A Phase 1b, Open-Label, Pharmacokinetic, Pharmacodynamic, Safety and Tolerability Study of CERC-006 in Adults (Aged 18-31 Years) With Complex Lymphatic Malformations

Avalo Therapeutics, Inc.3 sites in 1 countrySeptember 3, 2021

ConditionsLymphatic Malformation

InterventionsCERC-006

Overview

Phase: Phase 1
Intervention: CERC-006
Conditions: Lymphatic Malformation
Sponsor: Avalo Therapeutics, Inc.
Locations: 3
Primary Endpoint: Safety and Tolerability of CERC-006 in Adults (Aged 18-31 Years) with Active, Moderate to Severe Complex Lymphatic Malformations
Status: Withdrawn
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The primary objective of this study is to evaluate the safety and tolerability of CERC-006 in adults (aged 18-31 years) with active, moderate to severe complex lymphatic malformations.

Registry

clinicaltrials.gov

Start Date

September 3, 2021

End Date

June 2022

Last Updated

4 years ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Avalo Therapeutics, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Participants must fulfill the following requirements to be eligible for the study:
•Participant is 18 to 31 years of age (inclusive) at the time of consent.
•Participant has a verified diagnosis of a complex lymphatic malformation. Other moderate to severe vascular anomalies with associated lymphatic involvement will be considered, with approval by the study medical monitor.
•Participant's complex lymphatic malformation is considered, in the opinion of the investigator, to be moderate to severe.
•Participant has adequate liver function defined as:
•Total bilirubin (sum of conjugated and unconjugated) ≤1.5 × upper limit of normal (ULN)
•Aspartate transaminase/Alanine aminotransferase (AST/ALT) \<5 × ULN
•Serum albumin \> 2 g/dL
•Participant has fasting low-density lipoprotein (LDL) of \<160 mg/dL.
•Participant has adequate bone marrow function defined as:

Exclusion Criteria

•The presence of any of the following criteria excludes a participant from the study:
•Participant has a concurrent severe or uncontrolled medical disorder, which could compromise participation in the study.
•Participant has significant impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of CERC-
•Participant has taken any medication that is a strong cytochrome P450 3A4 (CYP3A4) enzyme inducer or inhibitor within 2 weeks prior to first dose of study drug, or in the opinion of the Investigator, subject may require such medication during the study
•Participant is receiving chronic treatment with systemic steroids or another immunosuppressive agent, or in the opinion of the Investigator, subject may require such medication during the study
•Participant has undergone myelosuppressive chemotherapy within 2 weeks, or radiation within 4 weeks prior to first dose of study drug.
•Participant has a known history of uncontrolled hypertension, cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, heart failure, exercise-related cardiac events including syncope and pre-syncope, or a known family history of sudden cardiac death or ventricular arrhythmia.
•Participant has received treatment with a medication that has the potential to prolong the QT interval within 1 week prior to the first dose of study drug, or in the opinion of the Investigator, subject may require such medication during the study.

Arms & Interventions

Cohort 1: CERC-006 (0.5 mg)

Approximately 5 participants will receive CERC-006 at a dose of 0.5 mg twice daily for 28 days.

Intervention: CERC-006

Cohort 2: CERC-006 (1 mg)

Following a safety review, if there are no clinically important safety findings in Cohort 1, a second cohort of approximately 5 participants will be enrolled to receive CERC-006 at a dose of 1 mg twice daily for 28 days.

Intervention: CERC-006

Outcomes

Primary Outcomes

Safety and Tolerability of CERC-006 in Adults (Aged 18-31 Years) with Active, Moderate to Severe Complex Lymphatic Malformations

Time Frame: Up to approximately 6 weeks

Safety and tolerability will be assessed by incidence of adverse events (AEs). Clinically significant changes from baseline in vital signs, ocular exams, cardiology tests including echocardiogram (ECHO) and electrocardiogram (ECG), and clinical laboratory results will be recorded as AEs.

Secondary Outcomes

Serum Concentrations of CERC-006(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
Change from Baseline in Karnofsky Performance Status Score(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
Change from Baseline in Pain Scale Assessment(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
Change from Baseline in Levels of mTOR Related Pathway Biomarkers(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
Change from Baseline in 36-Item Short Form Health Survey (SF-36) Score(Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks))
Change from baseline to end of treatment in radiologic assessment, if clinically indicated and/or clinical signs/symptoms of disease(Baseline (Screening) up to 3 days after last dose of study drug (up to approximately 8 weeks))