Vaso-occlusive Pain EpisodesPhase 2NCT01796678
Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
A Phase 2 study testing Arginine in people with Vaso-occlusive Pain Episodes, run by UCSF Benioff Children's Hospital Oakland at 1 site in United States.
Trial summary
Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits. Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Vaso-occlusive Pain Episodes
- What is being tested?
- Arginine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2000to Jun 2007
- 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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Established Diagnosis of Sickle Cell Disease(SS,SC,S-beta thal)
- Admitted to Hospital for pain
- Pain requiring hospitalization for parenteral narcotics, not attributable to non-sickle cell causes
- >3 year and older
Who cannot join
- Hemoglobin less than 5gm/dL or immediate need for red cell transfusion
- Hepatic Dysfunction: increased in SGPT to >2x normal value
- Renal Dysfunction: increased in creatinine to >2x normal value or >1.5
- Mental status or neurological changes
- Pregnancy
- >10 Hospitalizations per year or history of dependance to narcotics
Show all 9Show fewer
- Inability to take oral medications or allergy to arginine
- Inability to use a PCA device
- < 3 years of age
Trial locations and contacts
1 site in 1 country.
United States1 site
Childrens Hospital Research Center Oakland
Oakland, California, 94609
About this study
Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits. Currently efforts to treat painful episodes with use of non-steroidal pain relievers and intravenous narcotics offer symptomatic relief only, without targeting the underlying mechanisms of vaso-occlusion.Investigators have found that an arginine deficiency and low NO bioavailability occurs during painful events in SCD. Since arginine is the obligate substrate for NO production, and an acute deficiency is associated with VOE, investigators hypothesized that arginine supplementation may be a safe and beneficial treatment for sickle cell pain.
- Conditions
- Vaso-occlusive Pain Episodes
- Being tested
- Arginine, Placebo
- Keywords
- sickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxide
- 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.
ArginineDrug
Also called L-arginine, L-arginine-HCL
PlaceboDrug
Saline or Sugar pill was given as placebo
| Study group | Role | What they receive |
|---|---|---|
| Arginine | Experimental | 100 mg/kg T.I.D 3x a day IV or PO Receives: Drug: Arginine |
| Placebo | Placebo comparator | Saline or sugar pill Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Length of Hospital Stay
Measured: participants will be followed for the duration of hospital stay an expected average of 3-6 days, with re-admission data being collect for up to 30 days
Secondary outcomes (2)
Effect on Pain Score
Measured: participants will be followed for the duration of hospital stay an expected average of 3-6 days
Total Opioid Use (mg/kg)
Measured: participants will be followed for the duration of hospital stay an expected average of 3-6 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2013 (estimated)
- Study startSep 2000
- Primary completionJun 2007
- Study completionJun 2007
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 Vaso-occlusive Pain EpisodesStudies 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
- Single group
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Claudia R Morris, MD, Principal investigator, Childrens Hospital Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01796678
- Sponsor's protocol ID
- HL 14386-05
- Status last verified
- Jul 2013
- First submitted
- 19 Feb 2013
- Last updated
- 31 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Morris CR, Kuypers FA, Lavrisha L, Ansari M, Sweeters N, Stewart M, Gildengorin G, Neumayr L, Vichinsky EP. A randomized, placebo-controlled trial of arginine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes. Haematologica. 2013 Sep;98(9):1375-82. doi: 10.3324/haematol.2013.086637. Epub 2013 May 3.
PubMed 23645695
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
- ClinicalTrials.gov ID
- NCT01796678
- Acronym / sponsor ID
- HL 14386-05
- Conditions
- Vaso-occlusive Pain Episodes
- Keywords
- sickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- UCSF Benioff Children's Hospital OaklandUniversity, hospital or other
- Study start
- Sep 2000
- Primary completion
- Jun 2007 (actual)
- Study completion
- Jun 2007 (actual)
- First posted
- 22 Feb 2013 (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.
Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits. Currently efforts to treat painful episodes with use of non-steroidal pain relievers and intravenous narcotics offer symptomatic relief only, without targeting the underlying mechanisms of vaso-occlusion.Investigators have found that an arginine deficiency and low NO bioavailability occurs during painful events in SCD. Since arginine is the obligate substrate for NO production, and an acute deficiency is associated with VOE, investigators hypothesized that arginine supplementation may be a safe and beneficial treatment for sickle cell pain.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arginine | Experimental | Drug: Arginine100 mg/kg T.I.D 3x a day IV or PO |
| Placebo | Placebo comparator | Drug: PlaceboSaline or sugar pill |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Established Diagnosis of Sickle Cell Disease(SS,SC,S-beta thal)
- Admitted to Hospital for pain
- Pain requiring hospitalization for parenteral narcotics, not attributable to non-sickle cell causes
- >3 year and older
Key exclusion criteria (9 listed)
- Hemoglobin less than 5gm/dL or immediate need for red cell transfusion
- Hepatic Dysfunction: increased in SGPT to >2x normal value
- Renal Dysfunction: increased in creatinine to >2x normal value or >1.5
- Mental status or neurological changes
- 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 |
|---|---|---|
| ArginineAlso: L-arginine, L-arginine-HCL | Drug | Arginine |
| PlaceboSaline or Sugar pill was given as placebo | Drug | Placebo |
Primary outcome
Length of Hospital Stay
Time frame: participants will be followed for the duration of hospital stay an expected average of 3-6 days, with re-admission data being collect for up to 30 days
2 secondary outcomes
Effect on Pain Score
Time frame: participants will be followed for the duration of hospital stay an expected average of 3-6 days
Total Opioid Use (mg/kg)
Time frame: participants will be followed for the duration of hospital stay an expected average of 3-6 days
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 31 Jul 2013.
- Results publication: PubMed 23645695Morris CR, Kuypers FA, Lavrisha L, Ansari M, Sweeters N, Stewart M, Gildengorin G, Neumayr L, Vichinsky EP. A randomized, placebo-controlled trial of arginine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes. Haematologica. 2013 Sep;98(9):1375-82. doi: 10.3324/haematol.2013.086637. Epub 2013 May 3.
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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.
Claudia R Morris, MD principal investigator
Childrens 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 Arginine in people with Vaso-occlusive Pain Episodes, 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 4 inclusion and 9 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 31 Jul 2013. 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?
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?
Length of Hospital Stay (time frame: participants will be followed for the duration of hospital stay an expected average of 3-6 days, with re-admission data being collect for up to 30 days). 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 NCT01796678, 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 Jul 2013.