Tenosynovial Giant Cell Tumor of the Knee MedDRA version: 21.0 Level: LLT Classification code 10018253 Term: Giant cell tumor of tendon sheath System Organ Class: 100000004864
Conditions
Sponsors
AmMax Bio., Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject = 18 years must be able to communicate well with study staff, understand and comply with the requirements of the study, and read and voluntarily sign the ICF and the Health Insurance Portability and Accountability Act (HIPAA) authorization, if applicable, prior to the conduct of any study-specific procedures. 2. A diagnosis of TGCT of the knee joint, that has been histologically confirmed either by a pathologist at the treating institution or by a central pathologist. If not previously confirmed, biopsy with histological confirmation is required. 3. Measurable disease of at least 1 cm and based on RECIST v1.1 (Eisenhauer, 2009) assessed from MRI scans by a central radiologist. Subjects with one knee joint involvement only, and only limited posterior extra-articular nodular TGCT lesions as assessed by central radiologist and tumor review committee. 4. Stable prescription of analgesic regimen during the 2 weeks before Baseline 5. Negative urine drug screen (UDS) at Screening and Baseline 6. Women of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline 7. Agrees to follow contraception guidelines (see Section 5.3) 8. Adequate hematologic, hepatic, and renal function, at Screening visit, defined by: • Absolute neutrophil count = 1.5 × 109/L • Aspartate aminotransferase or alanine aminotransferase (AST or ALT) = 1.5 × upper limit of normal (ULN) • Hemoglobin > 10 g/dL • Total bilirubin = 1.5 × ULN • Platelet count = 100 × 109/L • Serum creatinine = 1.5 × ULN 9. Willing and able to complete the BPI, Worst Stiffness NRS item, PROMIS Physical Function Scale, EQ-5D-5L, and other self-assessment instruments throughout the study Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 12
Exclusion criteria
Exclusion criteria: 1. Prior investigational drug use within 4 weeks or 5 half-lives (whichever is longer) before Baseline 2. Previous use of pexidartinib, any biologic treatment targeting CSF1 or CSF1R or oral tyrosine kinase inhibitors (e.g., imatinib or nilotinib). 3. History of extensive knee surgery; except for prior diagnostic synovectomy which is not exclusionary if at least 6 months prior to baseline 4. Active cancer (either currently or within 1 year before Baseline) that requires therapy (e.g., surgery, chemotherapy, or radiation therapy), with the exception of adequately treated basal or squamous cell carcinoma of the skin, melanoma in situ, carcinoma in situ of the cervix or breast, or prostate carcinoma not requiring treatment apart from active surveillance 5. Known metastatic TGCT 6. Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with 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 that, in the Investigator's opinion, would likely interfere with the subject’s study participation or the interpretation of his or her results 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: safety and efficacy of AB-05X post intra-articular injection of 150 mg biweekly for up to 12 weeks in the treatment of TGCT.;Secondary Objective: not applicable;Primary end point(s): 1. Frequency and severity of reported treatment-emergent adverse events (TEAEs) ;Timepoint(s) of evaluation of this end point: Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Evaluations will be done every 3 months during the study, at the Data Monitoring Committee Meetings.;Secondary end point(s): 1. The proportion of subjects who achieve an overall tumor response (objective response [OR], which includes both complete response [CR] and partial response [PR]), per the Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) at week 12 2. Proportion of subjects with overall response based on tumor volume score (TVS), a TGCT–specific method that calculates tumor volume as a percentage of the estimated maximally distended synovial activity 3. Mean change from Baseline in range of motion (ROM) score 4. Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score 5. Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score 6. Percentage of subjects who respond with a decrease of at least 30% in mean Brief Pain Inventory (BPI) score 7. Mean change from Baseline in BPI 8. Mean change from Baseline in Worst Pain NRS score 9. EQ-5D-5L Health Assessment 10. Serum and synovial CSF1 levels 11. Serum and synovial AMB-05X levels 12. Serum and synovial anti-AMB-05X antibody levels | — |
Countries
Netherlands, Poland, Ukraine, United States
Contacts
Public ContactSenior Director, Clinical Operation
AmMax Bio., Inc.
Outcome results
None listed