Clinical Trials /

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

NCT04731675

Description:

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.

Related Conditions:
  • Tenosynovial Giant Cell Tumor
Recruiting Status:

Recruiting

Phase:

Phase 2

URL:

https://clinicaltrials.gov/show/NCT04731675

Trial Eligibility

Document

Title

  • 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

Clinical Trial IDs

  • ORG STUDY ID: AMB-051-01
  • NCT ID: NCT04731675

Conditions

  • Tenosynovial Giant Cell Tumor
  • Pigmented Villonodular Synovitis

Interventions

DrugSynonymsArms
AMB-05XAMB-05X

Purpose

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.

Trial Arms

NameTypeDescriptionInterventions
AMB-05XExperimentalSubjects 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.
  • AMB-05X

Eligibility Criteria

        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
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Treatment-emergent adverse events
Time Frame:Week 12
Safety Issue:
Description:Frequency and severity of reported treatment-emergent adverse events

Secondary Outcome Measures

Measure:Tumor response based on tumor volume
Time Frame:Week 12
Safety Issue:
Description:Proportion of subjects with overall response based on tumor volume score
Measure:Mean change from Baseline in range of motion (ROM)
Time Frame:Week 12
Safety Issue:
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.
Measure:Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
Time Frame:Week 12
Safety Issue:
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.
Measure:Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score
Time Frame:Week 12
Safety Issue:
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).
Measure:Mean change from Baseline in Worst Pain NRS score
Time Frame:Week 12
Safety Issue:
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).
Measure:Mean change from Baseline in the Brief Pain Inventory (BPI) score
Time Frame:Weeks 12
Safety Issue:
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.
Measure:EQ-5D-5L Health Assessment
Time Frame:Week 12
Safety Issue:
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).

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:AmMax Bio, Inc.

Trial Keywords

  • Tenosynovial Giant Cell Tumor
  • Pigmented villonodular synovitis
  • TGCT
  • PVNS

Last Updated

July 9, 2021