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Phase 2 N=11 Treatment

An Open-Label Study of Intra-articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor of the Knee

Tenosynovial Giant Cell Tumor · Pigmented Villonodular Synovitis (PVNS)

Enrolled (actual)
11
Serious AEs
0.0%
Results posted
Jun 2024
Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events — 7; 2 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
AMB-05X (Biological)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
AmMax Bio, Inc.
Primary completion
May 2022

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Participants With Treatment-emergent Adverse Events
7; 2
SECONDARY
Tumor Response Based on RECIST Version 1.1
3; 1
SECONDARY
Tumor Response Based on Tumor Volume (TV)
2; 1
SECONDARY
Mean Change From Baseline in Range of Motion (ROM): Flexion
7.1; 3.3
SECONDARY
Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Score
6.7; 8.7
SECONDARY
Mean Change From Baseline in Worst Stiffness Numeric Rating Scale (NRS) Score
-2.4; -2.3
SECONDARY
Mean Change From Baseline in the Brief Pain Inventory (BPI) Pain Interference Index Score
-2.8; -2.6
SECONDARY
EuroQol 5 Dimension 5 Level (EQ-5D-5L) Health Assessment
-0.9; -0.7
SECONDARY
Percentage of Subjects Who Respond With a Decrease of at Least 30% in Mean Brief Pain Inventory (BPI) Score
5; 2
SECONDARY
Serum Colony Stimulating Factor 1 (CSF1) Levels
503; 1030
SECONDARY
Serum AMB-05X Levels
2.1; 33.0
SECONDARY
Serum Anti-AMB-05X Antibody Levels
0; 0; 3; 8
SECONDARY
Synovial Fluid CSF1
37.1; 22.6
SECONDARY
Synovial AMB-05X Levels
36.3; 153
SECONDARY
Synovial Anti-AMB-05X Antibody (ADA) Levels
1; 0; 2; 8
SECONDARY
Proportions of Subjects Who Achieved Objective Response as Their Best Overall Tumor Response, Per Modified Response Evaluation Criteria in Solid Tumors (RECIST).
4; 1

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.

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

  • Prior investigational drug use within 4 weeks or 5 half-lives of Baseline
  • Previous use of therapeutics targeting CSF1 or CSF1R or oral tyrosine kinase inhibitors
  • History of extensive knee surgery
  • Active cancer (either currently or within 1 year before Baseline) that requires therapy (e.g., surgery, chemotherapy, or radiation therapy)
  • Metastatic TGCT
  • Hepatitis C virus (HCV) or hepatitis B virus (HBV) or human immunodeficiency virus (HIV)
  • Known active tuberculosis
  • Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history
  • Women who are breastfeeding
  • A screening Fridericia-corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women)
  • MRI contraindications (e.g., pacemaker, loose metallic implants)
  • History of hypersensitivity to any ingredient of the study drug
  • History of drug or alcohol abuse within 3 months before the first dose of study drug
  • 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
  • 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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04731675). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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