Sickle Cell DiseasePhase 1NCT02314689
A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
A Phase 1 study testing Intravenous (IV) citrulline in people with Sickle Cell Disease, run by Suvankar Majumdar at 1 site in United States.
Trial summary
The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease. Read more about this study
- Who is it for?
- Ages 18 to 25 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Intravenous (IV) citrulline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2015to Dec 2015
- Phase
- Phase 1
- 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 18 to 25 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+thalassemia, HbSC)
Who cannot join
- Presence of any acute illness defined by fever >100.4° F within the past 48 hours
- Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.
- Presence of acute chest syndrome or presence of any other complication related to sickle cell disease requiring hospitalization such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, and patients with diabetes etc.
- Severe anemia (hemoglobin < 5g/dL)
- History of red blood cell transfusion within the last 14 days
- Systemic steroid therapy within the last 48 hours
Show all 12Show fewer
- Pregnant (as confirmed by a negative urine pregnancy test) or lactating female
- Alanine/aspartate transferase >2x upper limit of normal laboratory range for age.
- Elevated serum creatinine >1.5mg/dL
- Patients with an inability to give consent will be excluded
- Medications that are known to be contra-indicated with use of L-citrulline (concurrent use of hydroxyurea will be allowed).
- History of diabetes due to risk of electrolyte imbalance
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Mississippi Medical Center
Jackson, Mississippi, 39216
About this study
The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease.
- Conditions
- Sickle Cell Disease
- Being tested
- Intravenous (IV) citrulline
- 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.
Intravenous (IV) citrullineDrug
| Study group | Role | What they receive |
|---|---|---|
| IV citrulline | Experimental | IV citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg. Receives: Drug: Intravenous (IV) citrulline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2014 (estimated)
- Study startJan 2015
- Primary completionDec 2015
- Study completionDec 2015
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 (1)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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Suvankar Majumdar
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Suvankar Majumdar, University of Mississippi Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02314689
- Sponsor's protocol ID
- SCD-124700.
- Status last verified
- Jul 2019
- First submitted
- 5 Dec 2014
- Last updated
- 3 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Barr FE, Tirona RG, Taylor MB, Rice G, Arnold J, Cunningham G, Smith HA, Campbell A, Canter JA, Christian KG, Drinkwater DC, Scholl F, Kavanaugh-McHugh A, Summar ML. Pharmacokinetics and safety of intravenously administered citrulline in children undergoing congenital heart surgery: potential therapy for postoperative pulmonary hypertension. J Thorac Cardiovasc Surg. 2007 Aug;134(2):319-26. doi: 10.1016/j.jtcvs.2007.02.043.
PubMed 17662768
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02314689
- Acronym / sponsor ID
- SCD-124700.
- Conditions
- Sickle Cell Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Suvankar MajumdarUniversity, hospital or other
- Study start
- Jan 2015 (actual)
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 11 Dec 2014 (estimated)
- Results
- Posted 7 Mar 2016
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 assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state 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 |
|---|---|---|
| IV citrulline | Experimental | Drug: Intravenous (IV) citrullineIV citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg. |
Who may qualify
- Ages
- 18 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+thalassemia, HbSC)
Key exclusion criteria (12 listed)
- Presence of any acute illness defined by fever >100.4° F within the past 48 hours
- Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.
- Presence of acute chest syndrome or presence of any other complication related to sickle cell disease requiring hospitalization such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, and patients with diabetes etc.
- Severe anemia (hemoglobin < 5g/dL)
- History of red blood cell transfusion within the last 14 days
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 |
|---|---|---|
| Intravenous (IV) citrulline | Drug | IV citrulline |
Primary outcome
Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 17662768Barr FE, Tirona RG, Taylor MB, Rice G, Arnold J, Cunningham G, Smith HA, Campbell A, Canter JA, Christian KG, Drinkwater DC, Scholl F, Kavanaugh-McHugh A, Summar ML. Pharmacokinetics and safety of intravenously administered citrulline in children undergoing congenital heart surgery: potential therapy for postoperative pulmonary hypertension. J Thorac Cardiovasc Surg. 2007 Aug;134(2):319-26. doi: 10.1016/j.jtcvs.2007.02.043.
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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 1 study testing Intravenous (IV) citrulline in people with Sickle Cell Disease, run by Suvankar Majumdar 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 1 inclusion and 12 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 Sep 2019. 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 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.
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 7 Mar 2016). 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?
Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria (time frame: 12 months). 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 NCT02314689, 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 3 Sep 2019.