Sickle Cell DiseasePhase 2SCOTNCT02973360
Dose-Finding Study of SC411 in Children With Sickle Cell Disease
A Phase 2 study testing Docosahexaenoic Acid in people with Sickle Cell Disease, run by Micelle BioPharma Inc at 11 sites in United States.
Trial summary
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a 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?
- Docosahexaenoic Acid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 11 sitesUnited States
- Study dates
- 2 Mar 2017to 2 Jan 2022
- 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 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Aged greater than or equal to 5 years and less than or equal to 17 years at screening;
- Has been diagnosed with SCD that includes the phenotypes HbSS, hemoglobin SC, and HbS/beta-thalassemia. Hemoglobin phenotyping must be previously documented by either hemoglobin high-performance liquid chromatography [HPLC] or electrophoresis at time of Screening. If a patient does not have documented hemoglobin phenotyping at the time of Screening, or has received a blood transfusion within the two months prior to the Screening Visit, hemoglobin phenotyping should be documented by hemoglobin HPLC;
- Has had at least two and no more than ten documented episodes of clinical sickle cell crises within 12 months prior to the Screening Visit. A sickle cell crisis is defined as an episode of vaso-occlusive event:
- Painful crisis defined as new onset of pain that lasts two or more hours for which there is no explanation other than vaso-occlusion, and which requires therapy with oral or parenteral opioids, non-steroidal anti-inflammatory drugs, or other analgesics prescribed by a healthcare provider in a medical setting such as a hospital, clinic, or emergency room visit, or documented telephone management (Ballas, 2010; Heeney, 2016; Jacob, 2005); and
- Acute chest syndrome defined as acute illness characterized by a new segmental pulmonary infiltrate on a chest x-ray, and fever (greater than or equal to 38.5°C) or respiratory symptoms such as hypoxia, chest pain, tachypnea, wheezing, or cough (Ballas, 2010);
- Is either not on hydroxyurea at the Screening Visit and does not plan on receiving it during the course of the first 12 weeks of the study (Part A), or has received hydroxyurea for a minimum of 6 months and, except for safety reasons, will remain on the same weight-based dose of hydroxyurea from screening throughout the duration of Part A of the study;
- Parent or guardian is able to give written informed consent, and the potential pediatric patient is able to provide assent in a manner approved by the Institutional Review Board (IRB) and comply with the requirements of the study other than for safety reasons; and
- If sexually active, agrees to use a reliable method of birth control (eg, barrier, birth control pills, abstinence) during the study and for one month following the last dose of study drug.
Who cannot join
- Has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the Screening Visit;
- Has a known allergy or hypersensitivity to fish or shellfish;
- Has a known allergy or hypersensitivity to soy;
- Is planning to initiate, terminate, or alter the dosing of hydroxyurea during the first 12 weeks of the study, other than for safety reasons;
- Has chronic daily use (more than 30 consecutive days during the last six months prior to enrollment) of opioid analgesia for any reason;
- Has a diagnosis of chronic pain or chronic pain syndrome (eg, chronic pain from the repeated vaso-occlusive events, chronic pain from avascular necrosis);
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- Has a history of Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C infection;
- Has a history of documented episode(s) of priapism within 12 months of the Screening Visit;
- Has a history of atrial or ventricular arrhythmia;
- Has an international normalized ratio (INR) >2.0, or is on regular anticoagulation therapy, or has a history of a known bleeding diathesis;
- Has thrombocytopenia (platelets less than 80,000) or is on chronic acetylsalicylic acid therapy;
- Has increased risk of stroke: documented abnormal or "high conditional" transcranial Doppler (TCD) mean velocity (TCD V) by STOP criteria (Adams, 1998) within the preceding year or has a history of known cerebrovascular disease:
- "High conditional" = TCD V greater than or equal to 185 to 199 cm/sec, or TCDi V greater than or equal to 170 to 184 cm/sec, or TCD maximum V greater than or equal to 250 cm/sec; or
- Abnormal = TCD V greater than or equal to 200 cm/sec, or abnormal high TCDi V greater than or equal to 185 cm/sec, or TCD maximum V greater than or equal to 250 cm/sec;
- Has received a blood transfusion or exchange transfusion in the three months prior to the Screening Visit;
- Has renal insufficiency (creatinine greater than 1.5 times upper limit of normal [ULN], or requiring peritoneal dialysis or hemodialysis);
- Has liver dysfunction (ALT greater than 2.0 times ULN);
- Has other concomitant chronic medical or psychiatric condition that, in the opinion of the Investigator, would compromise participation in the study 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 patient participating in the study);
- 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 during the course of the study;
- Has been treated with an experimental anti-sickling medication/treatment within 30 days of the Screening Visit or during the course of the study;
- Is currently taking or has been treated with any investigational drug for any disease within 30 days of the Screening Visit or during the course of the study;
- Is currently enrolled in an investigational drug or device study and/or has participated in such a study within 30 days of the Screening Visit or during the course of the study; or
- There are factors that would, in the judgment of the Investigator, make it difficult for the patient to comply with the requirements of the study (eg, inability to swallow capsules due to past history of stroke, or poor compliance).
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Children's Healthcare of Atlanta - Emory University
Atlanta, Georgia, 30322
Children's of Alabama - University of Alabama
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
Medical University of South Carolina
Charleston, South Carolina, 29425
Children's Hospital of Michigan
Detroit, Michigan, 48201
University of Florida Health at Shands
Gainesville, Florida, 32610
East Carolina University
Greenville, North Carolina, 27834
Texas Children's Hospital - Baylor College of Medicine
Houston, Texas, 77030
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Batchelor Children's Research Institute - University of Miami
Miami, Florida, 33136
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Finding Study and Open-Label Extension of SC411 in Children With Sickle Cell Disease
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.
Read the detailed description
This Phase 2 study is to be conducted in two parts. Part A is a randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with SCD. This part of the study will consist of a screening period of up to 2 weeks, followed by an eight-week treatment period. Part B is an optional 49-month open-label extension for patients who have completed Part A.
- Conditions
- Sickle Cell Disease
- Being tested
- Docosahexaenoic Acid, Placebo
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Docosahexaenoic AcidDrug
Also called SC411
Oral Capsule
PlaceboDrug
Also called Soybean oil
Oral Capsule
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1 | Experimental | Target dose 20 mg/kg Docosahexaenoic acid Receives: Drug: Docosahexaenoic Acid |
| Dose Level 2 | Experimental | Target dose 36 mg/kg Docosahexaenoic acid Receives: Drug: Docosahexaenoic Acid |
| Dose Level 3 | Experimental | Target dose 60 mg/kg Docosahexaenoic acid Receives: Drug: Docosahexaenoic Acid |
| Placebo | Placebo comparator | Soybean oil Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate safety & tolerability of SC411 & determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B.
Evaluate the safety and tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and tolerability will continue to be evaluated and reported during Part B.
Measured: Week 0 (baseline) through Month 52 (end of treatment)
Secondary outcomes (4)
Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.
Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.
The steady-state pharmacokinetic (PK) profile of the three doses and area under the curve will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).
Measured: 2 weeks before baseline (screening) through Week 52
Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.
Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.
The steady-state pharmacokinetic (PK) profile of the three doses and Tmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).
Measured: 2 weeks before baseline (screening) through Week 52
Measurement of the pharmacokinetic (PK) parameter of Cmax will be performed for three dose levels of SC411 will be measured.
The steady-state pharmacokinetic (PK) profile of the three doses and Cmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).
Measured: 2 weeks before baseline (screening) through Week 52
The safety & long term tolerability of SC411 will be evaluated in Part A, & a selected dose in Part B. The change from Baseline in blood cells omega-3 fatty acids index in subjects, treated with SC411 or placebo in Part A will be determined.
Evaluate the safety and long term tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and long term tolerability will continue to be evaluated and reported during Part B.
Measured: Week 0 (baseline) through Month 52 (end of treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2016 (estimated)
- Study start2 Mar 2017
- Primary completion26 Sep 2017
- Study completion2 Jan 2022 (estimated)
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 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Micelle BioPharma Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Matthew Heeney, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02973360
- Sponsor's protocol ID
- OMEG-411-02
- Acronym
- SCOT
- Status last verified
- Jan 2019
- First submitted
- 22 Nov 2016
- Last updated
- 31 Jan 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 Aug 14;2(15):1969-1979. doi: 10.1182/bloodadvances.2018021444.
PubMed 30097463
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Finding Study and Open-Label Extension of SC411 in Children With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02973360
- Acronym / sponsor ID
- SCOT / OMEG-411-02
- Conditions
- Sickle Cell Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Micelle BioPharma IncIndustry (pharma, biotech, devices)
- Study start
- 2 Mar 2017 (actual)
- Primary completion
- 26 Sep 2017 (actual)
- Study completion
- 2 Jan 2022 (estimated)
- First posted
- 25 Nov 2016 (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.
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.
Detailed description
This Phase 2 study is to be conducted in two parts. Part A is a randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with SCD. This part of the study will consist of a screening period of up to 2 weeks, followed by an eight-week treatment period. Part B is an optional 49-month open-label extension for patients who have completed Part A.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1 | Experimental | Drug: Docosahexaenoic AcidTarget dose 20 mg/kg Docosahexaenoic acid |
| Dose Level 2 | Experimental | Drug: Docosahexaenoic AcidTarget dose 36 mg/kg Docosahexaenoic acid |
| Dose Level 3 | Experimental | Drug: Docosahexaenoic AcidTarget dose 60 mg/kg Docosahexaenoic acid |
| Placebo | Placebo comparator | Drug: PlaceboSoybean oil |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Aged greater than or equal to 5 years and less than or equal to 17 years at screening;
- Has been diagnosed with SCD that includes the phenotypes HbSS, hemoglobin SC, and HbS/beta-thalassemia. Hemoglobin phenotyping must be previously documented by either hemoglobin high-performance liquid chromatography [HPLC] or electrophoresis at time of Screening. If a patient does not have documented hemoglobin phenotyping at the time of Screening, or has received a blood transfusion within the two months prior to the Screening Visit, hemoglobin phenotyping should be documented by hemoglobin HPLC;
- Has had at least two and no more than ten documented episodes of clinical sickle cell crises within 12 months prior to the Screening Visit. A sickle cell crisis is defined as an episode of vaso-occlusive event:
- Is either not on hydroxyurea at the Screening Visit and does not plan on receiving it during the course of the first 12 weeks of the study (Part A), or has received hydroxyurea for a minimum of 6 months and, except for safety reasons, will remain on the same weight-based dose of hydroxyurea from screening throughout the duration of Part A of the study;
- Parent or guardian is able to give written informed consent, and the potential pediatric patient is able to provide assent in a manner approved by the Institutional Review Board (IRB) and comply with the requirements of the study other than for safety reasons; and
Key exclusion criteria (24 listed)
- Has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the Screening Visit;
- Has a known allergy or hypersensitivity to fish or shellfish;
- Has a known allergy or hypersensitivity to soy;
- Is planning to initiate, terminate, or alter the dosing of hydroxyurea during the first 12 weeks of the study, other than for safety reasons;
- Has chronic daily use (more than 30 consecutive days during the last six months prior to enrollment) of opioid analgesia for any reason;
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 |
|---|---|---|
| Docosahexaenoic AcidAlso: SC411Oral Capsule | Drug | Dose Level 1, Dose Level 2, Dose Level 3 |
| PlaceboAlso: Soybean oilOral Capsule | Drug | Placebo |
Primary outcome
Evaluate safety & tolerability of SC411 & determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B.
Time frame: Week 0 (baseline) through Month 52 (end of treatment)
Evaluate the safety and tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and tolerability will continue to be evaluated and reported during Part B.
4 secondary outcomes
Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.
Time frame: 2 weeks before baseline (screening) through Week 52
Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.
Time frame: 2 weeks before baseline (screening) through Week 52
Measurement of the pharmacokinetic (PK) parameter of Cmax will be performed for three dose levels of SC411 will be measured.
Time frame: 2 weeks before baseline (screening) through Week 52
The safety & long term tolerability of SC411 will be evaluated in Part A, & a selected dose in Part B. The change from Baseline in blood cells omega-3 fatty acids index in subjects, treated with SC411 or placebo in Part A will be determined.
Time frame: Week 0 (baseline) through Month 52 (end of treatment)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2019.
- Results publication: PubMed 30097463Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 Aug 14;2(15):1969-1979. doi: 10.1182/bloodadvances.2018021444.
- EFFECTS OF SC411 ON BLOOD CELL MEMBRANE OMEGA-3 INDEX AND SELECT SICKLE CELL DISEASE BIOMARKERS IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTI-CENTER STUDYLink provided by the sponsor.
- CLINICAL EFFECT OF SC411 ON CHILDREN WITH SICKLE CELL DISEASE IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, DOSE-FINDING MULTI-CENTER STUDYLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Matthew Heeney, MD principal investigator
Boston Children's Hospital
11 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 Docosahexaenoic Acid in people with Sickle Cell Disease, run by Micelle BioPharma Inc at 11 sites in United States.
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 24 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 31 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. 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 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?
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?
Evaluate safety & tolerability of SC411 & determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B. (time frame: Week 0 (baseline) through Month 52 (end of treatment)). 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 NCT02973360, 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 31 Jan 2019.