Ignite Creation Date:
2025-12-25 @ 2:07 AM
Ignite Modification Date:
2026-03-03 @ 6:05 PM
Study NCT ID:
NCT01350960
Status:
TERMINATED
Last Update Posted:
2011-11-22
First Post:
2011-05-05
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Multiple Ascending Dose Study of SPC5001 in Treatment of Healthy Subjects and Subjects With FH
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006937', 'term': 'Hypercholesterolemia'}, {'id': 'D006949', 'term': 'Hyperlipidemias'}], 'ancestors': [{'id': 'D050171', 'term': 'Dyslipidemias'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000720888', 'term': 'SPC5001'}, {'id': 'D012965', 'term': 'Sodium Chloride'}], 'ancestors': [{'id': 'D002712', 'term': 'Chlorides'}, {'id': 'D006851', 'term': 'Hydrochloric Acid'}, {'id': 'D017606', 'term': 'Chlorine Compounds'}, {'id': 'D007287', 'term': 'Inorganic Chemicals'}, {'id': 'D017670', 'term': 'Sodium Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 24}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2011-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-11', 'completionDateStruct': {'date': '2011-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2011-11-21', 'studyFirstSubmitDate': '2011-05-05', 'studyFirstSubmitQcDate': '2011-05-09', 'lastUpdatePostDateStruct': {'date': '2011-11-22', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-05-10', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and Tolerability', 'timeFrame': 'Regularly over 78 days', 'description': 'Safety evaluation will assess adverse event (AE) profile, clinical laboratory safety tests, vital signs and ECG monitoring'}], 'secondaryOutcomes': [{'measure': 'Peak Plasma Concentration (Cmax) of SPC5001', 'timeFrame': 'up to 78 days'}, {'measure': 'Lipid lowering effect', 'timeFrame': 'Through out the study'}, {'measure': 'Area under the plasma concentration versus time curve (AUC) of SPC5001', 'timeFrame': 'up to 78 days'}]}, 'conditionsModule': {'keywords': ['hypercholesterolemia', 'hyperlipidemia', 'LDL', 'HDL', 'PCSK9', 'LNA-oligonucleotide'], 'conditions': ['Hypercholesterolemia']}, 'referencesModule': {'references': [{'pmid': '26261033', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': 'The purpose is to study Safety and Tolerability.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Healthy male or female subjects and subjects with heterozygous Familial Hypercholesterolemia\n\n * Healthy male or female subjects, age 18 to 65 years, inclusive will be enrolled in Cohorts 1 through 4.\n * 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.\n2. BMI of 18-33 kg/m2\n3. Screening hematology, clinical chemistries, coagulation and urinalysis consistent with overall good health and the following criteria are met:\n\n * LDL ≥.3.24 mmol/L (≥ 100 mg/dL)\n * Triglycerides (fasted) \\< 4.5 mmol/L (\\< 398 mg/dL)\n * ALT within normal limits for healthy subjects and ALT \\< 2 x ULN for FH subjects\n\nExclusion Criteria:\n\n1. 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\n\n \\- 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.\n2. Active acute or chronic infection, including, but not limited to: upper airway infection, urinary tract infection, and skin infection\n3. 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.\n4. 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.\n5. 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.'}, 'identificationModule': {'nctId': 'NCT01350960', 'briefTitle': 'Multiple Ascending Dose Study of SPC5001 in Treatment of Healthy Subjects and Subjects With FH', 'organization': {'class': 'INDUSTRY', 'fullName': 'Santaris Pharma A/S'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': 'SPC5001-901'}, 'secondaryIdInfos': [{'id': 'EudraCT 2011-000489-36'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'saline 0.9%', 'interventionNames': ['Drug: Saline 0.9%']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': '0.5 mg/kg in Healthy Subjects', 'interventionNames': ['Drug: SPC5001']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': '1.5 mg/kg in Healthy subjects', 'interventionNames': ['Drug: SPC5001']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': '5.0 mg/kg in Healthy subjects', 'interventionNames': ['Drug: SPC5001']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4', 'description': '10 mg/kg in Healthy subjects', 'interventionNames': ['Drug: SPC5001']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 5', 'description': 'TBD mg/kg in FH subjects', 'interventionNames': ['Drug: SPC5001']}], 'interventions': [{'name': 'SPC5001', 'type': 'DRUG', 'description': '3 weekly SC injections', 'armGroupLabels': ['Cohort 1']}, {'name': 'Saline 0.9%', 'type': 'DRUG', 'description': '3 weekly SC injections', 'armGroupLabels': ['saline 0.9%']}, {'name': 'SPC5001', 'type': 'DRUG', 'description': '3 weekly SC injections', 'armGroupLabels': ['Cohort 2']}, {'name': 'SPC5001', 'type': 'DRUG', 'description': '3 weekly SC injections', 'armGroupLabels': ['Cohort 3']}, {'name': 'SPC5001', 'type': 'DRUG', 'description': '3 weekly SC injections', 'armGroupLabels': ['Cohort 4']}, {'name': 'SPC5001', 'type': 'DRUG', 'description': '3 weekly SC injections', 'armGroupLabels': ['Cohort 5']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2333', 'city': 'Leiden', 'country': 'Netherlands', 'facility': 'Centre for Huma Drug Research (CHDR)', 'geoPoint': {'lat': 52.15833, 'lon': 4.49306}}], 'overallOfficials': [{'name': 'Koos Burggraaf, MD PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Centre for Human Drug Research (CHDR)'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Santaris Pharma A/S', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}