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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02100839




Registration number
NCT02100839
Ethics application status
Date submitted
27/03/2014
Date registered
1/04/2014
Date last updated
29/12/2015

Titles & IDs
Public title
Safety Study of AEM-28 to Treat Refractory Hypercholesterolemia
Scientific title
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Doses of AEM-28 in Healthy Subjects and Patients With Refractory Hypercholesterolemia
Secondary ID [1] 0 0
LPMX-112
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypercholesterolemia 0 0
Hyperlipoproteinemia Type II 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AEM-28
Treatment: Drugs - Normal Saline

Experimental: AEM-28 - Single Ascending Dose: Single IV dose for each cohort; dose range 0.032 mg/mL to 3.54 mg/mL

Multiple Ascending Dose: Three (3) IV doses for each cohort, one (1) dose every two (2) weeks; dose range 1 mg/kg to 3.54 mg/kg.

Placebo comparator: Normal Saline - Single Ascending Dose: Single IV dose for each cohort.

Multiple Ascending Dose: Three (3) IV doses for each cohort, one (1) dose every two (2) weeks.


Treatment: Drugs: AEM-28
Solution for injection

Treatment: Drugs: Normal Saline
0.9% saline for injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Who Incurred at Least One Treatment Emergent Event
Timepoint [1] 0 0
Part A (SAD): Day -1 to Day 15; Part B (MAD): Day 1 to Day 57
Primary outcome [2] 0 0
Number of Participants Who Incurred Mild Treatment Emergent Adverse Events
Timepoint [2] 0 0
Part A (SAD): Day -1 to Day 15; Part B (MAD): Day 1 to Day 57
Primary outcome [3] 0 0
Number of Participants Who Incurred Moderate Treatment Emergent Events
Timepoint [3] 0 0
Part A (SAD): Day -1 to Day 15; Part B (MAD): Day 1 to Day 57
Secondary outcome [1] 0 0
Very Low Density Lipoprotein Cholesterol (VLDL-C) Percent Change
Timepoint [1] 0 0
Part A (SAD): Day 1 to Day 15; Part B (MAD): Day 1 to Day 57

Eligibility
Key inclusion criteria
Single Ascending Dose (SAD) Study:

* Male or female non-smoker, =18 and =55 years of age, with BMI >18.5 and < 32.0 kg/m²
* Total cholesterol greater or equal to 5.0 mmol/L (=194 mg/dL) at screening

Multiple Ascending Dose (MAD) Study:

* Male or female non-smoker, =18 and =75 years of age, with BMI >18.5 and < 35.0 kg/m²
* Diagnosis of refractory hypercholesterolemia with LDL cholesterol levels > 2.5 mmol/L (97 mg/mL) at screening.
* On stable lipid lowering therapy for = 8 weeks
* On stable diet for = 12 weeks.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
SAD Study:

* Any clinically significant abnormality or abnormal laboratory test results found during medical screening or positive test for hepatitis B, hepatitis C, or HIV found during medical screening.
* History of allergic reactions to diphenhydramine, ranitidine, methylprednisone or other related drugs, or history of significant allergic or hypersensitivity reaction (e.g. angioedema) to any substance.

MAD Study:

* Significant health problems within 6 months prior to screening, which in the opinion of the Medical Sub-Investigator would prevent the subject from participating in the study, including but not limited to: unstable coronary heart disease; transient ischemic attack; stroke; revascularization procedure; uncontrolled hyperthyroidism; coagulation disorder; peptic ulcers or GI bleeding; significant disease of the central nervous system; liver or renal disease.
* History of allergic reactions to diphenhydramine, ranitidine, methylprednisone or other related drugs, or history of significant allergic or hypersensitivity reaction (e.g. angioedema) to any substance.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/03/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/12/2014
Sample size
Target
Accrual to date
Final
52
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Linear Clinical Research Ltd. - Nedlands
Recruitment postcode(s) [1] 0 0
6009 - Nedlands

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
LipimetiX Development, LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of the first part of this study is to determine the safety and tolerability of a single dose of AEM-28, an apolipoprotein E mimetic, in subjects with high total cholesterol who are otherwise healthy subjects. The pharmacokinetics and pharmacodynamics of AEM-28 will also be evaluated.

The second part of this study will be a multiple ascending dose evaluation of AEM-28 in patients with refractory hypercholesterolemia.

AEM-28 has demonstrated significant lipid lowering activity and positive effects on the artery wall. AEM-28 is being developed for the treatment of homozygous familial hypercholesterolemia.
Trial website
https://clinicaltrials.gov/study/NCT02100839
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Janakan Krishnarajah, MBBS, FRACP
Address 0 0
Linear Clinical Research
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02100839