HypercholesterolemiaPhase 1NCT01350960
Multiple Ascending Dose Study of SPC5001 in Treatment of Healthy Subjects and Subjects With FH
A Phase 1 study testing SPC5001 and Saline 0.9% in people with Hypercholesterolemia, run by Santaris Pharma A/S at 1 site in Netherlands.
Trial summary
The purpose is to study Safety and Tolerability. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypercholesterolemia
- What is being tested?
- SPC5001 and Saline 0.9%
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteNetherlands
- Study dates
- May 2011to Nov 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
- BMI of 18-33 kg/m2
- 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
Who cannot join
- 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.
- Active acute or chronic infection, including, but not limited to: upper airway infection, urinary tract infection, and skin infection
- 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.
- 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.
- 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 locations and contacts
1 site in 1 country.
About this study
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)
The purpose is to study Safety and Tolerability.
- Conditions
- Hypercholesterolemia
- Being tested
- SPC5001, Saline 0.9%
- Keywords
- hypercholesterolemia, hyperlipidemia, LDL, HDL, PCSK9, LNA-oligonucleotide
- Medical subject headings
- Hypercholesterolemia, Hyperlipidemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPC5001Drug
3 weekly SC injections
Saline 0.9%Drug
3 weekly SC injections
SPC5001Drug
3 weekly SC injections
SPC5001Drug
3 weekly SC injections
SPC5001Drug
3 weekly SC injections
SPC5001Drug
3 weekly SC injections
| Study group | Role | What they receive |
|---|---|---|
| saline 0.9% | Placebo comparator | Not described Receives: Drug: Saline 0.9% |
| Cohort 1 | Experimental | 0.5 mg/kg in Healthy Subjects Receives: Drug: SPC5001 |
| Cohort 2 | Experimental | 1.5 mg/kg in Healthy subjects Receives: Drug: SPC5001 |
| Cohort 3 | Experimental | 5.0 mg/kg in Healthy subjects Receives: Drug: SPC5001 |
| Cohort 4 | Experimental | 10 mg/kg in Healthy subjects Receives: Drug: SPC5001 |
| Cohort 5 | Experimental | TBD mg/kg in FH subjects Receives: Drug: SPC5001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Safety evaluation will assess adverse event (AE) profile, clinical laboratory safety tests, vital signs and ECG monitoring
Measured: Regularly over 78 days
Secondary outcomes (3)
Peak Plasma Concentration (Cmax) of SPC5001
Measured: up to 78 days
Lipid lowering effect
Measured: Through out the study
Area under the plasma concentration versus time curve (AUC) of SPC5001
Measured: up to 78 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2011 (estimated)
- Study startMay 2011
- Primary completionOct 2011
- Study completionNov 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santaris Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Koos Burggraaf, MD PhD, Principal investigator, Centre for Human Drug Research (CHDR)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01350960
- Sponsor's protocol ID
- SPC5001-901
- Other IDs
- EudraCT 2011-000489-36
- Status last verified
- Nov 2011
- First submitted
- 5 May 2011
- Last updated
- 22 Nov 2011
- Results posted
- No
Publications
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.
PubMed 26261033
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT01350960
- Acronym / sponsor ID
- SPC5001-901
- Conditions
- Hypercholesterolemia
- Keywords
- hypercholesterolemia, hyperlipidemia, LDL, HDL, PCSK9, LNA-oligonucleotide
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Santaris Pharma A/SIndustry (pharma, biotech, devices)
- Study start
- May 2011
- Primary completion
- Oct 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 10 May 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose is to study Safety and Tolerability.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| saline 0.9% | Placebo comparator | Drug: Saline 0.9% |
| Cohort 1 | Experimental | Drug: SPC50010.5 mg/kg in Healthy Subjects |
| Cohort 2 | Experimental | Drug: SPC50011.5 mg/kg in Healthy subjects |
| Cohort 3 | Experimental | Drug: SPC50015.0 mg/kg in Healthy subjects |
| Cohort 4 | Experimental | Drug: SPC500110 mg/kg in Healthy subjects |
| Cohort 5 | Experimental | Drug: SPC5001TBD mg/kg in FH subjects |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Healthy male or female subjects and subjects with heterozygous Familial Hypercholesterolemia
- BMI of 18-33 kg/m2
- Screening hematology, clinical chemistries, coagulation and urinalysis consistent with overall good health and the following criteria are met:
Key exclusion criteria (6 listed)
- 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
- Active acute or chronic infection, including, but not limited to: upper airway infection, urinary tract infection, and skin infection
- 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.
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPC50013 weekly SC injections | Drug | Cohort 1 |
| Saline 0.9%3 weekly SC injections | Drug | saline 0.9% |
| SPC50013 weekly SC injections | Drug | Cohort 2 |
| SPC50013 weekly SC injections | Drug | Cohort 3 |
| SPC50013 weekly SC injections | Drug | Cohort 4 |
| SPC50013 weekly SC injections | Drug | Cohort 5 |
Primary outcome
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
3 secondary outcomes
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
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2011.
- Derived from this study: PubMed 26261033van 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Koos Burggraaf, MD PhD principal investigator
Centre for Human Drug Research (CHDR)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SPC5001 and Saline 0.9% in people with Hypercholesterolemia, run by Santaris Pharma A/S at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Nov 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (time frame: Regularly over 78 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01350960, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2011.