Ignite Creation Date:
2025-12-25 @ 4:02 AM
Ignite Modification Date:
2025-12-26 @ 2:55 AM
Study NCT ID:
NCT06635902
Status:
RECRUITING
Last Update Posted:
2025-04-17
First Post:
2024-10-07
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D002956', 'term': 'Citrulline'}], 'ancestors': [{'id': 'D000599', 'term': 'Amino Acids, Diamino'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 99}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-12-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2029-03', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-04-14', 'studyFirstSubmitDate': '2024-10-07', 'studyFirstSubmitQcDate': '2024-10-09', 'lastUpdatePostDateStruct': {'date': '2025-04-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-10-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Citrulline and arginine bioavailability', 'timeFrame': 'Baseline to 30 days', 'description': 'Change in baseline citrulline and arginine concentration during hospitalization and 30 day follow up/'}, {'measure': 'Nitric oxide', 'timeFrame': 'Baseline to 30 days', 'description': 'Change in baseline nitric oxide concentration during hospitalization and 30 day follow up'}, {'measure': 'Cytokine interleukin IL-1ß', 'timeFrame': 'Baseline to 30 days', 'description': 'Change in baseline plasmatic levels of IL-1ß concentration during hospitalization and 30 day follow up'}, {'measure': 'Mitochondrial function', 'timeFrame': 'Baseline to 30 days', 'description': 'Mitochondrial respiratory complex activities will be measured to estimate mitochondrial function. Change in baseline mitochondrial respiratory complex activity during hospitalization and 30 day follow up'}, {'measure': 'Genetic', 'timeFrame': 'Baseline', 'description': 'DNA to test specific genetic polymorphisms related to nitric oxide pathway'}], 'primaryOutcomes': [{'measure': 'Time-to-crisis resolution', 'timeFrame': 'Baseline to 30 days', 'description': 'Change in time-to-crisis resolution as defined by the time (in hours) from first dose of intravenous study drug or placebo to the time of last dose of intravenous opioid during hospitalization'}], 'secondaryOutcomes': [{'measure': 'Safety', 'timeFrame': 'Baseline to 30 days', 'description': 'Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0'}, {'measure': 'Opioid consumption', 'timeFrame': 'Baseline to 30 days', 'description': 'Change in cumulative opioid consumption (calculated in morphine equivalents) from time of study drug administration to last dose of IV/oral opioid during hospitalization'}, {'measure': 'Pain scores', 'timeFrame': 'Baseline to 30 days', 'description': 'Change in baseline pain scores will be recorded from a 0-10 scale, 10 is worst pain'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['sickle cell disease', 'sickle cell pain crisis', 'citrulline'], 'conditions': ['Sickle Cell Disease', 'Vaso-occlusive Pain Episode']}, 'descriptionModule': {'briefSummary': 'The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline. The main questions it aims to answer are:\n\n* Does intravenous citrulline decrease the duration of sickle cell pain during hospitalization\n* What medical problems do participants have when taking intravenous citrulline? Researchers will compare intravenous citrulline to a placebo (a look-alike substance that contains no drug) to see if intravenous citrulline works to treat acute pain.\n\nParticipants will:\n\n* Receive baseline tests and intravenous citrulline for 16 hours during the hospital stay\n* After hospital discharge, visit the clinic in about 30 days for checkup and tests', 'detailedDescription': 'This is a single-center phase 2 randomized double-blind, placebo-controlled trial of intravenous L-citrulline for sickle cell patients ages 4 to 21 years experiencing a vaso-occlusive pain crisis episode (VOE). Eligible subjects will have a documented history of sickle cell disease and inpatient hospitalization for treatment of acute pain with parenteral opioid. Subjects will be randomized to receive high dose intravenous L-citrulline, low dose intravenous L-citrulline or placebo in addition to standard of care. Subjects will be followed closely to evaluate time-to-crisis resolution as the primary outcome defined by time from first dose of intravenous study drug/placebo to the last dose of parenteral opioid prior to hospital discharge. Participants will be monitored for any adverse events including 30-day re-hospitalization rates. Total opioid consumption during the time-to-crisis resolution will be compared between the three arms. In addition, exploratory outcomes will be evaluated for pain score, tissue blood flow, genetic and candidate biomarkers related to vaso-occlusion.\n\nObjectives Primary Objective\n\n• To demonstrate the efficacy of intravenous L-citrulline in reducing the time-to-crisis resolution in sickle cell subjects experiencing a vaso-occlusive pain crisis episode (VOE).\n\nSecondary Objectives\n\n* To evaluate the safety of intravenous L-citrulline in the treatment of VOE\n* To determine if intravenous L-citrulline improves cumulative opioid consumption during the treatment of VOE Exploratory Objectives\n* To determine if intravenous L-citrulline improves pain scores during the hospitalization\n* To determine if intravenous L-citrulline improves 30-day re-hospitalization rates\n* To determine the pharmacokinetic (PK) profile of intravenous L-citrulline\n* To evaluate whether intravenous L-citrulline improves tissue blood flow and candidate biomarkers related to vaso-occlusion.\n* To assess whether genetic single nucleotide polymorphisms related to the nitric oxide pathway influence study outcomes'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Sickle cell disease (all genotypes)\n* Patients with sickle cell disease ages 4 to 21 years old\n* Presence of sickle cell vaso-occlusive pain episode requiring hospitalization and parenteral opioid therapy\n* Able to randomize to study drug/placebo within 12 hours of first dose of parenteral opioid in the Emergency Department\n\nExclusion Criteria:\n\n* Current pain lasting \\>3 days.\n* \\>9 hospitalizations in the prior year\n* Presence of any other complication related to sickle cell disease requiring the hospitalization such as splenic sequestration, hepatic sequestration, stroke, transient ischemic attack, etc.\n* History of opioid use disorder, chronic pain or medical regimen requiring daily opioid use.\n* Severe anemia (hemoglobin \\<6g/dL)\n* Pregnant (as confirmed by a positive urine pregnancy test) or lactating female.\n* Alanine/aspartate transferase \\>2x upper limit of normal laboratory range for age.\n* Subject has the following serum creatinine:\n\n * Age 4 to 13 years \\> 0.9 mg/dL\n * Age 14 to 17 years 1.0 mg/dL\n * Age ≥18 years \\>1.5mg/dL\n* Presence of acute chest syndrome, sepsis, bacterial infection, hemodynamic instability\n* Use of L-glutamine\n* History of allergic reaction to L-citrulline products'}, 'identificationModule': {'nctId': 'NCT06635902', 'briefTitle': 'Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease', 'organization': {'class': 'OTHER', 'fullName': "Children's National Research Institute"}, 'officialTitle': 'A Phase 2 Randomized Double-blind, Placebo-controlled Clinical Trial of Intravenous Citrulline for Vaso-occlusive Pain Episode in Hospitalized Patients With Sickle Cell Disease (CONQUER SCD Pain Trial)', 'orgStudyIdInfo': {'id': '00000880'}, 'secondaryIdInfos': [{'id': '1R61HL167806', 'link': 'https://reporter.nih.gov/quickSearch/1R61HL167806', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Low dose intravenous L-citrulline', 'interventionNames': ['Drug: L-citrulline']}, {'type': 'EXPERIMENTAL', 'label': 'High dose intravenous L-citrulline', 'interventionNames': ['Drug: L-citrulline']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'L-citrulline', 'type': 'DRUG', 'description': 'Intravenous L-citrulline (50 mg/kg + 9mg/kg/hr.) for 16 hours', 'armGroupLabels': ['High dose intravenous L-citrulline']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Isotonic normal saline', 'armGroupLabels': ['Placebo']}, {'name': 'L-citrulline', 'type': 'DRUG', 'description': 'Intravenous L-citrulline (25 mg/kg + 9mg/kg/hr.) for 16 hours', 'armGroupLabels': ['Low dose intravenous L-citrulline']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jillian Baker, RN', 'role': 'CONTACT', 'email': 'jbaker5@childrensnational.org', 'phone': '202-476-1311'}, {'name': 'Suvankar Majumdar, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Children's National Hospital", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}], 'centralContacts': [{'name': 'Jillian Baker, RN', 'role': 'CONTACT', 'email': 'jbaker5@childrensnational.org', 'phone': '202-476-1311'}], 'overallOfficials': [{'name': 'Suvankar Majumdar, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's National Research Institute"}]}, 'ipdSharingStatementModule': {'infoTypes': ['ICF'], 'timeFrame': 'Beginning 3 months and ending 3 years after the publication of results', 'ipdSharing': 'YES', 'description': 'All IPD that underlie results in a publication will be shared', 'accessCriteria': 'Access to trial IPD can be requested by qualified researchers engaging in independent scientific research, and will be provided following review and approval of a research proposal and Statistical Analysis Plan (SAP). For more information or to submit a request, please contact smajumdar@childrensnational.org'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Suvankar Majumdar', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Chief of Hematology', 'investigatorFullName': 'Suvankar Majumdar', 'investigatorAffiliation': "Children's National Research Institute"}}}}