Sickle Cell DiseasePhase 3ASCENTNCT02604368
A Sickle CEll Disease ComplicatioN Trial
A Phase 3 study testing SC411 in people with Sickle Cell Disease, run by Micelle BioPharma Inc.
Trial summary
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- SC411
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 210 participants
- Where
- No sites listed yet
- Study dates
- Mar 2019to Dec 2020
- Phase
- Phase 3
- 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 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients who meet all of the following criteria will be eligible to participate in the study:
- 1. Aged ≥ 5 years and ≤ 17 years at screening;
- Has been diagnosed with SCD (that includes the genotypes HbSS, HbSC, and HbS/β°-thalassemia, documented by hemoglobin HPLC or electrophoresis);
- Has had between ≥ 2 to ≤10 episodes of acute SCC (as defined above) within 12 months of the Screening Visit. At least one crisis must have been managed in a hospital, clinic, or emergency room. For at least 2 of the episodes, the site must obtain the documentation created in a medical record at the time of the event.
- Must not be receiving HU or L-Glutamine, or if receiving HU and/or L-Glutamine must be at a stable weight-based treatment regimen (mg/kg), for at least 3 months prior to the Screening Visit with the intent to continue at a weight-based dose level at the discretion of the treating physician for the duration of the study, other than for safety reasons. If taking HU and/or L-glutamine, subjects must have had at least one SCC event (as previously defined) while on HU and/or L-Glutamine.
- Has a parent or guardian who is able to give written informed consent, and the potential pediatric subject must be able to provide assent in a manner approved by the Institutional Review Board (IRB) and comply with the requirements of the study; and
- If post pubertal in the opinion of the Investigator, must agree to use a reliable method of birth control (e.g., barrier, birth control pills, abstinence) during the study and for 1 month following the last dose of study drug.
Who cannot join
- Patients who meet any of the following criteria will be excluded from participation in the study:
- A known allergy or hypersensitivity to fish or shellfish;
- A known allergy or hypersensitivity to soy;
- Inability to swallow capsules;
- History of treatment with SC411;
- Confirmed diagnosis of chronic pain or chronic opioid use: Defined as pain experienced ≥3 days per week over a 6-month period or daily opioid use for pain management;
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- Active infection with Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C;
- Prothrombin time > 1.5 x ULN at screening;
- Required regular anticoagulation or chronic aspirin therapy;
- Moderate thrombocytopenia, defined as platelets < 80,000/µL at screening;
- History of stroke or Moyamoya syndrome;
- Abnormal results on most recent transcranial Doppler (TCD) evaluation;
- Received blood transfusion or blood products in the 2 months prior to the Screening Visit or on chronic blood transfusion;
- Chronic renal insufficiency, defined as a eGFR < 30ml/min at screening as estimated by the Schwartz equation (Appendix H), or requiring peritoneal or hemodialysis;
- Abnormal liver function tests (ALT > 3.0 x ULN) at screening;
- Received any organ transplant;
- Has a recent acute illness or other concomitant chronic medical or psychiatric condition that in the opinion of the Investigator would compromise participation in the study, prevent adherence to the protocol or confound the evaluation of the study outcome;
- Is pregnant or lactating, or has the intention of becoming pregnant during the study (if a female of child-bearing potential or partner of a female of child-bearing potential and sexually active and not willing to use an effective means of birth control);
- Is currently taking or has been treated with any form of omega-3 fatty acid or fish oil supplement within 30 days of the Screening Visit, or intends to do so during the course of the study;
- Has been treated with any investigational product within 30 days of the Screening Visit or intends to receive an investigational product during the course of this study; and
- There are factors that, in the judgment of the Investigator, would make it difficult for the patient to comply with the requirements of the study
Trial locations and contacts
No sites are listed yet.
About this study
Official title: A Phase 3, Prospective, Randomized, Double-Blind, Placebo Controlled, Multi-center Study of SC411 for Sickle Cell Disease
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
Read the detailed description
This Phase 3, prospective, randomized, double-blinded, placebo-controlled, multi-center study will enroll approximately 210 subjects at up to 70 sites in the United States. Participation will consist of a Screening Period, followed by a minimum 12-month Treatment Period. SC411 is administered orally as a soft gel mini capsule.
This study will enroll subjects aged ≥5 to ≤17 years who have a diagnosis of SCD that includes the phenotypes hemoglobin SS homozygous (HbSS), hemoglobin SC (HbSC), and hemoglobin S/β°-thalassemia (HbS/ β°-thalassemia); and have had at least 2 but no more than 10 documented SCC events (as defined above) within 12 months prior to the Screening Visit.
- Conditions
- Sickle Cell Disease
- Being tested
- SC411, Placebo
- Keywords
- hemoglobin SS, hemoglobin SC, hemoglobin S/β°-thalassemia
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobin SC Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SC411Drug
Also called Docosahexaenoic acid (DHA)
Soft gelatin capsule
PlaceboDrug
Also called Soybean Oil
Soft gelatin capsule
| Study group | Role | What they receive |
|---|---|---|
| SC411 | Experimental | Omega-3 docosahexaenoic acid, soft gelatin capsule, administered once a day on a per weight basis. Receives: Drug: SC411 |
| Placebo | Placebo comparator | Soybean oil, soft gelatin capsule, administered once a day on a per weight basis. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization.
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization.
The primary objective of this study is to assess the efficacy of orally administered SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects compared to placebo. A SCC event will be defined as either an acute painful crisis or an acute chest syndrome.
Measured: 52 weeks
Secondary outcomes (4)
Evaluation of the effect of SC411 compared to placebo by measuring the time until the patient's first sickle cell event.
Evaluation of days to the first event from randomization for each patient.
Measured: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD.
Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD.
Measured: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain.
To evaluate the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain.
Measured: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the number of crisis-free days.
To evaluate the effect of SC411 compared to placebo by measuring the number of crisis-free days. A crisis-free day is defined as any day with a zero entry on the eDiary pain intensity scale.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2015 (estimated)
- Study startMar 2019 (estimated)
- Primary completionDec 2020 (estimated)
- Study completionDec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 210 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Micelle BioPharma Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carton Dampier, MD, Study chair, Emory UniversityMatt Heeney, MD, Study chair, Harvard UniversityBeng Fuh, MD, Study chair, East Carolina University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02604368
- Sponsor's protocol ID
- OMEG-411-01
- Acronym
- ASCENT
- Status last verified
- Jan 2019
- First submitted
- 11 Nov 2015
- Last updated
- 1 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Prospective, Randomized, Double-Blind, Placebo Controlled, Multi-center Study of SC411 for Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02604368
- Acronym / sponsor ID
- ASCENT / OMEG-411-01
- Conditions
- Sickle Cell Disease
- Keywords
- hemoglobin SS, hemoglobin SC, hemoglobin S/β°-thalassemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 210 participants (estimated)
- Lead sponsor
- Micelle BioPharma IncIndustry (pharma, biotech, devices)
- Study start
- Mar 2019 (estimated)
- Primary completion
- Dec 2020 (estimated)
- Study completion
- Dec 2020 (estimated)
- First posted
- 13 Nov 2015 (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 objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
Detailed description
This Phase 3, prospective, randomized, double-blinded, placebo-controlled, multi-center study will enroll approximately 210 subjects at up to 70 sites in the United States. Participation will consist of a Screening Period, followed by a minimum 12-month Treatment Period. SC411 is administered orally as a soft gel mini capsule.
This study will enroll subjects aged ≥5 to ≤17 years who have a diagnosis of SCD that includes the phenotypes hemoglobin SS homozygous (HbSS), hemoglobin SC (HbSC), and hemoglobin S/β°-thalassemia (HbS/ β°-thalassemia); and have had at least 2 but no more than 10 documented SCC events (as defined above) within 12 months prior to the Screening Visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SC411 | Experimental | Drug: SC411Omega-3 docosahexaenoic acid, soft gelatin capsule, administered once a day on a per weight basis. |
| Placebo | Placebo comparator | Drug: PlaceboSoybean oil, soft gelatin capsule, administered once a day on a per weight basis. |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Patients who meet all of the following criteria will be eligible to participate in the study:
- 1. Aged ≥ 5 years and ≤ 17 years at screening;
- Has been diagnosed with SCD (that includes the genotypes HbSS, HbSC, and HbS/β°-thalassemia, documented by hemoglobin HPLC or electrophoresis);
- Has had between ≥ 2 to ≤10 episodes of acute SCC (as defined above) within 12 months of the Screening Visit. At least one crisis must have been managed in a hospital, clinic, or emergency room. For at least 2 of the episodes, the site must obtain the documentation created in a medical record at the time of the event.
- Must not be receiving HU or L-Glutamine, or if receiving HU and/or L-Glutamine must be at a stable weight-based treatment regimen (mg/kg), for at least 3 months prior to the Screening Visit with the intent to continue at a weight-based dose level at the discretion of the treating physician for the duration of the study, other than for safety reasons. If taking HU and/or L-glutamine, subjects must have had at least one SCC event (as previously defined) while on HU and/or L-Glutamine.
Key exclusion criteria (21 listed)
- Patients who meet any of the following criteria will be excluded from participation in the study:
- A known allergy or hypersensitivity to fish or shellfish;
- A known allergy or hypersensitivity to soy;
- Inability to swallow capsules;
- History of treatment with SC411;
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 |
|---|---|---|
| SC411Also: Docosahexaenoic acid (DHA)Soft gelatin capsule | Drug | SC411 |
| PlaceboAlso: Soybean OilSoft gelatin capsule | Drug | Placebo |
Primary outcome
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization.
Time frame: 52 weeks
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization. The primary objective of this study is to assess the efficacy of orally administered SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects compared to placebo. A SCC event will be defined as either an acute painful crisis or an acute chest syndrome.
4 secondary outcomes
Evaluation of the effect of SC411 compared to placebo by measuring the time until the patient's first sickle cell event.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the number of crisis-free days.
Time frame: 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2019.
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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.
Carton Dampier, MD study chair
Emory University
Matt Heeney, MD study chair
Harvard University
Beng Fuh, MD study chair
East Carolina University
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SC411 in people with Sickle Cell Disease, run by Micelle BioPharma Inc.
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 7 inclusion and 21 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: status not confirmed, as of the sponsor's last update on 1 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Uche Sampson, MD (615-678-2336, usampson@micellebio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization. (time frame: 52 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 1 Feb 2019.