A Phase 2, Adaptive, Open-Label, Multiple-Dose, Dose-Escalation Study to Evaluate the Efficacy, Safety,Tolerability, and Pharmacokinetics of Intravenous AMB-05X in Subjects with Tenosynovial Giant Cell Tumor

Phase 2

Conditions: Giant cell tumor
10013361

Registration Number: NL-OMON50912

Lead Sponsor: AmMax Bio Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 6

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) 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.

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.