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

Santaris Pharma

Status and phase: Terminated; Phase 1

Conditions: Hypercholesterolemia

Treatments: Drug: Saline 0.9%; Drug: SPC5001

Study type: Interventional

Funder types: Industry

Identifiers

NCT01350960

SPC5001-901

EudraCT 2011-000489-36

Details and patient eligibility

About

The purpose is to study Safety and Tolerability.

Enrollment: 24 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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24 participants in 6 patient groups, including a placebo group

saline 0.9%

Placebo Comparator group

Treatment:

Drug: Saline 0.9%

Cohort 1

Experimental group

Description:

0.5 mg/kg in Healthy Subjects

Treatment:

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Cohort 2

Experimental group

Description:

1.5 mg/kg in Healthy subjects

Treatment:

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Cohort 3

Experimental group

Description:

5.0 mg/kg in Healthy subjects

Treatment:

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Cohort 4

Experimental group

Description:

10 mg/kg in Healthy subjects

Treatment:

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Cohort 5

Experimental group

Description:

TBD mg/kg in FH subjects

Treatment:

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001

Drug: SPC5001