Clinical trial NCT04938180: A Phase 2 Study of Intravenous AMB-05X in Tenosynovial Giant Cell Tumor Patients
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Tenosynovial Giant Cell Tumor
Pigmented Villonodular Synovitis
TGCT
PVNS - Pigmented Villonodular Synovitis
Keywords: Tenosynovial Giant Cell Tumor, TGCT, PVNS
Lead sponsor: AmMax Bio, Inc.
Sponsor class: INDUSTRY
First posted: 2021-06-24
Enrollment: 4
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: The purpose of this Phase 2, open-label, multiple-dose, dose-escalation study is to evaluate intravenous AMB-05X in the treatment of subjects with TGCT.

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

Eligibility criteria: Inclusion Criteria:

1. Subject ≥ 18 years
2. A confirmed diagnosis of TGCT
3. Measurable disease based on RECIST v1.1
4. Symptomatic disease
5. Stable prescription of analgesic regimen
6. Agrees to follow contraception guidelines
7. Adequate hematologic, hepatic, and renal function, at Screening
8. 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. Current or prior radiotherapy within 3 months before Baseline
3. Current or prior active cancer within 3 years before Baseline that requires/required therapy (eg, surgery, chemotherapy, or radiation therapy)
4. Known metastatic TGCT or malignant transformation of diffuse-type TGCT
5. Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with human immuno deficiency 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
8. Women who are pregnant or breastfeeding
9. Screening Fridericia-corrected QT interval(QTcF) ≥450ms (men) or ≥470ms (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 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
15. A subject who, in the opinion of the Investigator, should not participate in the study for any reason, including if there is a question about the subject's ability to comply with study requirements

Primary outcomes:
measure: Treatment-emergent Adverse Events (TEAEs)
timeFrame: 6 months
Secondary outcomes:
measure: Overall Tumor Response (Objective Response) Per Response Evaluation Criteria in Solid Tumors (RECIST) v.1.1
timeFrame: 12 weeks
measure: Overall Response Based on Tumor Volume Score (TVS)
timeFrame: 24 weeks or ET visit
measure: Mean Change From Baseline Range of Motion (ROM) (Flexion, Knee) Scores
timeFrame: week 12
measure: Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Score
timeFrame: 12 weeks
measure: Serum Cmax for AMB-05X at Week 10 Post Dose
timeFrame: 10 weeks
measure: Number of Subjects With Anti-Drug Antibodies to AMB-05X at Week 10
timeFrame: 10 weeks
measure: Mean Change From Baseline in Worst Stiffness Numeric Rating Scale (NRS) Score
timeFrame: 12 weeks
measure: Brief Pain Inventory Pain Severity Domain Score
timeFrame: 12 weeks
measure: Mean Change From Baseline in Brief Pain Inventory Severity Interference
timeFrame: 12 weeks
measure: Worst Pain Numeric Rating Scale Score
timeFrame: 12 weeks
measure: EuroQol 5 Dimension 5 Level Health Assessment
timeFrame: 12 weeks
measure: Colony-stimulating Factor 1 Levels
timeFrame: 10 weeks
measure: Mean Change From Baseline Rnge of Motion (Flexion, Ankle) Scores
timeFrame: week 12
measure: Serum AMB-05X-Binding Anti-drug Antibody (ADA) Levels
timeFrame: Week 12
Other outcomes:
Locations:
city: Budapest
country: Hungary
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: