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

This study has been terminated.
Sponsor:
Information provided by (Responsible Party):
Santaris Pharma A/S
ClinicalTrials.gov Identifier:
NCT01350960
First received: May 5, 2011
Last updated: November 21, 2011
Last verified: November 2011
  Purpose

The purpose is to study Safety and Tolerability.


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

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
  • Lipid lowering effect [ Time Frame: Through out the study ] [ Designated as safety issue: No ]
  • Area under the plasma concentration versus time curve (AUC) of SPC5001 [ Time Frame: up to 78 days ] [ Designated as safety issue: No ]

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

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

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 ClinicalTrials.gov identifier: NCT01350960

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

No publications provided

Responsible Party: Santaris Pharma A/S
ClinicalTrials.gov 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
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by Santaris Pharma A/S:
hypercholesterolemia
hyperlipidemia
LDL
HDL
PCSK9
LNA-oligonucleotide

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on September 30, 2014