Clinical trial NCT05349643: A Study to Evaluate Safety and Efficacy of AMB-05X Injections in Subjects With TGCT
Official title: A Phase 2, Open-Label, Adaptive, Dose-Ranging Study With Long-Term Extension to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Intra Articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pigmented Villonodular Synovitis
TGCT
Tenosynovial Giant Cell Tumor
Keywords: Pigmented Villonodular Synovitis
Tenosynovial Giant Cell Tumor
TGCT
PVNS
Lead sponsor: AmMax Bio, Inc.
Sponsor class: INDUSTRY
First posted: 2022-04-27
Enrollment: 20
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-051-07 is an open-label, adaptive, dose-ranging study with long-term extension in adults with tenosynovial giant cell tumor

Detailed description: AMB-051-07 is an open-label, adaptive, dose-ranging study with long-term extension which will enroll up to 48 adult subjects with tenosynovial giant cell tumor for intra-articular doses over a 24-week dosing period (Part 1) with a Part 2 open-label extension of 6 treatment and/or observational cycles of 12 weeks each followed by 12 weeks of follow-up.

Eligibility criteria: Inclusion Criteria:

1. Subject ≥ 18 years
2. TGCT with only 1 joint involvement
3. Symptomatic Measurable disease of at least 1 cm based on RECIST v1.1
4. Stable prescription of analgesic regimen
5. Agrees to follow contraception guidelines
6. Women of childbearing potential must have a negative pregnancy test
7. Adequate hematologic, hepatic, and renal function

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 within 3 months prior to Baseline
3. History of extensive or reconstructive surgery on the affected joint
4. Active cancer (either currently or within 3 mo before Baseline) that requires/required therapy (e.g., surgery, chemotherapy, or radiation therapy)
5. Metastatic or malignant transformation of 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) ≥ 470 ms
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 baseline
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: Objective Response Rate - Part 1
timeFrame: Part 1 Week 24
measure: Treatment-emergent adverse events
timeFrame: Part 1 Week 24 and Part 2 Week 72
Secondary outcomes:
measure: Objective Response Rate
timeFrame: Part 2 Week 72
measure: Objective Response Rate (AMB-051-01 subjects)
timeFrame: Part 2 Week 72
measure: Objective Response Rate (ORR) per modified Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: Part 1 Week 24
measure: Objective Response Rate (ORR) per modified Response Evaluation Criteria in Solid Tumors Version 1.1
timeFrame: Part 2 Week 72
measure: Tumor response based on tumor volume score (TVS)
timeFrame: Part 1 Week 24
measure: Tumor response based on tumor volume score (TVS)
timeFrame: Part 2 Week 72
measure: Duration of Response by Response Evaluation Criteria in Solid Tumors
timeFrame: Part 1 Week 24
measure: Duration of Response by Response Evaluation Criteria in Solid Tumors v1.1
timeFrame: Part 2 Week 72
measure: Duration of Response by Modified Response Evaluation Criteria in Solid Tumors
timeFrame: Part 1 Week 24
measure: Duration of Response by Modified Response Evaluation Criteria in Solid Tumors
timeFrame: Part 2 Week 72
measure: Duration of Response by tumor volume score
timeFrame: Part 1 Week 24
measure: Duration of Response by tumor volume score
timeFrame: Part 2 Week 72
measure: Time to Response by Response Evaluation Criteria in Solid Tumors
timeFrame: Part 1 Week 24
measure: Time to Response by Response Evaluation Criteria in Solid Tumors
timeFrame: Part 2 Week 72
measure: Time to Response by Modified Response Evaluation Criteria in Solid Tumors
timeFrame: Part 1 week 24
measure: Time to Response by Modified Response Evaluation Criteria in Solid Tumors
timeFrame: Part 2 Week 72
measure: Time to Response per tumor volume score
timeFrame: Part 1 Week 24
measure: Time to Response per tumor volume score
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in joint range of motion (ROM)
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in joint range of motion (ROM)
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in Worst Stiffness Numeric Rating Scale score
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in Worst Stiffness Numeric Rating Scale score
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in the Brief Pain Inventory (BPI) score
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in the Brief Pain Inventory (BPI) score
timeFrame: Part 2 Week 72
measure: Decrease of at least 30% in mean Brief Pain Inventory (BPI) from Baseline
timeFrame: Part 1 Week 24
measure: Decrease of at least 30% in mean Brief Pain Inventory (BPI) from Baseline
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in Patient-Reported Outcomes Measurement Information System Pain Interference score
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in Patient-Reported Outcomes Measurement Information System Pain Interference score
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in Patient Global Impression of Change in Physical Functioning for capacity to perform everyday tasks
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in Patient Global Impression of Change in Physical Functioning for capacity to perform everyday tasks
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in Patient Global Impression of Change in tenosynovial giant cell tumor-related stiffness score
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in Patient Global Impression of Change in tenosynovial giant cell tumor-related stiffness score
timeFrame: Part 2 Week 72
measure: Mean change from Baseline in Worst Pain Numeric Rating Scale score
timeFrame: Part 1 Week 24
measure: Mean change from Baseline in Worst Pain Numeric Rating Scale score
timeFrame: Part 2 Week 72
measure: EuroQol 5 Dimension 5 Level Health Assessment
timeFrame: Part 1 Week 24
measure: EuroQol 5 Dimension 5 Level Health Assessment
timeFrame: Part 2 Week 72
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: AmMax Bio, Clinical Site
state: California
city: Miami
country: United States
facility: AmMax Bio, Clinical Site
state: Florida
city: Houston
country: United States
facility: AmMax Bio, Clinical Site
state: Texas
city: Camperdown
country: Australia
facility: AmMax Bio, Clinical Site
city: Woolloongabba
country: Australia
facility: AmMax Bio, Clinical Site
city: Leiden
country: Netherlands
facility: AmMax Bio, Clinical Site
Study officials:
affiliation: AmMax Bio
name: Dorothy Nguyen, MD
role: STUDY_CHAIR
References: