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Lymphatic MalformationPhase 1NCT04994002

A Pharmacokinetic, Pharmacodynamic, Safety and Tolerability Study of CERC-006 in Adults With Complex Lymphatic Malformations

A Phase 1 study testing CERC-006 in people with Lymphatic Malformation, run by Avalo Therapeutics, Inc. at 3 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 31 years
Conditions the study is for
Lymphatic Malformation
What is being tested?
CERC-006
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
3 sitesUnited States
Study dates
3 Sep 2021to Jun 2022
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 31 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • 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)
Show all 19Show fewer
  • 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:
  • Peripheral absolute neutrophil count (ANC) > 1000/µL
  • Hemoglobin > 8.0 g/dL
  • Platelet count ≥ 50,000/µL
  • Participant has adequate renal function defined as:
  • Serum creatinine ≤ 1.5 mg/dL or calculated creatinine clearance of > 50 mL/min according to the Cockcroft-Gault equation
  • Participant has agreed to and met the washout period as follows:
  • At least 14 days prior to initiation of CERC-006 if receiving sirolimus (also known as rapamycin), mitogen-activated protein kinase (MEK) inhibitors, phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA) inhibitors, interferon alfa 2b, vascular endothelial growth factor receptor 3 (VEGFR-3) inhibitors, and/or other systemic agents targeting lymphatic malformation
  • At least 7 days prior to initiation of CERC-006 if receiving topical agents targeting lymphatic malformation
  • Participant has a Karnofsky performance status of ≥50%.

Who cannot join

  • 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-006.
  • 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.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

Other trials in United States

About this study

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

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.

Being tested
CERC-006
Keywords
Lymphatic Malformation, Complex Lymphatic Malformations, CERC-006
Medical subject headings
Lymphatic Abnormalities

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CERC-006Drug

    Oral solution

Study groups in NCT04994002
Study groupRoleWhat they receive
Cohort 1: CERC-006 (0.5 mg)ExperimentalApproximately 5 participants will receive CERC-006 at a dose of 0.5 mg twice daily for 28 days.
Receives: Drug: CERC-006
Cohort 2: CERC-006 (1 mg)ExperimentalFollowing 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.
Receives: Drug: CERC-006

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    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.

    Measured: Up to approximately 6 weeks

Secondary outcomes (6)

  1. Serum Concentrations of CERC-006

    Measured: Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks)

  2. Change from Baseline in Levels of mTOR Related Pathway Biomarkers

    Measured: Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks)

  3. Change from baseline to end of treatment in radiologic assessment, if clinically indicated and/or clinical signs/symptoms of disease

    Radiologic disease assessment will be done if clinically indicated, at the discretion of the investigator.

    Measured: Baseline (Screening) up to 3 days after last dose of study drug (up to approximately 8 weeks)

  4. Change from Baseline in 36-Item Short Form Health Survey (SF-36) Score

    SF-36 score will measure eight scales: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. Scores on each scale will be directly transformed into a 0-100 scale, where a lower score indicates more disability.

    Measured: Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks)

  5. Change from Baseline in Karnofsky Performance Status Score

    Karnofsky Performance Status score will be measured on a scale from 0 percent (%) to 100%, where a lower percentage score indicates a worse disease state.

    Measured: Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks)

  6. Change from Baseline in Pain Scale Assessment

    Pain scale assessment will be done using a 100 mm Visual Analog Scale (VAS) from no pain (0 mm) to extreme pain (100 mm).

    Measured: Baseline (Day 1) up to 3 days after last dose of study drug (up to approximately 4 weeks)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Aug 2021
  2. Study start3 Sep 2021
  3. Primary completionJun 2022 (estimated)
  4. Study completionJun 2022 (estimated)

Why it stopped: Sponsor Decision

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04994002
Sponsor's protocol ID
CERC-006-LM-101
Status last verified
Mar 2022
First submitted
20 Jul 2021
Last updated
22 Mar 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CERC-006 in people with Lymphatic Malformation, run by Avalo Therapeutics, Inc. at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 22 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04994002.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Mar 2022.

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