Sickle Cell DiseasePhase 2NCT02536170
Arginine Therapy for Sickle Cell Disease Pain
A Phase 2 study testing L-arginine and L-arginine Loading Dose in people with Sickle Cell Disease and Vaso-occlusive Pain Episode, run by Emory University at 3 sites in United States.
Trial summary
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores, decrease the need for pain medications or decrease length of hospital stay or emergency… Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Sickle Cell Disease, Vaso-occlusive Pain Episode
- What is being tested?
- L-arginine and L-arginine Loading Dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 108 participants
- Where
- 3 sitesUnited States
- Study dates
- Feb 2016to 21 Feb 2021
- 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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Established diagnosis of sickle cell disease (SCD); all genotypes
- Pain requiring medical care in an acute care setting (such as the emergency department or ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, that is moderate-to-severe requiring parenteral opioids
Who cannot join
- Decision to discharge home from the acute care setting
- Hemoglobin less than 5 gm/dL or immediate need for red cell transfusion anticipated within next 12 hours
- Hepatic dysfunction of SGPT greater than 3 times the upper value
- Renal dysfunction of creatinine greater than 1.0
- Mental status or neurological changes
- Acute stroke or clinical concern for stroke
Show all 18Show fewer
- Pregnancy
- Allergy to arginine
- Two (2) or more ED visits for VOE within the last 7 days prior to CURRENT ED visit
- Hospitalization within 14 days
- Previous randomization in this arginine RCT (patient consented and screen failed before receiving study drug or placebo remains eligible for future participation).
- Use of inhaled nitric oxide, sildenafil or arginine within the last month
- PICU admission from the emergency department
- Hypotension requiring treatment with clinical intervention
- Acidosis with Co2≤ 16
- Newly started on HU for <3 months
- Not an appropriate candidate in the investigator's judgment
- Patient refusal
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Children's Healthcare of Atlanta at Hugh Spalding
Atlanta, Georgia, 30303
Children's Healthcare of Atlanta at Egleston
Atlanta, Georgia, 30322
Children's Healthcare of Atlanta at Scottish Rite
Atlanta, Georgia, 30342
About this study
Official title: Phase 2 Randomized Control Trial of Arginine Therapy for Pediatric Sickle Cell Disease Pain
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores, decrease the need for pain medications or decrease length of hospital stay or emergency department visit. Funding Source - FDA OOPD.
Read the detailed description
The purpose of this study is to determine the effects of IV L-arginine hydrochloride therapy in children with sickle cell disease (SCD) and vaso-occlusive pain events (VOE). Specifically, the impact on total opioid use (mg/kg) over the duration of their emergency department (ED) visit and hospital stay will be evaluated.
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode
- Being tested
- L-arginine, L-arginine Loading Dose, Placebo
- Keywords
- Arginine Therapy
- 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.
L-arginineDrug
Also called Arginine
L-arginine will be dispensed intravenously (IV) in the standard dose of 100 mg/kg three times a day until discharge from the emergency department (ED) or hospital.
L-arginine Loading DoseDrug
Also called Arginine
One loading dose of L-arginine will be dispensed intravenously (IV) at 200 mg/kg
PlaceboOther
Placebo of intravenous (IV) normal saline 1-2 ml/kg three times a day until discharge from the emergency department (ED) or hospital.
| Study group | Role | What they receive |
|---|---|---|
| L-Arginine | Experimental | Participants will be randomized to receive an intravenous (IV) infusion of L-arginine (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital. Receives: Drug: L-arginine |
| Loading Dose and L-Arginine | Experimental | Participants will be randomized to receive an intravenous (IV) infusion of one-time loading dose of L-arginine (200 mg/kg) followed by standard dose (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital. Receives: Drug: L-arginine; Drug: L-arginine Loading Dose |
| Placebo | Placebo comparator | Participants will be randomized to receive an intravenous (IV) infusion of placebo (normal saline 1-2 ml/kg) three times a day until time of discharge from the emergency department (ED) or hospital. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Parenteral Opioid Use in IV Morphine Equivalents
The total amount of parenteral opioids used by participants measured in mg/kg of IV morphine equivalents. The total is calculated after study drug delivery for participants in the emergency department (ED) and during hospital stay.
Measured: Post study drug delivery to discharge from the hospital (Up to 8 days)
Secondary outcomes (16)
Length of Hospital Stay
The total number of hours spent in the hospital from study drug delivery to time of discharge.
Measured: Discharge (Up to 8 days)
Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department
The total number of hours between study drug delivery and the last parenteral opioid.
Measured: Post study drug delivery (Up to 8 hours)
Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital
The total number of hours between study drug delivery and time of last parenteral opioid use, pain relief improved to tolerate oral pain medications
Measured: Post study drug delivery until discharge (up to 8 days)
Change in Vaso-occlusive Pain (VOE) Scores
Pain associated with VOE will be measured on a scale of 0-10, by asking subjects to rate their pain level on a subjective scale from 0 to 10, with the ends representing the extreme limits of "no-pain" (0) and "worst pain" (10).
Measured: Baseline, Time of discharge (Up to 8 days)
Length of Emergency Department (ED) Stay
Total hours from time of ED triage to ED discharge or hospital admission.
Measured: Until discharge or Hospital Admission (Up to 24 hours)
Rate of Emergency Department (ED) Discharge
Number of participants discharged from ED without a hospital ward admission.
Measured: Post emergency department admission (Up to 24 hours)
Total Opioid Dose (ORAL + Parenteral) in mg/kg IV Morphine Equivalents
Total opioid dose (ORAL + Parenteral) in mg/kg IV morphine equivalents after study drug delivery up to hospital discharge (up to 8 days)
Measured: Post study drug delivery up to hospital discharge (Up to 8 days)
Total Number of Study Drug Doses
The total number of study drug doses given throughout the study period.
Measured: Duration of study (Up to 8 days)
Rate of Acute Chest Syndrome
Number of participants who develop acute chest syndrome (not diagnosed prior to study drug delivery) throughout the study period.
Measured: Duration of study (Up to 8 days)
Rate of Blood Transfusion
Number of participants requiring a blood transfusion throughout the study period.
Measured: Duration of study (Up to 8 days)
Oxygen Saturation Level
Average oxygen saturation level of participants at time of ED arrival
Measured: At time of Emergency Department Admission
Oxygen Saturation Level
The difference in oxygen saturation levels from emergency department arrival to hospital discharge.
Measured: At time of hospital admission and at time of Hospital discharge (Up to 8 days)
Rate of Return Visits to Emergency Department (ED) Within 72 Hours
Number of ED visits from patients who have been discharged within the previous 72 hours.
Measured: Post hospital discharge (within 72 hours)
Rate of Hospital Re-admissions Within 72 Hours
Number of patients readmitted to the hospital within 72 hours of discharge.
Measured: Post hospital discharge (within 72 hours)
Rate of Return Visits to Emergency Department (ED) Within 30 Days
Number of ED visits from patients who have been discharged within the previous 30 days.
Measured: Post hospital discharge (within 30 days)
Rate of Hospital Re-admissions With 30 Days
Number of patients readmitted to the hospital within 30 days of discharge.
Measured: Post hospital discharge (within 30 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2015 (estimated)
- Study startFeb 2016
- Primary completion21 Feb 2021
- Study completion21 Feb 2021
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 (5)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 108 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Healthcare of Atlanta, National Center for Complementary and Integrative Health (NCCIH)
- Responsible party
- Principal investigator, Claudia R. Morris, Emory University
- Investigators
- Claudia Morris, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02536170
- Sponsor's protocol ID
- IRB00076988
- Other IDs
- FD-R-004814, 1K24AT009893-01
- Status last verified
- Aug 2023
- First submitted
- 27 Aug 2015
- Last updated
- 6 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Bakshi N, Liu Z, Gillespie S, Keesari R, Leake D, Khemani K, Kumari P, Rees CA, Dampier C, Morris CR. Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode. Blood Adv. 2023 Sep 12;7(17):5103-5107. doi: 10.1182/bloodadvances.2021006794. No abstract available.
PubMed 36322873Reyes LZ, Figueroa J, Leake D, Khemani K, Kumari P, Bakshi N, Lane PA, Dampier C, Morris CR. Safety of intravenous arginine therapy in children with sickle cell disease hospitalized for vaso-occlusive pain: A randomized placebo-controlled trial in progress. Am J Hematol. 2022 Jan 1;97(1):E21-E24. doi: 10.1002/ajh.26396. Epub 2021 Nov 12. No abstract available.
PubMed 34724240Korman R, Yasmine M, Hatabah D, Alzraikat N, Harris F, Brown LA, Chonat S, Bakshi N, Rees CA, Dampier C, Morris CR. Secretory Phospholipase A2 in Patients With Sickle Cell Disease Hospitalized for Vaso-Occlusive Pain Episodes. Pediatr Blood Cancer. 2026 Aug 4:e70615. doi: 10.1002/1545-5017.70615. Online ahead of print.
PubMed 42549972Morris CR, Hatabah D, Korman R, Gillespie S, Bakshi N, Brown LA, Harris F, Leake D, Rees CA, Khemani K, Vichinsky EP, Locke A, Wynn B, Griffiths MA, Wilkinson H, Kumari P, Sudmeier L, Shiva S, Dampier CD. Arginine Therapy for Pain in Sickle Cell Disease: A Phase-2 Randomized, Placebo-Controlled Trial. Am J Hematol. 2025 Jul;100(7):1119-1131. doi: 10.1002/ajh.27692. Epub 2025 Apr 24.
PubMed 40270092Morris CR, Brown LAS, Reynolds M, Dampier CD, Lane PA, Watt A, Kumari P, Harris F, Manoranjithan S, Mendis RD, Figueroa J, Shiva S. Impact of arginine therapy on mitochondrial function in children with sickle cell disease during vaso-occlusive pain. Blood. 2020 Sep 17;136(12):1402-1406. doi: 10.1182/blood.2019003672.
PubMed 32384147
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Randomized Control Trial of Arginine Therapy for Pediatric Sickle Cell Disease Pain
- ClinicalTrials.gov ID
- NCT02536170
- Acronym / sponsor ID
- IRB00076988
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode
- Keywords
- Arginine Therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 108 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Children's Healthcare of Atlanta, National Center for Complementary and Integrative Health (NCCIH)
- Study start
- Feb 2016
- Primary completion
- 21 Feb 2021 (actual)
- Study completion
- 21 Feb 2021 (actual)
- First posted
- 31 Aug 2015 (estimated)
- Results
- Posted 6 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores, decrease the need for pain medications or decrease length of hospital stay or emergency department visit. Funding Source - FDA OOPD.
Detailed description
The purpose of this study is to determine the effects of IV L-arginine hydrochloride therapy in children with sickle cell disease (SCD) and vaso-occlusive pain events (VOE). Specifically, the impact on total opioid use (mg/kg) over the duration of their emergency department (ED) visit and hospital stay will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| L-Arginine | Experimental | Drug: L-arginineParticipants will be randomized to receive an intravenous (IV) infusion of L-arginine (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital. |
| Loading Dose and L-Arginine | Experimental | Drug: L-arginine, Drug: L-arginine Loading DoseParticipants will be randomized to receive an intravenous (IV) infusion of one-time loading dose of L-arginine (200 mg/kg) followed by standard dose (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital. |
| Placebo | Placebo comparator | Other: PlaceboParticipants will be randomized to receive an intravenous (IV) infusion of placebo (normal saline 1-2 ml/kg) three times a day until time of discharge from the emergency department (ED) or hospital. |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Established diagnosis of sickle cell disease (SCD); all genotypes
- Pain requiring medical care in an acute care setting (such as the emergency department or ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, that is moderate-to-severe requiring parenteral opioids
Key exclusion criteria (18 listed)
- Decision to discharge home from the acute care setting
- Hemoglobin less than 5 gm/dL or immediate need for red cell transfusion anticipated within next 12 hours
- Hepatic dysfunction of SGPT greater than 3 times the upper value
- Renal dysfunction of creatinine greater than 1.0
- Mental status or neurological changes
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-arginineAlso: ArginineL-arginine will be dispensed intravenously (IV) in the standard dose of 100 mg/kg three times a day until discharge from the emergency department (ED) or hospital. | Drug | L-Arginine, Loading Dose and L-Arginine |
| L-arginine Loading DoseAlso: ArginineOne loading dose of L-arginine will be dispensed intravenously (IV) at 200 mg/kg | Drug | Loading Dose and L-Arginine |
| PlaceboPlacebo of intravenous (IV) normal saline 1-2 ml/kg three times a day until discharge from the emergency department (ED) or hospital. | Other | Placebo |
Primary outcome
Total Parenteral Opioid Use in IV Morphine Equivalents
Time frame: Post study drug delivery to discharge from the hospital (Up to 8 days)
The total amount of parenteral opioids used by participants measured in mg/kg of IV morphine equivalents. The total is calculated after study drug delivery for participants in the emergency department (ED) and during hospital stay.
16 secondary outcomes
Length of Hospital Stay
Time frame: Discharge (Up to 8 days)
Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department
Time frame: Post study drug delivery (Up to 8 hours)
Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital
Time frame: Post study drug delivery until discharge (up to 8 days)
Change in Vaso-occlusive Pain (VOE) Scores
Time frame: Baseline, Time of discharge (Up to 8 days)
Length of Emergency Department (ED) Stay
Time frame: Until discharge or Hospital Admission (Up to 24 hours)
Rate of Emergency Department (ED) Discharge
Time frame: Post emergency department admission (Up to 24 hours)
Total Opioid Dose (ORAL + Parenteral) in mg/kg IV Morphine Equivalents
Time frame: Post study drug delivery up to hospital discharge (Up to 8 days)
Total Number of Study Drug Doses
Time frame: Duration of study (Up to 8 days)
Rate of Acute Chest Syndrome
Time frame: Duration of study (Up to 8 days)
Rate of Blood Transfusion
Time frame: Duration of study (Up to 8 days)
Oxygen Saturation Level
Time frame: At time of Emergency Department Admission
Oxygen Saturation Level
Time frame: At time of hospital admission and at time of Hospital discharge (Up to 8 days)
Rate of Return Visits to Emergency Department (ED) Within 72 Hours
Time frame: Post hospital discharge (within 72 hours)
Rate of Hospital Re-admissions Within 72 Hours
Time frame: Post hospital discharge (within 72 hours)
Rate of Return Visits to Emergency Department (ED) Within 30 Days
Time frame: Post hospital discharge (within 30 days)
Rate of Hospital Re-admissions With 30 Days
Time frame: Post hospital discharge (within 30 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 36322873Bakshi N, Liu Z, Gillespie S, Keesari R, Leake D, Khemani K, Kumari P, Rees CA, Dampier C, Morris CR. Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode. Blood Adv. 2023 Sep 12;7(17):5103-5107. doi: 10.1182/bloodadvances.2021006794. No abstract available.
- Results publication: PubMed 34724240Reyes LZ, Figueroa J, Leake D, Khemani K, Kumari P, Bakshi N, Lane PA, Dampier C, Morris CR. Safety of intravenous arginine therapy in children with sickle cell disease hospitalized for vaso-occlusive pain: A randomized placebo-controlled trial in progress. Am J Hematol. 2022 Jan 1;97(1):E21-E24. doi: 10.1002/ajh.26396. Epub 2021 Nov 12. No abstract available.
- Derived from this study: PubMed 42549972Korman R, Yasmine M, Hatabah D, Alzraikat N, Harris F, Brown LA, Chonat S, Bakshi N, Rees CA, Dampier C, Morris CR. Secretory Phospholipase A2 in Patients With Sickle Cell Disease Hospitalized for Vaso-Occlusive Pain Episodes. Pediatr Blood Cancer. 2026 Aug 4:e70615. doi: 10.1002/1545-5017.70615. Online ahead of print.
- Derived from this study: PubMed 40270092Morris CR, Hatabah D, Korman R, Gillespie S, Bakshi N, Brown LA, Harris F, Leake D, Rees CA, Khemani K, Vichinsky EP, Locke A, Wynn B, Griffiths MA, Wilkinson H, Kumari P, Sudmeier L, Shiva S, Dampier CD. Arginine Therapy for Pain in Sickle Cell Disease: A Phase-2 Randomized, Placebo-Controlled Trial. Am J Hematol. 2025 Jul;100(7):1119-1131. doi: 10.1002/ajh.27692. Epub 2025 Apr 24.
- Derived from this study: PubMed 32384147Morris CR, Brown LAS, Reynolds M, Dampier CD, Lane PA, Watt A, Kumari P, Harris F, Manoranjithan S, Mendis RD, Figueroa J, Shiva S. Impact of arginine therapy on mitochondrial function in children with sickle cell disease during vaso-occlusive pain. Blood. 2020 Sep 17;136(12):1402-1406. doi: 10.1182/blood.2019003672.
- 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.
Claudia Morris, MD principal investigator
Emory University
3 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 L-arginine and L-arginine Loading Dose in people with Sickle Cell Disease and Vaso-occlusive Pain Episode, run by Emory University at 3 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 2 inclusion and 18 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 6 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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?
Yes. Results are posted in the registry (first posted 6 Jun 2022). 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?
Total Parenteral Opioid Use in IV Morphine Equivalents (time frame: Post study drug delivery to discharge from the hospital (Up to 8 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 NCT02536170, which also lists 5 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 6 Sep 2023.