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

NCT04731675 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: AMB-051-01
• Study Type: interventional
• Target: 11
• Locations: 4 countries
• Sites: 6

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.

Conditions

Tenosynovial Giant Cell Tumor; Pigmented Villonodular Synovitis (PVNS)

Keywords

Tenosynovial Giant Cell Tumor (TGCT); Pigmented villonodular synovitis (PVNS); TGCT; PVNS

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-emergent Adverse Events

  Frequency and severity of reported treatment-emergent adverse events

  24 weeks

Secondary Outcomes (15)

• Tumor Response Based on RECIST Version 1.1

  Week 12

• Tumor Response Based on Tumor Volume (TV)

  Week 12

• Mean Change From Baseline in Range of Motion (ROM): Flexion

  Week 12

• Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Score

  Week 12

• Mean Change From Baseline in Worst Stiffness Numeric Rating Scale (NRS) Score

  Week 12

Study Arms (1)

AMB-05X

EXPERIMENTAL

Subjects will receive an intra-articular injection of AMB-05X once every 2 weeks for 12 weeks (for 6 treatments total).

Biological: AMB-05X

Interventions

AMB-05X BIOLOGICAL

AMB-05X is a fully human antibody antagonist (immunoglobulin G, type 2 \[IgG2\]) specific to the extracellular domain of human CSF1R

AMB-05X

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AmMax Bio, Inc.: lead

Study Sites (6)

AmMax Bio Clinical Site

Columbus, Ohio, 43201, United States

Location

AmMax Bio Clinical Site

Leiden, Netherlands

Location

AmMax Bio Clinical Site

Warsaw, Poland

Location

AmMax Bio Clinical Site

Dnipro, Ukraine

Location

AmMax Bio Clinical Site

Kharkiv, Ukraine

Location

AmMax Bio Clinical Site

Kyiv, Ukraine

Location

MeSH Terms

Conditions

Giant Cell Tumor of Tendon Sheath; Synovitis, Pigmented Villonodular

Condition Hierarchy (Ancestors)

Giant Cell Tumors; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Results Point of Contact

• Title: Dorothy Nguyen, MD
• Organization: AmMax Bio, Inc.

clinical@ammaxbio.com

(650)285-6560

Study Officials

• Dorothy Nguyen, MD

  AmMax Bio, Inc.

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2021
• First Posted: February 1, 2021
• Study Start: May 25, 2021
• Primary Completion: May 5, 2022
• Study Completion: May 5, 2022
• Last Updated: June 27, 2024
• Results First Posted: June 27, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); NL (1); UA (3); PL (1)