Sickle Cell DiseasePhase 1/2NCT02947100
Omega-3 Fatty Acids in Sickle Cell Disease
A Phase 1/2 study testing SCD-Omegatex™ in people with Sickle Cell Disease, run by Robin E. Miller at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease. Read more about this study
- Who is it for?
- Ages 8 to 25 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- SCD-Omegatex™
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jan 2018to 15 Oct 2018
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 8 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects who meet all of the following criteria are eligible for enrollment into the study:
- Participant has signed the informed consent/assent with parent signing informed consent as age appropriate.
- Established diagnosis of HbSS, HbSC or HbSβo Thalassemia
- History of ≥1 vasoocclusive events (managed at home and/or in hospital) in preceding 12 months.
- Regular compliance with comprehensive care.
- Aged 8 years or greater and less than 26 years.
- At enrollment, subject should be in his/her baseline steady state and not in the midst of any acute complication due to SCD. Must be at least 2 weeks from infection or vasoocclusive crisis at time of screening labs
Who cannot join
- Baseline hemoglobin levels <5.5 gm/dL.
- Inability to swallow capsules
- Poor compliance with previous treatment regimens.
- Hepatic dysfunction
- Renal dysfunction
- PT and/or PTT ≥ 20% outside of normal
Show all 19Show fewer
- Allergy to fish, shell fish or soy
- Triglyceride levels <80mg/dL.
- Pregnancy.
- Chronic Transfusion Therapy.
- Transfusion within the last 30 days.
- Treatment with any investigational drug or regular fish oil supplementations in last 60 days.
- Currently receiving another investigational agent, or on such an agent with the last 60 days.
- Dosage changes in preceding 3 months if on hydroxyurea
- Diagnosed bleeding disorder or patient on concomitant anti-coagulation.
- Conditional or abnormal result on most recent transcranial doppler or history of stroke.
- Other active chronic illness that could adversely affect subjects performance
- Children in Care
- Platelet count less than 100,000
Trial locations and contacts
1 site in 1 country.
United States1 site
Nemours/Alfred I duPont Hospital for Children
Wilmington, Delaware, 19899
About this study
Official title: Phase I/II Safety and Dose Escalation Trial of the Omega-3 Fatty Acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) in Children and Young Adults With Sickle Cell Disease (SCD)
The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease.
- Conditions
- Sickle Cell Disease
- Being tested
- SCD-Omegatex™
- Keywords
- Sickle Cell Disease, omega-3 fatty acids, Docosahexaenoic Acid (DHA), Eicosapentaenoic Acid (EPA)
- 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.
SCD-Omegatex™Drug
Also called Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule
Subjects will receive SCD-Omegatex™ (Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule, 450 mg) at one of two daily doses, orally, once a day for 6 months. The trial will follow a "3+3" design using two dose levels. In the phase I portion, subjects will be treated with a dose of 25 mg/kg/day DHA and EPA. If this is tolerated without dose limiting toxicity (DLT), a subsequent cohort of patients will be treated at a dose of 37.5 mg/kg/day with a maximum total daily dose of 4 grams. Once a maximum tolerated dose (MTD) is determined, subjects on the phase II portion of the study will be treated at that dose.
| Study group | Role | What they receive |
|---|---|---|
| SCD-Omegatex™ | Experimental | single arm Receives: Drug: SCD-Omegatex™ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Safety, in a Dose Escalation Trial of SCD-Omegatex™ as Evidenced by an Absence of Adverse Events.
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Measured: 6 months with continuous monitoring
Determine Whether 6 Months of Supplementation With SCD-Omegatex™ Will Reduce Thermal Sensitivity by Quantitative Sensory Testing to Below Pre-treatment Levels
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Measured: 6 months
Secondary outcomes (3)
Health-associated Quality of Life
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Measured: 6 months
Number of Days With Pain Measured by iPad Daily Report Pain Calendar
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Measured: 8 months
Changes in Individual Thermal Sensitivity Thresholds by QST
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Measured: 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2016 (estimated)
- Study start25 Jan 2018
- Primary completion5 Oct 2018
- Study completion15 Oct 2018
Why it stopped: manufacturing problem with study drug
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Robin E. Miller
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of General Medical Sciences (NIGMS), Thomas Jefferson University, Solutex GC S.L.
- Responsible party
- Sponsor-investigator, Robin E. Miller, Nemours Children's Clinic
- Investigators
- Robin E Miller, MD, Principal investigator, Nemours Children's Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02947100
- Sponsor's protocol ID
- RM002
- Other IDs
- P20GM109021
- Status last verified
- Sep 2020
- First submitted
- 18 Oct 2016
- Last updated
- 19 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Daak AA, Ghebremeskel K, Hassan Z, Attallah B, Azan HH, Elbashir MI, Crawford M. Effect of omega-3 (n-3) fatty acid supplementation in patients with sickle cell anemia: randomized, double-blind, placebo-controlled trial. Am J Clin Nutr. 2013 Jan;97(1):37-44. doi: 10.3945/ajcn.112.036319. Epub 2012 Nov 28.
PubMed 23193009Serhan CN. Resolution phase of inflammation: novel endogenous anti-inflammatory and proresolving lipid mediators and pathways. Annu Rev Immunol. 2007;25:101-37. doi: 10.1146/annurev.immunol.25.022106.141647.
PubMed 17090225Calder PC. Marine omega-3 fatty acids and inflammatory processes: Effects, mechanisms and clinical relevance. Biochim Biophys Acta. 2015 Apr;1851(4):469-84. doi: 10.1016/j.bbalip.2014.08.010. Epub 2014 Aug 20.
PubMed 25149823Tomer A, Kasey S, Connor WE, Clark S, Harker LA, Eckman JR. Reduction of pain episodes and prothrombotic activity in sickle cell disease by dietary n-3 fatty acids. Thromb Haemost. 2001 Jun;85(6):966-74.
PubMed 11434703
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Safety and Dose Escalation Trial of the Omega-3 Fatty Acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) in Children and Young Adults With Sickle Cell Disease (SCD)
- ClinicalTrials.gov ID
- NCT02947100
- Acronym / sponsor ID
- RM002
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, omega-3 fatty acids, Docosahexaenoic Acid (DHA), Eicosapentaenoic Acid (EPA)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: manufacturing problem with study drug
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Robin E. MillerUniversity, hospital or other
- Collaborators
- National Institute of General Medical Sciences (NIGMS), Thomas Jefferson University, Solutex GC S.L.
- Study start
- 25 Jan 2018 (actual)
- Primary completion
- 5 Oct 2018 (actual)
- Study completion
- 15 Oct 2018 (actual)
- First posted
- 27 Oct 2016 (estimated)
- Results
- Posted 19 Oct 2020
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 determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SCD-Omegatex™ | Experimental | Drug: SCD-Omegatex™single arm |
Who may qualify
- Ages
- 8 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Subjects who meet all of the following criteria are eligible for enrollment into the study:
- Participant has signed the informed consent/assent with parent signing informed consent as age appropriate.
- Established diagnosis of HbSS, HbSC or HbSβo Thalassemia
- History of ≥1 vasoocclusive events (managed at home and/or in hospital) in preceding 12 months.
- Regular compliance with comprehensive care.
Key exclusion criteria (19 listed)
- Baseline hemoglobin levels <5.5 gm/dL.
- Inability to swallow capsules
- Poor compliance with previous treatment regimens.
- Hepatic dysfunction
- Renal dysfunction
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 |
|---|---|---|
| SCD-Omegatex™Also: Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin CapsuleSubjects will receive SCD-Omegatex™ (Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule, 450 mg) at one of two daily doses, orally, once a day for 6 months. The trial will follow a "3+3" design using two dose levels. In the phase I portion, subjects will be treated with a dose of 25 mg/kg/day DHA and EPA. If this is tolerated without dose limiting toxicity (DLT), a subsequent cohort of patients will be treated at a dose of 37.5 mg/kg/day with a maximum total daily dose of 4 grams. Once a maximum tolerated dose (MTD) is determined, subjects on the phase II portion of the study will be treated at that dose. | Drug | SCD-Omegatex™ |
Primary outcome
Clinical Safety, in a Dose Escalation Trial of SCD-Omegatex™ as Evidenced by an Absence of Adverse Events.
Time frame: 6 months with continuous monitoring
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
Determine Whether 6 Months of Supplementation With SCD-Omegatex™ Will Reduce Thermal Sensitivity by Quantitative Sensory Testing to Below Pre-treatment Levels
Time frame: 6 months
No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened.
3 secondary outcomes
Health-associated Quality of Life
Time frame: 6 months
Number of Days With Pain Measured by iPad Daily Report Pain Calendar
Time frame: 8 months
Changes in Individual Thermal Sensitivity Thresholds by QST
Time frame: 8 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23193009Daak AA, Ghebremeskel K, Hassan Z, Attallah B, Azan HH, Elbashir MI, Crawford M. Effect of omega-3 (n-3) fatty acid supplementation in patients with sickle cell anemia: randomized, double-blind, placebo-controlled trial. Am J Clin Nutr. 2013 Jan;97(1):37-44. doi: 10.3945/ajcn.112.036319. Epub 2012 Nov 28.
- Background: PubMed 17090225Serhan CN. Resolution phase of inflammation: novel endogenous anti-inflammatory and proresolving lipid mediators and pathways. Annu Rev Immunol. 2007;25:101-37. doi: 10.1146/annurev.immunol.25.022106.141647.
- Background: PubMed 25149823Calder PC. Marine omega-3 fatty acids and inflammatory processes: Effects, mechanisms and clinical relevance. Biochim Biophys Acta. 2015 Apr;1851(4):469-84. doi: 10.1016/j.bbalip.2014.08.010. Epub 2014 Aug 20.
- Background: PubMed 11434703Tomer A, Kasey S, Connor WE, Clark S, Harker LA, Eckman JR. Reduction of pain episodes and prothrombotic activity in sickle cell disease by dietary n-3 fatty acids. Thromb Haemost. 2001 Jun;85(6):966-74.
- 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.
Robin E Miller, MD principal investigator
Nemours Children's Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SCD-Omegatex™ in people with Sickle Cell Disease, run by Robin E. Miller at 1 site 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 7 inclusion and 19 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 19 Oct 2020. 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 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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?
Yes. Results are posted in the registry (first posted 19 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Clinical Safety, in a Dose Escalation Trial of SCD-Omegatex™ as Evidenced by an Absence of Adverse Events. (time frame: 6 months with continuous monitoring). The study lists 2 primary outcomes in all. 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 NCT02947100, which also lists 4 linked publications.
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 19 Oct 2020; AzTrials last refreshed its copy on 6 Nov 2026.