Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

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

This study has been terminated.
Information provided by (Responsible Party):
Santaris Pharma A/S Identifier:
First received: May 5, 2011
Last updated: November 21, 2011
Last verified: November 2011
The purpose is to study Safety and Tolerability.

Condition Intervention Phase
Drug: SPC5001
Drug: Saline 0.9%
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (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)

Resource links provided by NLM:

Further study details as provided by Santaris Pharma A/S:

Primary Outcome Measures:
  • Safety and Tolerability [ Time Frame: 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 [ Time Frame: up to 78 days ]
  • Lipid lowering effect [ Time Frame: Through out the study ]
  • Area under the plasma concentration versus time curve (AUC) of SPC5001 [ Time Frame: up to 78 days ]

Enrollment: 24
Study Start Date: May 2011
Study Completion Date: November 2011
Primary Completion Date: October 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01350960

Centre for Huma Drug Research (CHDR)
Leiden, Netherlands, 2333
Sponsors and Collaborators
Santaris Pharma A/S
Principal Investigator: Koos Burggraaf, MD PhD Centre for Human Drug Research (CHDR)
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Santaris Pharma A/S Identifier: NCT01350960     History of Changes
Other Study ID Numbers: SPC5001-901
EudraCT 2011-000489-36
Study First Received: May 5, 2011
Last Updated: November 21, 2011

Keywords provided by Santaris Pharma A/S:

Additional relevant MeSH terms:
Lipid Metabolism Disorders
Metabolic Diseases processed this record on April 25, 2017