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

Phase 1

Terminated

• Conditions: Hypercholesterolemia
• Interventions: Drug: SPC5001; Drug: Saline 0.9%

• Registration Number: NCT01350960
• Lead Sponsor: Santaris Pharma A/S

Brief Summary

The purpose is to study Safety and Tolerability.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-05
• Study First Submitted: 2011-05-05
• Study First Submitted QC: 2011-05-09
• Study First Posted: 2011-05-10
• Primary Completion: 2011-10
• Status Verified: 2011-11
• Study Completion: 2011-11
• Last Update Submitted: 2011-11-21
• Last Update Posted: 2011-11-22

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 24

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

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 4	SPC5001	10 mg/kg in Healthy subjects
Cohort 5	SPC5001	TBD mg/kg in FH subjects
saline 0.9%	Saline 0.9%	-
Cohort 1	SPC5001	0.5 mg/kg in Healthy Subjects
Cohort 2	SPC5001	1.5 mg/kg in Healthy subjects
Cohort 3	SPC5001	5.0 mg/kg in Healthy subjects

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	Regularly over 78 days	Safety evaluation will assess adverse event (AE) profile, clinical laboratory safety tests, vital signs and ECG monitoring

Secondary Outcome Measures

Name	Time	Method
Lipid lowering effect	Through out the study
Area under the plasma concentration versus time curve (AUC) of SPC5001	up to 78 days
Peak Plasma Concentration (Cmax) of SPC5001	up to 78 days

Trial Locations

Locations (1)

Centre for Huma Drug Research (CHDR); 🇳🇱 Leiden, Netherlands