of Intra-articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor of the Knee

AmMax Bio, Inc.11 enrolled

Overview

AMB-051-01 is a multicenter study with an adaptive design that will enroll subjects with Tenosynovial Giant Cell Tumor (TGCT) of the knee for 12 weeks of multiple-dose, open-label treatment with intra-articular AMB-05X.

AMB-05X drug substance is a human monoclonal antibody against the colony-stimulating factor 1 receptor (CSF1R). Study

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Tenosynovial Giant Cell Tumor Pigmented Villonodular Synovitis (PVNS)

Interventions

AMB-05XBIOLOGICAL

AMB-05X is a fully human antibody antagonist (immunoglobulin G, type 2 \[IgG2\]) specific to the extracellular domain of human CSF1R

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Subject ≥ 18 years 2. A confirmed diagnosis of tenosynovial giant cell tumor (TGCT) of the knee joint 3. Measurable disease based on RECIST v1.1 4. Stable prescription of analgesic regimen 5. Negative urine drug screen (UDS) at Screening and Baseline 6. Women of childbearing potential must have a negative pregnancy test 7. Agrees to follow contraception guidelines 8. Adequate hematologic, hepatic, and renal function, at Screening 9. Willing and able to complete self-assessment instruments throughout the study Exclusion Criteria: 1. Prior investigational drug use within 4 weeks or 5 half-lives of Baseline 2. Previous use of therapeutics targeting CSF1 or CSF1R or oral tyrosine kinase inhibitors 3. History of extensive knee surgery 4. Active cancer (either currently or within 1 year before Baseline) that requires therapy (e.g., surgery, chemotherapy, or radiation therapy) 5. Metastatic TGCT 6. Hepatitis C virus (HCV) or hepatitis B virus (HBV) or human immunodeficiency virus (HIV) 7. Known active tuberculosis 8. Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history 9. Women who are breastfeeding 10. A screening Fridericia-corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women) 11. MRI contraindications (e.g., pacemaker, loose metallic implants) 12. History of hypersensitivity to any ingredient of the study drug 13. History of drug or alcohol abuse within 3 months before the first dose of study drug 14. Any other severe acute or chronic medical or psychiatric condition or clinically significant laboratory abnormality that may increase the risk associated with study participation/treatment or interfere with interpretation of study results and, in the Investigator's opinion, make the subject inappropriate for this study 15. Subjects who, in the Investigator's opinion, should not participate in the study for any reason, including if there is a question about their ability to comply with study requirements

Locations (6)

AmMax Bio Clinical Site

Columbus, Ohio, United States

AmMax Bio Clinical Site

Leiden, Netherlands

AmMax Bio Clinical Site

Warsaw, Poland

AmMax Bio Clinical Site

Dnipro, Ukraine

Outcomes

Primary Outcomes

Number of Participants With Treatment-emergent Adverse Events

Frequency and severity of reported treatment-emergent adverse events

Time frame: 24 weeks

Secondary Outcomes

Tumor Response Based on RECIST Version 1.1

Proportion of subjects who achieve an overall tumor response (Objective Response or OR) per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 at week 12, Objective Response is defined as complete response \[CR\] and partial response \[PR\]) per RECIST v1.1 (Eisenhauer 2009) at Week 12. Complete response: disappearance of all target lesions; Partial Response: \>/=30% decrease in the sum of diameters of target lesions.

Time frame: Week 12

Tumor Response Based on Tumor Volume (TV)

Proportion of subjects with overall response based on tumor volume: Complete response (CR): lesion is completely gone; Partial response (PR): \>/=50% decrease in tumor volume relative to baseline; Progressive disease (PD): \>/=30% increase in tumor volume relative to the lowest volume during the study; Stable disease (SD): does not meet any of the other classifications. Tumor response based on tumor volume score ("TVS") is defined as CR or PR.

Time frame: Week 12

Mean Change From Baseline in Range of Motion (ROM): Flexion

ROM of the joint will be assessed by qualified assessors. Measurements will be recorded in degrees. At baseline, the plane of movement with the smallest (worst) relative value will be identified; only this plane will be used for evaluating change in ROM subsequently.

Time frame: Week 12

Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Score

The PROMIS Physical Function Scale will be used to assess physical function. The scale incudes 10 questions, with scores that range from 1 to 5, where higher scores represent better outcomes. Five questions address the degree to which the subject's health limits certain physical activities, and subjects select a response to each question that ranges from 1 ("cannot do") to 5 ("not at all"). Five additional questions address the degree to which the subject is able to perform certain physical activities, and subjects select a response to each question that ranges from 1 ("cannot do") to 5 ("without any difficulty"). The score range for the PROMIS is 10 to 50, with higher scores indicated worse physical function.

Data from ClinicalTrials.gov

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