Sickle Cell DiseasePhase 3NCT01142219
L-Arginine and Sickle Cell Disease
A Phase 3 study testing L-arginine in people with Sickle Cell Disease, run by Hospital de Clinicas de Porto Alegre at 1 site in Brazil.
Trial summary
One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- L-arginine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteBrazil
- Study dates
- Sep 2006to Oct 2009
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of sickle cell disease confirmed through hemoglobin electrophoresis
- Genotypes SS, SC and Sβ thalassemia
- Age > 1 year
Who cannot join
- Liver dysfunction (alanine aminotransferase ALT > 3 times the normal levels
- Renal dysfunction (creatinine > twice the normal levels)
- Increase in methemoglobin levels (> 5 times the normal levels)
- History of recent stroke (< 1 month) and priapism
- Pregnancy
- Allergy to L-arginine
- Use of sildenafil, calcium channel blockers, nitroglycerin or other nitrates
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-001
About this study
Official title: A Randomized, Controlled, Double-blind Clinical Trial of L-arginine as Adjuvant Therapy for Sickle Cell Disease
One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects.
Read the detailed description
This is a phase III, randomized, double-blind, placebo-controlled clinical trial with sickle cell disease patients older than 1 year of age. The patients were randomly assigned to take 0.1 g/kg/day of either L-arginine or placebo orally. Adverse events were monitored by a safety committee. The variables were assessed while patients were in remission, as part of their routine care: weight, blood pressure, full blood cell count, creatinine and dosage of methemoglobin at baseline and at each follow-up visit. Peripheral oxygen saturation (SpO2), fetal hemoglobin, lactate dehydrogenase and tricuspid regurgitant jet velocity were measured through transthoracic Doppler echocardiogram before and after treatment.
- Conditions
- Sickle Cell Disease
- Being tested
- L-arginine, Placebo
- Keywords
- sickle cell disease, L-arginine, hemolysis, nitric oxide, pulmonary arterial hypertension
- Medical subject headings
- Anemia, Sickle Cell, Hemolysis, Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-arginineDrug
L-arginine was administered orally at a dose of 0.1g/kg/day during 6 months.
PlaceboDrug
Mannitol was administered orally at a dose of 01 g/kg/day for six months.
| Study group | Role | What they receive |
|---|---|---|
| L-arginine | Experimental | 0.1g/kg/day for 6 months Receives: Drug: L-arginine; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tricuspid regurgitant jet velocity >2.5 m/s
Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.
Measured: 6 months
Secondary outcomes (1)
Lactate dehydrogenase levels
The effect of L-argigine administration on hemolysis was assessed through the variation of serum levels of lactate dehydrogenase.
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 posted11 Jun 2010 (estimated)
- Study startSep 2006
- Primary completionJan 2008
- Study completionOct 2009
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.
- More trials run by Hospital de Clinicas de Porto AlegreThe sponsor's other registered studies.
- 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
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital de Clinicas de Porto Alegre
- Sponsor type
- University, hospital or other
- Collaborators
- Fundo de Incentivo à Pesquisa do Hospital de Clinicas de Porto Alegre (FIPE), Associação de Amigos da Hematologia (HEMOAMIGOS), Ajinomoto Interamericana Indústria e Comércio Ltda. (donation of L-arginine)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01142219
- Sponsor's protocol ID
- 04-487 cep@hcpa.ufrgs.br
- Status last verified
- Jun 2010
- First submitted
- 10 Jun 2010
- Last updated
- 11 Jun 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Controlled, Double-blind Clinical Trial of L-arginine as Adjuvant Therapy for Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01142219
- Acronym / sponsor ID
- 04-487 cep@hcpa.ufrgs.br
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, L-arginine, hemolysis, nitric oxide, pulmonary arterial hypertension
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Hospital de Clinicas de Porto AlegreUniversity, hospital or other
- Collaborators
- Fundo de Incentivo à Pesquisa do Hospital de Clinicas de Porto Alegre (FIPE), Associação de Amigos da Hematologia (HEMOAMIGOS), Ajinomoto Interamericana Indústria e Comércio Ltda. (donation of L-arginine)
- Study start
- Sep 2006
- Primary completion
- Jan 2008 (actual)
- Study completion
- Oct 2009 (actual)
- First posted
- 11 Jun 2010 (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.
One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects.
Detailed description
This is a phase III, randomized, double-blind, placebo-controlled clinical trial with sickle cell disease patients older than 1 year of age. The patients were randomly assigned to take 0.1 g/kg/day of either L-arginine or placebo orally. Adverse events were monitored by a safety committee. The variables were assessed while patients were in remission, as part of their routine care: weight, blood pressure, full blood cell count, creatinine and dosage of methemoglobin at baseline and at each follow-up visit. Peripheral oxygen saturation (SpO2), fetal hemoglobin, lactate dehydrogenase and tricuspid regurgitant jet velocity were measured through transthoracic Doppler echocardiogram before and after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| L-arginine | Experimental | Drug: L-arginine, Drug: Placebo0.1g/kg/day for 6 months |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of sickle cell disease confirmed through hemoglobin electrophoresis
- Genotypes SS, SC and Sβ thalassemia
- Age > 1 year
Key exclusion criteria (7 listed)
- Liver dysfunction (alanine aminotransferase ALT > 3 times the normal levels
- Renal dysfunction (creatinine > twice the normal levels)
- Increase in methemoglobin levels (> 5 times the normal levels)
- History of recent stroke (< 1 month) and priapism
- Pregnancy
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 |
|---|---|---|
| L-arginineL-arginine was administered orally at a dose of 0.1g/kg/day during 6 months. | Drug | L-arginine |
| PlaceboMannitol was administered orally at a dose of 01 g/kg/day for six months. | Drug | L-arginine |
Primary outcome
Tricuspid regurgitant jet velocity >2.5 m/s
Time frame: 6 months
Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.
1 secondary outcome
Lactate dehydrogenase levels
Time frame: 6 months
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 11 Jun 2010.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing L-arginine in people with Sickle Cell Disease, run by Hospital de Clinicas de Porto Alegre at 1 site in Brazil.
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 3 inclusion and 7 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 11 Jun 2010. 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 Brazil. 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 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?
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?
Tricuspid regurgitant jet velocity >2.5 m/s (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 11 Jun 2010; AzTrials last refreshed its copy on 6 Nov 2026.