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

Primary Purpose

Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AMB-05X
Sponsored by
AmMax Bio, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Tenosynovial Giant Cell Tumor focused on measuring Tenosynovial Giant Cell Tumor, Pigmented villonodular synovitis, TGCT, PVNS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Subject ≥ 18 years
  2. A confirmed diagnosis of TGCT of the knee joint
  3. Measurable disease based on RECIST v1.1
  4. Stable prescription of analgesic regimen
  5. Negative urine drug screen (UDS) at Screening and Baseline
  6. Women of childbearing potential must have a negative pregnancy test
  7. Agrees to follow contraception guidelines
  8. Adequate hematologic, hepatic, and renal function, at Screening
  9. 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. Previous use of therapeutics targeting CSF1 or CSF1R or oral tyrosine kinase inhibitors
  3. History of extensive knee surgery
  4. Active cancer (either currently or within 1 year before Baseline) that requires therapy (e.g., surgery, chemotherapy, or radiation therapy)
  5. Metastatic 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) ≥ 450 ms (men) or ≥ 470 ms (women)
  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 the first dose of study drug
  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

Sites / Locations

  • AmMax Bio Clinical Site
  • AmMax Bio Clinical Site
  • AmMax Bio Clinical Site
  • AmMax Bio Clinical Site
  • AmMax Bio Clinical Site
  • AmMax Bio Clinical Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

AMB-05X

Arm Description

Subjects will receive an injection of AMB-05X once every 2 weeks for 12 weeks (for 6 treatments total). Based on ongoing review of the available safety, PK, PD, and efficacy data, the Sponsor may either increase or decrease the dose.

Outcomes

Primary Outcome Measures

Treatment-emergent adverse events
Frequency and severity of reported treatment-emergent adverse events
Tumor response based on RECIST
Proportion of subjects who achieve an overall tumor response per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

Secondary Outcome Measures

Tumor response based on tumor volume
Proportion of subjects with overall response based on tumor volume score
Mean change from Baseline in range of motion (ROM)
ROM of the joint will be assessed by qualified assessors. Measurements will be recorded in degrees. At baseline, the plane of movement with the smallest (worst) relative value will be identified; only this plane will be used for evaluating change in ROM subsequently.
Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
The PROMIS Physical Function Scale will be used to assess physical function. The scale ranges from 1 ('unable to do' or 'cannot do') to 5 ('without any difficulty' or 'not at all'), where higher scores represent better outcomes.
Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score
The Worst Stiffness NRS is a 1-item, self-administered questionnaire assessing the "worst" stiffness within the last 24 hours. The NRS for this item ranges from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Mean change from Baseline in Worst Pain NRS score
The Worst Pain NRS is a component of the Brief Pain Inventory assessing the "worst" pain in the last 24 hours. The NRS for this item ranges from 0 (no pain) to 10 (pain as bad as you can imagine).
Mean change from Baseline in the Brief Pain Inventory (BPI) score
Brief Pain Inventory (BPI) Short Form is a self-administered questionnaire used to evaluate the severity of a subject's pain and the impact of this pain on the subject's daily functioning. The subject is asked to rate their worst, least, average, and current pain intensity, list current treatments and their perceived effectiveness, and rate the degree that pain interferes with general activity, mood, walking ability, normal work, relations with other persons, sleep, and enjoyment of life on a scale from 0 to 10.
EQ-5D-5L Health Assessment
EQ-5D-5L is a widely used quality of life instrument that includes questions in each of 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The choices include 5 levels of severity for each domain followed by a general health visual analogue scale (VAS).

Full Information

First Posted
January 19, 2021
Last Updated
October 5, 2022
Sponsor
AmMax Bio, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04731675
Brief Title
An Open-Label Study of Intra-articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor of the Knee
Official Title
An Adaptive, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Intra-articular AMB-05X Injections in Subjects With Tenosynovial Giant Cell Tumor of the Knee
Study Type
Interventional

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
May 25, 2021 (Actual)
Primary Completion Date
May 5, 2022 (Actual)
Study Completion Date
October 31, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AmMax Bio, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
AMB-051-01 is a multicenter study with an adaptive design that will enroll approximately 12 subjects with TGCT of the knee for 12 weeks of multiple-dose, open-label treatment with AMB-05X.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis
Keywords
Tenosynovial Giant Cell Tumor, Pigmented villonodular synovitis, TGCT, PVNS

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
AMB-05X
Arm Type
Experimental
Arm Description
Subjects will receive an injection of AMB-05X once every 2 weeks for 12 weeks (for 6 treatments total). Based on ongoing review of the available safety, PK, PD, and efficacy data, the Sponsor may either increase or decrease the dose.
Intervention Type
Biological
Intervention Name(s)
AMB-05X
Intervention Description
A fully human monoclonal immunoglobulin (IgG2) directed against c-fms
Primary Outcome Measure Information:
Title
Treatment-emergent adverse events
Description
Frequency and severity of reported treatment-emergent adverse events
Time Frame
Week 12
Title
Tumor response based on RECIST
Description
Proportion of subjects who achieve an overall tumor response per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
Tumor response based on tumor volume
Description
Proportion of subjects with overall response based on tumor volume score
Time Frame
Week 12
Title
Mean change from Baseline in range of motion (ROM)
Description
ROM of the joint will be assessed by qualified assessors. Measurements will be recorded in degrees. At baseline, the plane of movement with the smallest (worst) relative value will be identified; only this plane will be used for evaluating change in ROM subsequently.
Time Frame
Week 12
Title
Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
Description
The PROMIS Physical Function Scale will be used to assess physical function. The scale ranges from 1 ('unable to do' or 'cannot do') to 5 ('without any difficulty' or 'not at all'), where higher scores represent better outcomes.
Time Frame
Week 12
Title
Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score
Description
The Worst Stiffness NRS is a 1-item, self-administered questionnaire assessing the "worst" stiffness within the last 24 hours. The NRS for this item ranges from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Time Frame
Week 12
Title
Mean change from Baseline in Worst Pain NRS score
Description
The Worst Pain NRS is a component of the Brief Pain Inventory assessing the "worst" pain in the last 24 hours. The NRS for this item ranges from 0 (no pain) to 10 (pain as bad as you can imagine).
Time Frame
Week 12
Title
Mean change from Baseline in the Brief Pain Inventory (BPI) score
Description
Brief Pain Inventory (BPI) Short Form is a self-administered questionnaire used to evaluate the severity of a subject's pain and the impact of this pain on the subject's daily functioning. The subject is asked to rate their worst, least, average, and current pain intensity, list current treatments and their perceived effectiveness, and rate the degree that pain interferes with general activity, mood, walking ability, normal work, relations with other persons, sleep, and enjoyment of life on a scale from 0 to 10.
Time Frame
Weeks 12
Title
EQ-5D-5L Health Assessment
Description
EQ-5D-5L is a widely used quality of life instrument that includes questions in each of 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The choices include 5 levels of severity for each domain followed by a general health visual analogue scale (VAS).
Time Frame
Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subject ≥ 18 years A confirmed diagnosis of 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dorothy Nguyen, MD
Organizational Affiliation
AmMax Bio, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
AmMax Bio Clinical Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43201
Country
United States
Facility Name
AmMax Bio Clinical Site
City
Leiden
Country
Netherlands
Facility Name
AmMax Bio Clinical Site
City
Warsaw
Country
Poland
Facility Name
AmMax Bio Clinical Site
City
Dnipro
Country
Ukraine
Facility Name
AmMax Bio Clinical Site
City
Kharkiv
Country
Ukraine
Facility Name
AmMax Bio Clinical Site
City
Kyiv
Country
Ukraine

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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