A Phase 1 Study of AMV564 in Patients With Intermediate or High-Risk Myelodysplastic Syndromes

NCT03516591 | Completed Phase 1 FDA Drug

• 1 other identifier: AMV564-201
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 5

Brief Summary

An open label, Phase 1, study of AMV564 as monotherapy to assess the safety and efficacy in patients with Myelodysplastic Syndromes

Conditions

Myelodysplastic Syndrome (MDS)

Outcome Measures

Primary Outcomes (2)

• Dose limiting toxicity (Dose Escalation)

  Dose limiting toxicity to be measured by AEs and SAEs by dose level

  DLTs will be evaluated through 28 days for the 14-Day Continuous Intravenous Infusion Infusion regimen, and 35 days for the Intermittent Intravenous Dosing regimen

• Overall Response Rate (Dose Expansion)

  Overall response rate (ORR), defined as the proportion of patients who achieve a CR, marrow CR or PR by IWG criteria. Point estimates for ORR, along with the approximate lower 1-sided 90% confidence intervals, will be calculated.

  The treatment period will extend from initiation of AMV564 treatment until the Safety Follow Up visit (30 days after the end of infusion), or response assessment of the last induction cycle, whichever occurs later.

Study Arms (2)

Dose Escalation (3+3 design)

EXPERIMENTAL

A 3 + 3 design, with dose-escalation of AMV564, up to a Maximum Tolerated Dose (MTD) level. AMV564 will be tested as a 14-Day CIV regimen (14-Day Continuous Intravenous Infusion Regimen).

Drug: AMV564 14-Day CIV

Dose Expansion

EXPERIMENTAL

Following determination of the MTD of AMV564, the study will expand at the MTD or a dose level lower than the MTD to obtain initial estimates of response rates and additional information on safety.

Drug: AMV564 14-Day CIV

Interventions

AMV564 14-Day CIV DRUG

A 14-Day Continuous Intravenous Infusion regimen

Dose Escalation (3+3 design); Dose Expansion

Eligibility Criteria

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

You may qualify if:

• ≥ 18 years of age
• Diagnosis of MDS according to WHO 2016 criteria
• ECOG performance status of 0 or 1
• Intermediate-2 or high-risk disease per IPSS
• Fewer than 20% blasts in the bone marrow or peripheral blood
• Disease that is refractory to or relapsed from either a hypomethylating agent (e.g. decitabine or azacitidine) or a standard AML-type intensive regimen
• Adequate organ function
• Prior allogeneic transplant performed ≥ 3 months prior to first dose of AMV564 is allowed provided there is no evidence of active graft-versus-host disease (GVHD) and the patient has been off immunosuppressive therapy for ≥ 4 weeks.

You may not qualify if:

• History of, or known, central nervous system (CNS) disease involvement, or prior history of National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Events (CTCAE) Grade ≥ 3 drug-related CNS toxicity
• Prior allogeneic transplant if performed \< 3 months prior to first dose of AMV564, if patient has active GVHD, or if patient has not been off immunosuppressive
• Prior treatment with a therapeutic agent targeting CD33 (e.g. gemtuzumab ozogamicin, SGN-CD33A or AMG 330).

Sponsors & Collaborators

• Amphivena Therapeutics, Inc.: lead

Study Sites (5)

City of Hope Comprehensive Cancer Center

Duarte, California, 91010, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Washington University, Siteman Cancer Center

St Louis, Missouri, 63110, United States

Location

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 42310, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Electron Transport Complex IV

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Multienzyme Complexes; Multiprotein Complexes; Macromolecular Substances; Electron Transport Chain Complex Proteins; Enzymes; Enzymes and Coenzymes; Oxidoreductases; Proton Pumps; Cation Transport Proteins; Ion Pumps; Membrane Transport Proteins; Carrier Proteins; Proteins; Amino Acids, Peptides, and Proteins; Membrane Proteins

Study Officials

• Patrick Chun, MD

  Amphivena Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 24, 2018
• First Posted: May 4, 2018
• Study Start: June 22, 2018
• Primary Completion: July 31, 2020
• Study Completion: July 31, 2020
• Last Updated: October 12, 2021

Record last verified: 2021-10

Locations

US (5)