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
Interventions
Product Name: AMB-05X
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: not yet assigned
CAS Number: not assigned
Current Sponsor code: AMB-05X
Other descriptive name: AMG 820
Concentrat
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 and understand and comply with the requirements of the study. Reads and voluntarily signs 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 has been histologically confirmed by a pathologist. If the diagnosis has not been previously histologically confirmed, biopsy with histological confirmation is required before enrollment. 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. If subject uses prescription analgesic, subject must be on a stable prescription analgesic regimen during the 2 weeks before Baseline. 6. Agrees to follow contraception guidelines. 7. 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 (elevated bilirubin secondary to a known, relatively benign condition [eg, Gilbert’s syndrome] is not exclusionary) • Platelet count = 100 × 109/L • Serum creatinine = 1.5 × ULN 8. 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 39 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. Use of pexidartinib, any other oral tyrosine kinbase inhibitor (eg, imatinib or nilotinib), or any biologic treatment targeting CSF1 or CSF1R within 3 months before Baseline. 3. Current or prior radiotherapy within 3 months before Baseline. 4. Current or prior active cancer within 3 years before Baseline that requires/required therapy (eg, surgery, chemotherapy, or radiation therapy), except 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 or malignant transformation of diffuse-type TGCT. 6. Any history of complex or reconstructive surgery on the affected joint (eg, involving plates, screws, or metal implants). 7. Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with human immunodeficiency virus (HIV). 8. Known active tuberculosis (TB). 9. 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. 10. A woman who is pregnant or breastfeeding. For women of childbearing potential, a positive pregnancy test at either Screening or Baseline will be exclusionary. 11. A screening Fridericia-corrected QT interval (QTcF) =450 ms (men) or =470 ms (women) 12. MRI contraindications (eg, pacemaker, loose metallic implants) 13. History of hypersensitivity to any ingredient in the study drug. 14. History of drug or alcohol abuse within 3 months before Baseline. 15. 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. 16. A person who is held in detention as the result of a judicial or official decision or who is in a subordinate relationship to the Sponsor or Investigator. 17. 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 safety, efficacy and PK of intravenous (IV) AMB-05X in the treatment of TGCT;Secondary Objective: not applicable;Primary end point(s): Frequency and severity of reported treatment-emergent adverse events (TEAEs);Timepoint(s) of evaluation of this end point: A Sponsor data monitoring committee (DMC) composed of qualified medical/clinical representatives from the Sponsor will review the available safety, tolerability, PK, pharmacodynamics (PD), and efficacy data on an ongoing basis and provide recommendations regarding appropriate next steps in study conduct. The DMC will pay special attention to any clinically significant adverse events (AEs). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy The following efficacy endpoints will be assessed at Week 12: • 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) (Eisenhauer, 2009) • 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 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: The DMC will begin to review study data after the first 3 subjects complete Week 6 and will continue to review data throughout the study (each time 3 additional subjects [from any cohort] complete Week 12). Whenever a new dose is instituted during the study, the DMC will again review the available data once 3 subjects have completed Week 6 at the new dose. | — |
Countries
Czechia, Czech Republic, Germany, Hungary, Netherlands, Poland, Ukraine, United States
Contacts
Public ContactSenior Director, Clinical Operation
AmMax Bio., Inc.
Outcome results
None listed