A Study to evaluate treatment with intravenous AMB-05X in subjects with Tenosynovial Giant Cell Tumor
- Conditions
- Tenosynovial Giant Cell TumorMedDRA version: 21.0Level: LLTClassification code 10018253Term: Giant cell tumor of tendon sheathSystem Organ Class: 100000004864Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2020-004870-22-NL
- Lead Sponsor
- AmMax Bio., Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 36
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
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: to evaluate the safety, efficacy, and PK of AMB-05X in the treatment of TGCT;Secondary Objective: not applicable;Primary end point(s): Frequency and severity of reported treatment-emergent adverse events<br>(TEAEs)<br>;Timepoint(s) of evaluation of this end point: at Week 12
- Secondary Outcome Measures
Name Time Method