Homozygous Familial HypercholesterolemiaPhase 2NCT03455777
Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
A Phase 2 study testing AKCEA-ANGPTL3-LRX in people with Homozygous Familial Hypercholesterolemia, run by Akcea Therapeutics at 1 site in Canada.
Trial summary
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- AKCEA-ANGPTL3-LRX
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteCanada
- Study dates
- 12 Apr 2018to 31 Dec 2018
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Body mass index (BMI) ≤ 35 kg/m2,
- Genetic confirmation of two mutant alleles at the LDLR, APOB, PCSK9, or LDLRAP1 gene locus OR an untreated LDL-C > 500 mg/dL (13 mmol/L) or treated LDL-C ≥ 300 mg/dL (2.59 mmol/L) together with either cutaneous or tendon xanthoma before age 10 years OR familial medical history of genetically confirmed heterozygous FH in both parents OR untreated elevated LDL-C and TC > 250 mg/dL consistent with the disease,
- Patients must be on stable LDL-C lowering agents or on regular apheresis
Who cannot join
- Myocardial infarction, percutaneous transluminal coronary intervention, or coronary artery bypass graft surgery within 12 weeks prior to Screening, or cerebrovascular accident within 24 weeks prior to Screening.
- Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH).
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- AKCEA-ANGPTL3-LRX
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AKCEA-ANGPTL3-LRXDrug
Also called ISIS 703802
Single open-label cohort
| Study group | Role | What they receive |
|---|---|---|
| AKCEA-ANGPTL3-LRX Dose 1 | Experimental | Not described Receives: Drug: AKCEA-ANGPTL3-LRX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of low density lipoprotein cholesterol (LDL-C).
Percent change in LDL-C from Baseline to week 14.
Measured: 14 weeks
Secondary outcomes (4)
Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).
Absolute and percentage change in ANGPTL3 protein from Baseline to Week 14 will be summarized.
Measured: 7 and 14 Weeks
Effect of ISIS 703802 on lipid parameters.
Absolute and percentage change from Baseline to Week 14 will be summarized.
Measured: 7 and 14 Weeks
Evaluate plasma trough levels of ISIS 703802.
Plasma trough levels of ISIS 703802 during treatment period and those during post-treatment follow up period will be descriptively summarized.
Measured: 14 Weeks
The safety of ISIS 703802 by the incidence of treatment-emergent adverse events
The safety of ISIS 703802 will be assessed by determining adverse effects.
Measured: 14 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2018
- Study start12 Apr 2018
- Primary completion30 Sep 2018 (estimated)
- Study completion31 Dec 2018 (estimated)
Why it stopped: Study withdrawn due to lack of available patients meeting entry criteria
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous Familial 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Akcea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03455777
- Sponsor's protocol ID
- ISIS 703802-CS4
- Status last verified
- Nov 2018
- First submitted
- 22 Feb 2018
- Last updated
- 3 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)
- ClinicalTrials.gov ID
- NCT03455777
- Acronym / sponsor ID
- ISIS 703802-CS4
- Conditions
- Homozygous Familial Hypercholesterolemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study withdrawn due to lack of available patients meeting entry criteria
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Akcea TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Study start
- 12 Apr 2018 (actual)
- Primary completion
- 30 Sep 2018 (estimated)
- Study completion
- 31 Dec 2018 (estimated)
- First posted
- 7 Mar 2018 (actual)
- 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.
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AKCEA-ANGPTL3-LRX Dose 1 | Experimental | Drug: AKCEA-ANGPTL3-LRX |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Body mass index (BMI) ≤ 35 kg/m2,
- Genetic confirmation of two mutant alleles at the LDLR, APOB, PCSK9, or LDLRAP1 gene locus OR an untreated LDL-C > 500 mg/dL (13 mmol/L) or treated LDL-C ≥ 300 mg/dL (2.59 mmol/L) together with either cutaneous or tendon xanthoma before age 10 years OR familial medical history of genetically confirmed heterozygous FH in both parents OR untreated elevated LDL-C and TC > 250 mg/dL consistent with the disease,
- Patients must be on stable LDL-C lowering agents or on regular apheresis
Key exclusion criteria (2 listed)
- Myocardial infarction, percutaneous transluminal coronary intervention, or coronary artery bypass graft surgery within 12 weeks prior to Screening, or cerebrovascular accident within 24 weeks prior to Screening.
- Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
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 |
|---|---|---|
| AKCEA-ANGPTL3-LRXAlso: ISIS 703802Single open-label cohort | Drug | AKCEA-ANGPTL3-LRX Dose 1 |
Primary outcome
Reduction of low density lipoprotein cholesterol (LDL-C).
Time frame: 14 weeks
Percent change in LDL-C from Baseline to week 14.
4 secondary outcomes
Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).
Time frame: 7 and 14 Weeks
Effect of ISIS 703802 on lipid parameters.
Time frame: 7 and 14 Weeks
Evaluate plasma trough levels of ISIS 703802.
Time frame: 14 Weeks
The safety of ISIS 703802 by the incidence of treatment-emergent adverse events
Time frame: 14 Weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2018.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AKCEA-ANGPTL3-LRX in people with Homozygous Familial Hypercholesterolemia, run by Akcea Therapeutics at 1 site in Canada.
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 3 inclusion and 2 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: withdrawn, as of the sponsor's last update on 3 Dec 2018. 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 Canada. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 withdrawn before enrolling participants, so there are no results.
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?
Reduction of low density lipoprotein cholesterol (LDL-C). (time frame: 14 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Dec 2018.