Tenosynovial Giant Cell Tumor 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. Male or female =18 years of age. 2. Able to communicate well with study staff, understand and comply with the requirements of the study, and read and voluntarily sign the informed consent form (ICF) and the Health Insurance Portability and Accountability Act (HIPAA) authorization (if applicable) before the conduct of any study-specific procedures. 3. TGCT that meets both of the following criteria: a. Diagnosis has been histologically confirmed by a pathologist. If the diagnosis has not been previously histologically confirmed, biopsy with histological confirmation is required before enrollment. b. Has not been surgically resected either because surgical resection would be associated with potentially worsening functional limitation / severe morbidity (locally advanced disease) or because the subject declined surgery. 4. Measurable disease as defined by RECIST v1.1 (except with a minimum size of 2 cm), assessed from MRI scans by a central radiologist. 5. Symptomatic disease defined as one or both of the following: a. A score of at least 4 on the Worst Pain NRS at Screening. b. A score of at least 4 on the Worst Stiffness NRS at Screening. 6. Stable prescription of analgesic regimen during the 2 weeks before Baseline. 7. Women of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline. 8. Agrees to follow contraception guidelines (see Section 5.3). 9. Adequate hematologic, hepatic, and renal function at Screening, defined by: • Absolute neutrophil count = 1.5 × 109/L • Aspartate aminotransferase (AST) and alanine aminotransferase (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 10. Willing and able to complete the PROMIS Physical Function Scale, Worst Stiffness NRS, BPI, and EQ-5D-5L 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 27 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 9
Exclusion criteria
Exclusion criteria: 1. Use of any investigational drug within 4 weeks or 5 half-lives (whichever is longer) before Baseline. 2. Any previous use of pexidartinib or any biologic treatment targeting CSF1 or CSF1R. Use of an oral tyrosine kinase inhibitor other than pexidartinib (eg, imatinib or nilotinib) within 3 months before Baseline. 3. Active cancer (either currently or within 1 year before Baseline) that requires/required therapy (eg, 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. 4. Known metastatic TGCT. 5. Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with human immunodeficiency virus (HIV). 6. Known active tuberculosis (TB). 7. 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 the subject’s results. 8. A woman who is breastfeeding. 9. A screening Fridericia-corrected QT interval (QTcF) =450 ms (men) or =470 ms (women) 10. MRI contraindications (eg, pacemaker, loose metallic implants) 11. History of hypersensitivity to any ingredient in the study drug. 12. History of drug or alcohol abuse within 3 months before Baseline. 13. Has 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, interfere with interpretation of study results, or, in the Investigator’s opinion, make the subject inappropriate for this study. 14. A subject who, in the opinion of the Investigator, should not participate in this study for any reason, including instances where the subject’s stability or ability to comply with study requirements is in question.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: to evaluate the efficacy, safety, and PK of AMB-05X in the treatment of TGCT;Secondary Objective: not applicable;Primary end point(s): 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);Timepoint(s) of evaluation of this end point: at Week 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy The following efficacy endpoints will be assessed at Week 12: • 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 cavity • Mean change from Baseline in range of motion (ROM) score • Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score • Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score • Percentage of subjects who respond with a decrease of at least 30% in mean Brief Pain Inventory (BPI) score from Baseline • Mean change from Baseline in BPI score • Mean change from Baseline in Worst Pain NRS score • Mean change from Baseline in EQ-5D-5L health assessment Safety • Frequency and severity of reported treatment-emergent adverse events (TEAEs) Pharmacokinetics and Pharmacodynamics • Serum (and optional synovial) AMB-05X levels • Serum (and optional synovial) AMB-05X–binding anti-drug antibody (ADA) levels • Serum (and optional synovial) colony-stimulating factor 1 (CSF1) levels;Timepoint(s) of evaluation of this end point: DMC will begin to review study data after the first 3 subjects complete Week 6 and continue to review data throughout the study (each time at least 3 additional subjects [from any cohort] complete Week 12) | — |
Countries
Czechia, Czech Republic, Germany, Hungary, Netherlands, Poland, Ukraine, United States
Contacts
Public ContactSenior Director, Clinical Operation
AmMax Bio., Inc.
Outcome results
None listed