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

Sponsor

Santaris Pharma A/S (Industry)

Overall Status

Terminated

CT.gov ID

NCT01350960

Collaborator

(none)

Enrollment

Location

Arms

Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose is to study Safety and Tolerability.

Condition or Disease	Intervention/Treatment	Phase
Hypercholesterolemia	Drug: SPC5001 Drug: Saline 0.9% Drug: SPC5001 Drug: SPC5001 Drug: SPC5001 Drug: SPC5001	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

24 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

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)

Study Start Date :

May 1, 2011

Actual Primary Completion Date :

Oct 1, 2011

Actual Study Completion Date :

Nov 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: saline 0.9%	Drug: Saline 0.9% 3 weekly SC injections
Experimental: Cohort 1 0.5 mg/kg in Healthy Subjects	Drug: SPC5001 3 weekly SC injections
Experimental: Cohort 2 1.5 mg/kg in Healthy subjects	Drug: SPC5001 3 weekly SC injections
Experimental: Cohort 3 5.0 mg/kg in Healthy subjects	Drug: SPC5001 3 weekly SC injections
Experimental: Cohort 4 10 mg/kg in Healthy subjects	Drug: SPC5001 3 weekly SC injections
Experimental: Cohort 5 TBD mg/kg in FH subjects	Drug: SPC5001 3 weekly SC injections

Outcome Measures

Primary Outcome Measures

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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

Inclusion Criteria:

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.

BMI of 18-33 kg/m2
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:

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.

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