Sickle Cell DiseasePhase 1NCT00343954
PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
A Phase 1 study testing L-citrulline tablets, 1000 mg in people with Sickle Cell Disease, run by Angiogenix at 1 site in United States.
Trial summary
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- L-citrulline tablets, 1000 mg
- Type of treatment
- Pill or oral medicine
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Phase 1
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female, 10 years of age or older on the day of dosing, and generally healthy as determined by medical history, physical examination, and laboratory test values
- Diagnosis of sickle cell anemia (Hb SS)
- For female of child-bearing potential, a negative serum pregnancy test and using an adequate method of contraception
- Has signed and received a copy of the written informed consent form approved by the investigator's Institutional Review Board (IRB), understands the purposes and risks of the study and agrees to follow the restrictions and schedule of procedures as defined by this protocol
Who cannot join
- History of sickle-cell-related pain crisis within two weeks of study
- Pregnant or breast feeding
- Transfusion within last 90 days
- Creatinine >1.5 X upper limit of normal
- SGPT > 2 X upper limit of normal
- History of allergic reaction to arginine or citrulline product
Show all 12Show fewer
- Requires chronic medication other than study drug that cannot be discontinued during the study period
- Unable to take or tolerate oral medications
- Unreliable venous access
- Noncompliant with regular care
- Participation in an investigational drug or medical device study within previous 30 days
- In the opinion of the investigator is not a good candidate for participation in the study
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System
Pittsburgh, Pennsylvania, 15213
Lakshmanan Krishnamurti, MD, 412-692-7192
Sidney Morris, PhD, (412) 648-9338
About this study
Official title: Phase 1 Pharmacokinetic and Pharmacodynamic Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.
Read the detailed description
Phase 1, single center, open label, multiple-dose study consisting of a screening period followed by four weeks of treatment. At least 12 male and female patients, 10 years of age or older, with sickle cell disease but otherwise healthy, will be enrolled in the study.
PK measurements include plasma concentrations of L-citrulline, L-arginine, L-ornithine, and L-proline following the first dose of orally administered L-citrulline and after four weeks of twice daily administration of the drug.
PD measurements include intercellular and vascular adhesion molecules (ICAM, VCAM, and E-selectin), surrogate markers of sickle cell disease activity, and PAT, a measurement of vascular function.
- Conditions
- Sickle Cell Disease
- Being tested
- L-citrulline tablets, 1000 mg
- Keywords
- sickle cell disease
- 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-citrulline tablets, 1000 mgDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
PK and PD responses to L-citrulline
Endpoints will be determined as change from baseline
Secondary outcomes (1)
Assessment of safety and tolerability of L-citrulline,
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2006 (estimated)
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.
- 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants
Sponsor and investigators
- Lead sponsor
- Angiogenix
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Lakshmanam Krishnamurti, MD, Principal investigator, Children's Hospital of Pittsburgh, Univ. of Pittsburgh Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00343954
- Sponsor's protocol ID
- ANGX-3227-01
- Status last verified
- Jun 2006
- First submitted
- 21 Jun 2006
- Last updated
- 23 Jun 2006
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Pharmacokinetic and Pharmacodynamic Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00343954
- Acronym / sponsor ID
- ANGX-3227-01
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants
- Lead sponsor
- AngiogenixIndustry (pharma, biotech, devices)
- First posted
- 23 Jun 2006 (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.
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.
Detailed description
Phase 1, single center, open label, multiple-dose study consisting of a screening period followed by four weeks of treatment. At least 12 male and female patients, 10 years of age or older, with sickle cell disease but otherwise healthy, will be enrolled in the study.
PK measurements include plasma concentrations of L-citrulline, L-arginine, L-ornithine, and L-proline following the first dose of orally administered L-citrulline and after four weeks of twice daily administration of the drug.
PD measurements include intercellular and vascular adhesion molecules (ICAM, VCAM, and E-selectin), surrogate markers of sickle cell disease activity, and PAT, a measurement of vascular function.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female, 10 years of age or older on the day of dosing, and generally healthy as determined by medical history, physical examination, and laboratory test values
- Diagnosis of sickle cell anemia (Hb SS)
- For female of child-bearing potential, a negative serum pregnancy test and using an adequate method of contraception
- Has signed and received a copy of the written informed consent form approved by the investigator's Institutional Review Board (IRB), understands the purposes and risks of the study and agrees to follow the restrictions and schedule of procedures as defined by this protocol
Key exclusion criteria (12 listed)
- History of sickle-cell-related pain crisis within two weeks of study
- Pregnant or breast feeding
- Transfusion within last 90 days
- Creatinine >1.5 X upper limit of normal
- SGPT > 2 X upper limit of normal
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-citrulline tablets, 1000 mg | Drug | – |
Primary outcome
PK and PD responses to L-citrulline
Endpoints will be determined as change from baseline
1 secondary outcome
Assessment of safety and tolerability of L-citrulline,
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2006.
Related trials for Sickle Cell Disease
RecruitingObservational study
Blood Sampling for Research Related to Sickle Cell Disease
Sickle Cell Trait, Sickle Cell Disease
Active, not recruitingPhase 2/3
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Sickle Cell Disease
Enrolling by invitationObservational study
Blood Collection for Research Related to Certain Diseases Involving Blood Vessels
Sickle Cell Disease
RecruitingPhase 1/2
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Myeloid Chimerism
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
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.
Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System
Pittsburgh, United States
Lakshmanam Krishnamurti, MD principal investigator
Children's Hospital of Pittsburgh, Univ. of Pittsburgh Medical Center
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 L-citrulline tablets, 1000 mg in people with Sickle Cell Disease, run by Angiogenix 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 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: status not confirmed, as of the sponsor's last update on 23 Jun 2006. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PK and PD responses to L-citrulline. The study lists 2 primary outcomes in all. 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 23 Jun 2006.