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

NCT ID: NCT01350960

Last Updated: 2011-11-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-05-31

Study Completion Date

2011-11-30

Brief Summary

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The purpose is to study Safety and Tolerability.

Detailed Description

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Conditions

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Hypercholesterolemia

Keywords

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hypercholesterolemia hyperlipidemia LDL HDL PCSK9 LNA-oligonucleotide

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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saline 0.9%

Group Type PLACEBO_COMPARATOR

Saline 0.9%

Intervention Type DRUG

3 weekly SC injections

Cohort 1

0.5 mg/kg in Healthy Subjects

Group Type EXPERIMENTAL

SPC5001

Intervention Type DRUG

3 weekly SC injections

Cohort 2

1.5 mg/kg in Healthy subjects

Group Type EXPERIMENTAL

SPC5001

Intervention Type DRUG

3 weekly SC injections

Cohort 3

5.0 mg/kg in Healthy subjects

Group Type EXPERIMENTAL

SPC5001

Intervention Type DRUG

3 weekly SC injections

Cohort 4

10 mg/kg in Healthy subjects

Group Type EXPERIMENTAL

SPC5001

Intervention Type DRUG

3 weekly SC injections

Cohort 5

TBD mg/kg in FH subjects

Group Type EXPERIMENTAL

SPC5001

Intervention Type DRUG

3 weekly SC injections

Interventions

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SPC5001

3 weekly SC injections

Intervention Type DRUG

Saline 0.9%

3 weekly SC injections

Intervention Type DRUG

SPC5001

3 weekly SC injections

Intervention Type DRUG

SPC5001

3 weekly SC injections

Intervention Type DRUG

SPC5001

3 weekly SC injections

Intervention Type DRUG

SPC5001

3 weekly SC injections

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Santaris Pharma A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Koos Burggraaf, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Centre for Human Drug Research (CHDR)

Locations

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Centre for Huma Drug Research (CHDR)

Leiden, , Netherlands

Site Status

Countries

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Netherlands

References

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van Poelgeest EP, Hodges MR, Moerland M, Tessier Y, Levin AA, Persson R, Lindholm MW, Dumong Erichsen K, Orum H, Cohen AF, Burggraaf J. Antisense-mediated reduction of proprotein convertase subtilisin/kexin type 9 (PCSK9): a first-in-human randomized, placebo-controlled trial. Br J Clin Pharmacol. 2015 Dec;80(6):1350-61. doi: 10.1111/bcp.12738. Epub 2015 Oct 24.

Reference Type DERIVED
PMID: 26261033 (View on PubMed)

Other Identifiers

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EudraCT 2011-000489-36

Identifier Type: -

Identifier Source: secondary_id

SPC5001-901

Identifier Type: -

Identifier Source: org_study_id