Lipoprotein Lipase Deficiency, FamilialPhase 2NCT02098278
Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia
A Phase 2 study testing CAT-2003 in people with Lipoprotein Lipase Deficiency, Familial and Hyperlipoproteinemia Type I, run by Catabasis Pharmaceuticals at 2 sites in Canada.
Trial summary
The purpose of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as postprandial total and chylomicron triglyceride clearance. This is a single-blind study. Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I, Chylomicronemia, Familial
- What is being tested?
- CAT-2003
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 13 participants
- Where
- 2 sitesCanada
- Study dates
- Mar 2014to Apr 2015
- 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 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Familial Chylomicronemia (Fasting triglycerides ≥ 880 mg/dL at Screening and documented history of plasma post-heparin LDL activity < 20% of normal or genetic confirmation of homozygosity or compound heterozygosity for loss-of-function mutations in familial chylomicronemia-causing genes) OR
- Non-familial Chylomicronemia (Fasting triglycerides ≥ 440 mg/dL at Screening and documented history of fasting triglycerides ≥ 880 mg/dL)
Who cannot join
- History of any major cardiovascular event within 6 months of Screening
- Type I diabetes mellitus or use of insulin
- History of pancreatitis within 3 month of Screening
Trial locations and contacts
2 sites in 1 country.
Canada2 sites
Study site
Chicoutimi, Quebec, G7H 7K9
Study site
Québec, Quebec, G1V 4M6
About this study
Official title: A Phase 2 Pilot Study to Assess the Safety and Efficacy of CAT-2003 in Patients With Chylomicronemia
The purpose of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as postprandial total and chylomicron triglyceride clearance.
This is a single-blind study. All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.
- Conditions
- Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I, Chylomicronemia, Familial, Hypertriglyceridemia
- Being tested
- CAT-2003, Placebo
- Medical subject headings
- Hyperlipoproteinemia Type I, Hypertriglyceridemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAT-2003Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| CAT-2003 or Placebo | Experimental | All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period. Receives: Drug: CAT-2003; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in fasting triglycerides in patients with chylomicronemia
Measured: 12 Weeks
Secondary outcomes (4)
Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels
Measured: 12 Weeks
Absolute and percent change from baseline on chylomicron triglyceride clearance
Measured: 12 Weeks
Absolute and percent change from baseline in plasma non-HDL-C
Measured: 12 Weeks
Frequency of adverse events
Measured: 13 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2014 (estimated)
- Study startMar 2014
- Primary completionApr 2015
- Study completionApr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lipoprotein Lipase Deficiency, FamilialStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catabasis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02098278
- Sponsor's protocol ID
- CAT-2003-203
- Status last verified
- Jul 2016
- First submitted
- 24 Mar 2014
- Last updated
- 21 Jul 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Pilot Study to Assess the Safety and Efficacy of CAT-2003 in Patients With Chylomicronemia
- ClinicalTrials.gov ID
- NCT02098278
- Acronym / sponsor ID
- CAT-2003-203
- Conditions
- Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I, Chylomicronemia, Familial, Hypertriglyceridemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Catabasis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Mar 2014
- Primary completion
- Apr 2015 (actual)
- Study completion
- Apr 2015 (actual)
- First posted
- 28 Mar 2014 (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 of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as postprandial total and chylomicron triglyceride clearance.
This is a single-blind study. All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- Single blind (participant)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAT-2003 or Placebo | Experimental | Drug: CAT-2003, Drug: PlaceboAll patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period. |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Familial Chylomicronemia (Fasting triglycerides ≥ 880 mg/dL at Screening and documented history of plasma post-heparin LDL activity < 20% of normal or genetic confirmation of homozygosity or compound heterozygosity for loss-of-function mutations in familial chylomicronemia-causing genes) OR
- Non-familial Chylomicronemia (Fasting triglycerides ≥ 440 mg/dL at Screening and documented history of fasting triglycerides ≥ 880 mg/dL)
Key exclusion criteria (3 listed)
- History of any major cardiovascular event within 6 months of Screening
- Type I diabetes mellitus or use of insulin
- History of pancreatitis within 3 month of Screening
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 |
|---|---|---|
| CAT-2003 | Drug | CAT-2003 or Placebo |
| Placebo | Drug | CAT-2003 or Placebo |
Primary outcome
Percent change from baseline in fasting triglycerides in patients with chylomicronemia
Time frame: 12 Weeks
4 secondary outcomes
Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels
Time frame: 12 Weeks
Absolute and percent change from baseline on chylomicron triglyceride clearance
Time frame: 12 Weeks
Absolute and percent change from baseline in plasma non-HDL-C
Time frame: 12 Weeks
Frequency of adverse events
Time frame: 13 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2016.
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CAT-2003 in people with Lipoprotein Lipase Deficiency, Familial and Hyperlipoproteinemia Type I, run by Catabasis Pharmaceuticals at 2 sites 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 2 inclusion and 3 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: finished, as of the sponsor's last update on 21 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Percent change from baseline in fasting triglycerides in patients with chylomicronemia (time frame: 12 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 21 Jul 2016.