Multiple Ascending Dose Study of SPC5001 in Treatment of Healthy Subjects and Subjects With FH

Hypercholesterolemia Clinical Trial

Official title:

A First-in-Human (FIH), Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SPC5001 Administered to Healthy Subjects and Subjects With Familial Hypercholesterolemia (FH)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01350960
• Other study ID #: SPC5001-901
• Secondary ID: EudraCT 2011-000
• Status: Terminated
• Phase: Phase 1
• First received: May 5, 2011
• Last updated: November 21, 2011
• Start date: May 2011
• Est. completion date: November 2011

Study information

• Verified date: November 2011
• Source: Santaris Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial Summary

The purpose is to study Safety and Tolerability.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 24
• Est. completion date: November 2011
• Est. primary completion date: October 2011
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

1. Healthy male or female subjects and subjects with heterozygous Familial Hypercholesterolemia

• Healthy male or female subjects, age 18 to 65 years, inclusive will be enrolled in Cohorts 1 through 4.

• In Cohort 5, male or female subjects with heterozygous Familial Hypercholesterolemia, confirmed through genetic testing, without a history of cardiovascular disease (e.g. coronary artery, peripheral artery or cerebrovascular disease), hypertension or diabetes mellitus age 18-45 years, inclusive, will be enrolled.

2. BMI of 18-33 kg/m2

3. Screening hematology, clinical chemistries, coagulation and urinalysis consistent with overall good health and the following criteria are met:

• LDL =.3.24 mmol/L (= 100 mg/dL)

• Triglycerides (fasted) < 4.5 mmol/L (< 398 mg/dL)

• ALT within normal limits for healthy subjects and ALT < 2 x ULN for FH subjects

Exclusion Criteria:

1. Any uncontrolled or active major systemic disease including, but not limited to:

cardiovascular, pulmonary, gastrointestinal, metabolic, urogenital, neurological, immunological, psychiatric, or neoplastic disorder with metastatic potential

• History or presence of malignancy within the past year is an exclusion criterion. Subjects who have been successfully treated with no recurrence of basal cell carcinoma of the skin or carcinoma in-situ of the cervix may be enrolled.

2. Active acute or chronic infection, including, but not limited to: upper airway infection, urinary tract infection, and skin infection

3. Use of prescription medication within 14 days prior to the planned first drug administration and throughout the study. For the FH subjects statin therapy (and other lipid lowering therapies) will be prohibited within 4 weeks prior to the first study drug administration.

4. Use of non-prescription or over-the-counter medications is prohibited within 7 days prior to the planned first drug administration and throughout the study. This includes all vitamins, herbal supplements, or remedies. An exception can be made for medication or supplements that in the opinion of both the investigator and the Sponsor do not complicate or compromise the study or interfere with the study objectives.

5. Positive results on the following Screening laboratory tests: urine or serum pregnancy test (women only), alcohol breath test, urine drugs of abuse, hepatitis B surface antigen, hepatitis C antibody, and human immunodeficiency virus (HIV) antibody.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hypercholesterolemia

Intervention

Drug:
• SPC5001
3 weekly SC injections
• Saline 0.9%
3 weekly SC injections
• SPC5001
3 weekly SC injections
• SPC5001
3 weekly SC injections
• SPC5001
3 weekly SC injections
• SPC5001
3 weekly SC injections

Locations

Country	Name	City	State
Netherlands	Centre for Huma Drug Research (CHDR)	Leiden

Sponsors (1)

Lead Sponsor	Collaborator
Santaris Pharma A/S

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability	Safety evaluation will assess adverse event (AE) profile, clinical laboratory safety tests, vital signs and ECG monitoring	Regularly over 78 days	Yes
Secondary	Peak Plasma Concentration (Cmax) of SPC5001		up to 78 days	No
Secondary	Lipid lowering effect		Through out the study	No
Secondary	Area under the plasma concentration versus time curve (AUC) of SPC5001		up to 78 days	No