Clinical trial NCT04731675: An Open-Label Study of Intra-articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor of the Knee
Official title: An Adaptive, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Intra-articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor of the Knee
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Tenosynovial Giant Cell Tumor
Pigmented Villonodular Synovitis (PVNS)
Keywords: Tenosynovial Giant Cell Tumor (TGCT)
Pigmented villonodular synovitis (PVNS)
TGCT
PVNS
Lead sponsor: AmMax Bio, Inc.
Sponsor class: INDUSTRY
First posted: 2021-02-01
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: AMB-051-01 is a multicenter study with an adaptive design that will enroll subjects with Tenosynovial Giant Cell Tumor (TGCT) of the knee for 12 weeks of multiple-dose, open-label treatment with intra-articular AMB-05X.

Detailed description: AMB-05X drug substance is a human monoclonal antibody against the colony-stimulating factor 1 receptor (CSF1R). Study

Eligibility criteria: Inclusion Criteria:

1. Subject ≥ 18 years
2. A confirmed diagnosis of tenosynovial giant cell tumor (TGCT) of the knee joint
3. Measurable disease based on RECIST v1.1
4. Stable prescription of analgesic regimen
5. Negative urine drug screen (UDS) at Screening and Baseline
6. Women of childbearing potential must have a negative pregnancy test
7. Agrees to follow contraception guidelines
8. Adequate hematologic, hepatic, and renal function, at Screening
9. Willing and able to complete self-assessment instruments throughout the study

Exclusion Criteria:

1. Prior investigational drug use within 4 weeks or 5 half-lives of Baseline
2. Previous use of therapeutics targeting CSF1 or CSF1R or oral tyrosine kinase inhibitors
3. History of extensive knee surgery
4. Active cancer (either currently or within 1 year before Baseline) that requires therapy (e.g., surgery, chemotherapy, or radiation therapy)
5. Metastatic TGCT
6. Hepatitis C virus (HCV) or hepatitis B virus (HBV) or 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
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

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events
timeFrame: 24 weeks
Secondary outcomes:
measure: Tumor Response Based on RECIST Version 1.1
timeFrame: Week 12
measure: Tumor Response Based on Tumor Volume (TV)
timeFrame: Week 12
measure: Mean Change From Baseline in Range of Motion (ROM): Flexion
timeFrame: Week 12
measure: Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Score
timeFrame: Week 12
measure: Mean Change From Baseline in Worst Stiffness Numeric Rating Scale (NRS) Score
timeFrame: Week 12
measure: Mean Change From Baseline in the Brief Pain Inventory (BPI) Pain Interference Index Score
timeFrame: Week 12
measure: EuroQol 5 Dimension 5 Level (EQ-5D-5L) Health Assessment
timeFrame: Week 12
measure: Percentage of Subjects Who Respond With a Decrease of at Least 30% in Mean Brief Pain Inventory (BPI) Score
timeFrame: week 12
measure: Serum Colony Stimulating Factor 1 (CSF1) Levels
timeFrame: week 10
measure: Serum AMB-05X Levels
timeFrame: week 10
measure: Serum Anti-AMB-05X Antibody Levels
timeFrame: week 10
measure: Synovial Fluid CSF1
timeFrame: Week 10
measure: Synovial AMB-05X Levels
timeFrame: Week 10
measure: Synovial Anti-AMB-05X Antibody (ADA) Levels
timeFrame: Week 10
measure: Proportions of Subjects Who Achieved Objective Response as Their Best Overall Tumor Response, Per Modified Response Evaluation Criteria in Solid Tumors (RECIST).
timeFrame: 12 Weeks
Other outcomes:
Locations:
city: Columbus
country: United States
facility: AmMax Bio Clinical Site
state: Ohio
city: Leiden
country: Netherlands
facility: AmMax Bio Clinical Site
city: Warsaw
country: Poland
facility: AmMax Bio Clinical Site
city: Dnipro
country: Ukraine
facility: AmMax Bio Clinical Site
city: Kharkiv
country: Ukraine
facility: AmMax Bio Clinical Site
city: Kyiv
country: Ukraine
facility: AmMax Bio Clinical Site
Study officials:
affiliation: AmMax Bio, Inc.
name: Dorothy Nguyen, MD
role: STUDY_CHAIR
References: