Anemia, Sickle CellPhase 2NCT01054768
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
A Phase 2 study testing alpha-lipoic acid and acetyl-L-carnitine in people with Anemia, Sickle Cell, run by UCSF Benioff Children's Hospital Oakland at 1 site in United States.
Trial summary
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the frequency of vaso-occlusive pain episodes and improve their quality of life. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- alpha-lipoic acid and acetyl-L-carnitine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2009to Apr 2013
- Phase
- Phase 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Proven diagnosis of sickle cell disease, either homozygous sickle disease or Hb S Beta zero thalassemia genotype
- Age at entry at least 14 years. Younger children will not be included since the combination alpha-lipoic acid and acetyl-L-carnitine tablets are not available in a smaller dose at this time.
Who cannot join
- More than 3 packed red blood transfusions in the past 12 months
- Coexisting illness that could contribute to inflammation. These include chronic hepatitis, lupus, arthritis, inflammatory bowel disease, chronic osteomyelitis, and other similar conditions.
- Acute sickle cell disease related symptoms requiring a hospital visit in the past 4 weeks
- Women who are pregnant, attempting to get pregnant, or breast feeding
- Active participation in other investigational drug or device studies
- Participants who start hydroxyurea or regular transfusion therapy during the course of the study on the recommendation of their primary hematologist will be ineligible for further participation.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital & Research Center Oakland
Oakland, California, 94609
About this study
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the frequency of vaso-occlusive pain episodes and improve their quality of life.
Read the detailed description
People with sickle cell disease have more inflammation (a response of body tissues to injury or irritation) than people without sickle cell disease. This inflammation can be measured in the blood by checking the level of a protein called C reactive protein as well as other changes we see in blood due to inflammation (such as changes in platelets and other cells). There is even more inflammation during sickle-related complications (like pain or acute chest syndrome). We want to test if inflammation in people with sickle cell disease can be reduced by the use of antioxidant compounds.
Antioxidants are nutrients (certain vitamins, minerals and enzymes) that can counteract the effects of oxidative stress arising from free radicals in our cells. The formation of free radicals is a normal cell process, but uncontrolled oxidative stress can cause problems for us. One such harmful problem is inflammation.
We know from other research studies that antioxidants help with some conditions related to inflammation. In this study the antioxidant being tested is a combination of alpha-lipoic acid and acetyl-L-carnitine, both of which are natural parts of many of the foods we eat and are needed by our cells to make energy from food.
- Conditions
- Anemia, Sickle Cell
- Being tested
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Keywords
- Antioxidant, Inflammation, Sickle Cell Disease, Oxidative Stress, Cytokines, anti-inflammatory, Acetylcarnitine
- Medical subject headings
- Anemia, Sickle Cell, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alpha-lipoic acid and acetyl-L-carnitineDrug
Also called Experimental
none to report
ControlDrug
Also called Placebo
none to report
| Study group | Role | What they receive |
|---|---|---|
| alpha-lipoic acid and acetyl-L-carnitine | Experimental | alpha-lipoic acid and acetyl-L-carnitine1400 mg tablet twice a day for 6 months. Receives: Drug: alpha-lipoic acid and acetyl-L-carnitine |
| Placebo | Placebo comparator | 1400 mg placebo tablet twice a day for 6 months. Receives: Drug: Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
C-Reactive Protein
C-Reactive Protein (CRP) was measured by a clinical laboratory. Measuring the quantity of serum CRP is helpful in recognizing inflammatory states.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2010 (estimated)
- Study startAug 2009
- Primary completionApr 2013
- Study completionApr 2013
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.
- Other trials for Anemia, Sickle CellStudies 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Sponsor type
- University, hospital or other
- Collaborators
- National Center for Complementary and Integrative Health (NCCIH)
- Responsible party
- Sponsor
- Investigators
- Elliott Vichinsky, M.D., Principal investigator, UCSF Benioff Children's Hospital OaklandBruce N. Ames, Ph.D., Study chair, UCSF Benioff Children's Hospital OaklandAshutosh Lal, M.D., Study director, UCSF Benioff Children's Hospital Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01054768
- Sponsor's protocol ID
- 2009-003
- Other IDs
- 1R21AT004493-01
- Status last verified
- Jul 2021
- First submitted
- 20 Jan 2010
- Last updated
- 3 Aug 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01054768
- Acronym / sponsor ID
- 2009-003
- Conditions
- Anemia, Sickle Cell
- Keywords
- Antioxidant, Inflammation, Sickle Cell Disease, Oxidative Stress, Cytokines, anti-inflammatory, Acetylcarnitine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- UCSF Benioff Children's Hospital OaklandUniversity, hospital or other
- Collaborators
- National Center for Complementary and Integrative Health (NCCIH)
- Study start
- Aug 2009
- Primary completion
- Apr 2013 (actual)
- Study completion
- Apr 2013 (actual)
- First posted
- 22 Jan 2010 (estimated)
- Results
- Posted 3 Aug 2021
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 whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the frequency of vaso-occlusive pain episodes and improve their quality of life.
Detailed description
People with sickle cell disease have more inflammation (a response of body tissues to injury or irritation) than people without sickle cell disease. This inflammation can be measured in the blood by checking the level of a protein called C reactive protein as well as other changes we see in blood due to inflammation (such as changes in platelets and other cells). There is even more inflammation during sickle-related complications (like pain or acute chest syndrome). We want to test if inflammation in people with sickle cell disease can be reduced by the use of antioxidant compounds.
Antioxidants are nutrients (certain vitamins, minerals and enzymes) that can counteract the effects of oxidative stress arising from free radicals in our cells. The formation of free radicals is a normal cell process, but uncontrolled oxidative stress can cause problems for us. One such harmful problem is inflammation.
We know from other research studies that antioxidants help with some conditions related to inflammation. In this study the antioxidant being tested is a combination of alpha-lipoic acid and acetyl-L-carnitine, both of which are natural parts of many of the foods we eat and are needed by our cells to make energy from food.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| alpha-lipoic acid and acetyl-L-carnitine | Experimental | Drug: alpha-lipoic acid and acetyl-L-carnitinealpha-lipoic acid and acetyl-L-carnitine1400 mg tablet twice a day for 6 months. |
| Placebo | Placebo comparator | Drug: Control1400 mg placebo tablet twice a day for 6 months. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Proven diagnosis of sickle cell disease, either homozygous sickle disease or Hb S Beta zero thalassemia genotype
- Age at entry at least 14 years. Younger children will not be included since the combination alpha-lipoic acid and acetyl-L-carnitine tablets are not available in a smaller dose at this time.
Key exclusion criteria (6 listed)
- More than 3 packed red blood transfusions in the past 12 months
- Coexisting illness that could contribute to inflammation. These include chronic hepatitis, lupus, arthritis, inflammatory bowel disease, chronic osteomyelitis, and other similar conditions.
- Acute sickle cell disease related symptoms requiring a hospital visit in the past 4 weeks
- Women who are pregnant, attempting to get pregnant, or breast feeding
- Active participation in other investigational drug or device studies
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 |
|---|---|---|
| alpha-lipoic acid and acetyl-L-carnitineAlso: Experimentalnone to report | Drug | alpha-lipoic acid and acetyl-L-carnitine |
| ControlAlso: Placebonone to report | Drug | Placebo |
Primary outcome
C-Reactive Protein
Time frame: 6 months
C-Reactive Protein (CRP) was measured by a clinical laboratory. Measuring the quantity of serum CRP is helpful in recognizing inflammatory states.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Elliott Vichinsky, M.D. principal investigator
UCSF Benioff Children's Hospital Oakland
Bruce N. Ames, Ph.D. study chair
UCSF Benioff Children's Hospital Oakland
Ashutosh Lal, M.D. study director
UCSF Benioff Children's Hospital Oakland
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 alpha-lipoic acid and acetyl-L-carnitine in people with Anemia, Sickle Cell, run by UCSF Benioff Children's Hospital Oakland 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 2 inclusion and 6 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 3 Aug 2021. 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. 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?
Yes. Results are posted in the registry (first posted 3 Aug 2021). 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?
C-Reactive Protein (time frame: 6 months). 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 Aug 2021.