A Phase 2 Study of Intravenous AMB-05X in Tenosynovial Giant Cell Tumor Patients

NCT04938180 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: AMB-051-02
• Study Type: interventional
• Target: 4
• Locations: 3 countries
• Sites: 5

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.

Conditions

Tenosynovial Giant Cell Tumor; Pigmented Villonodular Synovitis; TGCT; PVNS - Pigmented Villonodular Synovitis

Keywords

Tenosynovial Giant Cell Tumor, TGCT, PVNS

Outcome Measures

Primary Outcomes (1)

• Treatment-emergent Adverse Events (TEAEs)

  The frequency and severity of reported TEAEs in Subjects with Tenosynovial Giant Cell Tumor (TGCT) receiving Intravenous AMB 05X.

  6 months

Secondary Outcomes (14)

• Overall Tumor Response (Objective Response) Per Response Evaluation Criteria in Solid Tumors (RECIST) v.1.1

  12 weeks

• Overall Response Based on Tumor Volume Score (TVS)

  24 weeks or ET visit

• Mean Change From Baseline Range of Motion (ROM) (Flexion, Knee) Scores

  week 12

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

  12 weeks

• Serum Cmax for AMB-05X at Week 10 Post Dose

  10 weeks

Study Arms (1)

Cohort A

EXPERIMENTAL

Each subject will receive a low dose of AMB-05X every 2 weeks, for a total of 6 doses over the 12-week treatment period.

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

Cohort A

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 TGCT
• Measurable disease based on RECIST v1.1
• Symptomatic disease
• Stable prescription of analgesic regimen
• 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
• Current or prior radiotherapy within 3 months before Baseline
• Current or prior active cancer within 3 years before Baseline that requires/required therapy (eg, surgery, chemotherapy, or radiation therapy)
• Known metastatic TGCT or malignant transformation of diffuse-type TGCT
• Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with human immuno deficiency virus (HIV)
• Known active tuberculosis (TB)
• Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history
• Women who are pregnant or breastfeeding
• Screening Fridericia-corrected QT interval(QTcF) ≥450ms (men) or ≥470ms (women)
• MRI contraindications (eg, pacemaker, loose metallic implants)
• History of hypersensitivity to any ingredient in the study drug
• History of drug or alcohol abuse within 3 months before Baseline
• 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
• 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
• 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

Sponsors & Collaborators

• AmMax Bio, Inc.: lead

Study Sites (5)

AmMax Bio Clinical Site

Budapest, Hungary

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

Limitations and Caveats

A decision was made in November 2021 to stop further enrollment into the study to allow development to focus on AMB-05X intra-articular administration for TGCT. A total of 4 subjects had been enrolled into the study at the time the decision to cease further enrollment was made, limiting the ability to interpret the resulting data.

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: June 16, 2021
• First Posted: June 24, 2021
• Study Start: September 16, 2021
• Primary Completion: April 20, 2022
• Study Completion: May 17, 2022
• Last Updated: May 24, 2024
• Results First Posted: May 24, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

HU (1); UA (3); PL (1)