Ignite Creation Date:
2025-12-24 @ 5:49 PM
Ignite Modification Date:
2026-01-01 @ 8:22 PM
Study NCT ID:
NCT06974968
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-11-25
First Post:
2025-05-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy of iPSC-Derived Motor Neuron Cells (XS228) in Subacute Spinal Cord Injury: A Phase II Randomized Controlled Trial
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013119', 'term': 'Spinal Cord Injuries'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020196', 'term': 'Trauma, Nervous System'}, {'id': 'D014947', 'term': 'Wounds and Injuries'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR'], 'maskingDescription': 'The Phase II study adopts a randomized, double-blind, placebo-controlled design. Investigators and participants remain blinded to treatment allocation. All investigational products and placebos will be identically packaged to maintain blinding for participants, investigators (physicians and coordinators), and monitors. Treatment assignments will remain confidential until database lock and prior to statistical analysis, except in cases of medical emergencies.\n\nDue to the need for cell-based product preparation based on randomization results, an unblinded team will be established to handle drug preparation and packaging. Unblinded team members will not perform any clinical assessments. Further details are provided in the Drug Management Manual.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 60}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2028-05-25', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2031-05-26', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-19', 'studyFirstSubmitDate': '2025-05-08', 'studyFirstSubmitQcDate': '2025-05-08', 'lastUpdatePostDateStruct': {'date': '2025-11-25', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-05-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2031-05-25', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Improvement in ASIA Impairment Scale (AIS) grade', 'timeFrame': 'Improvement in ASIA Impairment Scale (AIS) grade from baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.'}], 'secondaryOutcomes': [{'measure': 'Changes in American Spinal Injury Association (ASIA) Motor Score', 'timeFrame': 'From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.', 'description': 'Changes in motor scores assessed by the ASIA score scale (total score range from 0 to 100, higher values represent a better outcome)'}, {'measure': 'Changes in American Spinal Injury Association (ASIA) Sensory Score', 'timeFrame': 'From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.', 'description': 'Changes in sensory scores assessed by the ASIA score scale (total score range from 0 to 224, higher values represent a better outcome)'}, {'measure': 'Changes in Spinal Cord Independence Measure-III (SCIM-III)', 'timeFrame': 'From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.', 'description': 'SCIM-III(range: 0-100, with lower score indicating greater disability)'}, {'measure': 'Safety and Tolerability', 'timeFrame': 'From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.', 'description': 'Description: Systematic evaluation of:\n\nAdverse events (AEs) up to 360 days post-infusion Immunogenicity (anti-HLA antibodies, T-cell responses)\n\nAssessment Methods:\n\nCTCAE v5.0 grading Neurological monitoring (ISNCSCI exams) CSF analysis for inflammation markers'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Spinal Cord Injury', 'Safety', 'efficacy', 'clinical trial', 'Induced Pluripotent Stem Cells', 'Human motor neuron progenitor', 'Transplantation', 'RCT'], 'conditions': ['Spinal Cord Injury', 'Safety', 'Efficacy', 'Clinical Trial', 'Induced Pluripotent Stem Cells', 'Human Motor Neuron Progenitor', 'Transplantation', 'RCT']}, 'descriptionModule': {'briefSummary': 'Purpose: This clinical trial is studying an investigational cell therapy called XS228-a lab-made stem cell product designed to help repair damaged nerves in the spinal cord. The goal is to see if XS228 is safe and can improve movement, sensation, and function in people with recent spinal cord injuries.\n\nStudy Treatment: XS228 contains specialized nerve-supporting cells derived from human stem cells. These cells are injected into the spinal fluid (intrathecal administration) in a single dose.\n\nWho Can Join? Adults aged 18-65 with a spinal cord injury (thoracic or lumbar level) that occurred 2-12 weeks before enrollment. Participants must have severe but incomplete paralysis (ASIA Impairment Scale Grade A , B or C).\n\nStudy Plan:\n\nPhase II (Main Study): About 60 participants will be randomly assigned to receive either XS228 or a placebo (inactive solution) in a 2:1 ratio.\n\nFollow-up: Patients will be monitored for 1 year, with regular check-ups to assess safety, nerve function, and recovery progress.\n\nWhat Researchers Are Looking For:\n\nPrimary Goal: Measure changes in leg and arm function using the ASIA Motor Score at 6 months.\n\nSecondary Goals:\n\nImprovement in ASIA Impairment Scale (AIS) grade (e.g., from "complete" to "incomplete" paralysis).\n\nRecovery of sensation and bladder/bowel control. Safety (monitoring for side effects like infections or immune reactions). Exploratory Tests: MRI scans and biomarker tests in spinal fluid to see if the treatment helps nerve regrowth.\n\nWhy This Study Matters: If successful, XS228 could become the first stem cell therapy to promote meaningful recovery in spinal cord injury patients. Currently, no treatments exist to repair nerve damage-this trial aims to change that.', 'detailedDescription': 'Title: A Randomized, Double-Blind, Placebo-Controlled Phase II Trial to Evaluate the Efficacy and Safety of XS228 Cell Injection in Patients with Subacute Thoracic or Lumbar SCI\n\nStudy Type: Interventional (Clinical Trial) Phase: Phase II Allocation: Randomized (2:1, XS228 vs. Placebo) Masking: Double-blind (Participant, Investigator) Primary Purpose: Treatment\n\nHypothesis:\n\nXS228 (allogeneic iPSC-derived motor neuron progenitor cells) will demonstrate statistically significant improvement in motor function (ASIA Motor Score) compared to placebo at 6 months post-treatment.\n\nThe therapy will exhibit an acceptable safety profile with no dose-limiting toxicities (DLTs).\n\nIntervention Details\n\nInvestigational Product:\n\nXS228 Cell Injection: Cryopreserved suspension of optimal dose from Phase I. Placebo: Normal saline with identical packaging/labeling. Administration: 4 intrathecal injection via lumbar puncture (Day 1, Day 15, Day 29 and Day 43).\n\nConcomitant Therapies:\n\nStandard rehabilitation protocols (uniform across sites). Prohibited: Other experimental therapies or stem cell treatments during the study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Age: 18 to 65 years (inclusive), regardless of gender.\n\nEtiology: Cervical (C4) to lumbar (L2) spinal cord injury (SCI) caused by traumatic injury or surgery-related factors.\n\nSeverity:\n\nClassified as ASIA Impairment Scale (AIS) Grades A, B, or C. MRI-confirmed evidence of spinal cord injury.\n\nDisease Stage:\n\nPrimary SCI occurring 14 to 60 days prior to screening (subacute phase).\n\nContraception:\n\nParticipants of childbearing potential (male and female) must agree to use effective non-hormonal contraceptive methods during the trial and for 6 months after trial completion.\n\nCompliance:\n\nVoluntarily participate in the clinical study. Ability to understand and comply with study procedures. Participant or legal guardian can provide written informed consent.\n\nExclusion Criteria:\n\n* Neurological Inability\n\nPrimary spinal cord injury (SCI) during screening with concomitant severe traumatic brain injury precluding neurological function assessment.\n\nRespiratory/Circulatory Instability\n\nHigh cervical SCI (C1-C3) causing respiratory/circulatory compromise requiring endotracheal intubation or tracheostomy.\n\nLife-Threatening Multiorgan Dysfunction\n\nConcurrent severe injuries to other organ systems with life-threatening dysfunction.\n\nUnstable Thoracoabdominal Injuries\n\nInjuries to lungs, liver, kidneys, spleen, etc., deemed unstable by the investigator.\n\nPrior Spinal Pathology\n\nHistory of SCI or coexisting spinal disorders (e.g., ankylosing spondylitis, spinal deformities, primary/metastatic spinal tumors, spinal vascular malformations, syringomyelia).\n\nLocal Infection/Increased ICP\n\nActive infection at the lumbar puncture site or intracranial hypertension during screening.\n\nSevere Infections\n\nSepsis, septic shock, or severe pneumonia (per IDSA/ATS 2007 diagnostic criteria).\n\nConfounding Neurological/Psychiatric Conditions\n\nParkinson's disease, severe dementia, myasthenia gravis, stroke, Guillain-Barré syndrome, diabetic neuropathy, or other conditions interfering with study assessments.\n\nCardiac Abnormalities (any of the following):\n\nCongestive heart failure (NYHA Class III/IV). Severe uncontrolled arrhythmias (e.g., sick sinus syndrome, third-degree AV block).\n\nUnstable angina or acute myocardial infarction within 3 months prior. Pulmonary Complications\n\nPulmonary hypertension, pulmonary embolism, or suspected embolism during screening.\n\nUncontrolled Hypertension/Hypotension\n\nSystolic BP \\>160 mmHg or diastolic BP \\>100 mmHg; or systolic BP \\<90 mmHg or diastolic BP \\<60 mmHg.\n\nActive Autoimmune Diseases\n\nRequiring immunosuppressants (e.g., uncontrolled hyperthyroidism, systemic lupus erythematosus).\n\nImmunosuppressant Non-Compliance\n\nUnwillingness or inability to use immunosuppressants per protocol.\n\nLaboratory Abnormalities (any of the following):\n\nALT/AST \\>2×ULN or total bilirubin \\>2×ULN. eGFR \\<60 mL/min/1.73m² (CKD-EPI 2021 formula). APTT/PT \\>2.5×ULN (without anticoagulants). Platelets \\<100×10⁹/L or hemoglobin \\<90 g/L. Allergy\n\nHistory of severe allergies or hypersensitivity to trial drug/excipients (human albumin, lactated Ringer's solution).\n\nInfectious Diseases\n\nHBsAg+ with HBV DNA \\>1000 IU/mL; HCV-Ab+; HIV-Ab+; or TP-Ab+. Lumbar Puncture Refusal\n\nUnwillingness to undergo intrathecal administration procedures. Pregnancy/Lactation\n\nFemales who are pregnant or breastfeeding. Malignancy\n\nActive malignancy or anticancer therapy within 5 years prior. Recent Clinical Trial Participation\n\nEnrollment in another drug trial within 3 months prior. Investigator Discretion\n\nAny condition deemed unsuitable for participation by the investigator"}, 'identificationModule': {'nctId': 'NCT06974968', 'briefTitle': 'Efficacy of iPSC-Derived Motor Neuron Cells (XS228) in Subacute Spinal Cord Injury: A Phase II Randomized Controlled Trial', 'organization': {'class': 'INDUSTRY', 'fullName': 'XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.'}, 'officialTitle': 'A Phase II Clinical Study Evaluating the Preliminary Efficacy of Human Allogeneic Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells (XS228 Cell Injection) in Patients With Subacute Spinal Cord Injury', 'orgStudyIdInfo': {'id': 'XS228-Allo-SCI-CN2-P01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'XS228 Interventional Group', 'description': 'Investigational Product:\n\nXS228 Cell Injection: Cryopreserved suspension of optimal dose from Phase I. Placebo: Normal saline with identical packaging/labeling. Administration: 4 intrathecal injection via lumbar puncture (Day 1, Day 15, Day 29, Day 43).', 'interventionNames': ['Biological: Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo Control Group', 'description': 'Standard rehabilitation protocols', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells', 'type': 'BIOLOGICAL', 'description': 'Description:\n\nXS228 Cell Injection is an investigational, allogeneic cell therapy product composed of motor neuron progenitor cells (MNPCs) derived from human induced pluripotent stem cells (iPSCs). This advanced therapy medicinal product (ATMP) is being developed for the treatment of spinal cord injury (SCI) and represents a novel approach in regenerative medicine.', 'armGroupLabels': ['XS228 Interventional Group']}, {'name': 'Placebo', 'type': 'OTHER', 'otherNames': ['Standard rehabilitation protocols'], 'description': 'Standard rehabilitation protocols', 'armGroupLabels': ['Placebo Control Group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '510630', 'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'contacts': [{'name': 'Limin Rong', 'role': 'CONTACT', 'email': 'ronglm@mail.sysu.edu.cn', 'phone': '02085253333'}, {'name': 'Limin Rong, prof and M.D', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'The Third Affiliated Hospital of Sun Yat-sen University', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}], 'centralContacts': [{'name': 'Michael LEE', 'role': 'CONTACT', 'email': 'CEO@xellsmart.com', 'phone': '+86 21 64027719'}, {'name': 'Mao Pang, prof and M.D', 'role': 'CONTACT', 'email': 'pangmao6@mail.sysu.edu.cn', 'phone': '+86-020-85252015'}], 'overallOfficials': [{'name': 'Limin Rong, prof and M.D', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Third Affiliated Hospital, Sun Yat-Sen University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'At this stage, the sponsor has not yet finalized a decision regarding the sharing of individual participant data (IPD). The potential risks and benefits of data sharing, including patient privacy, regulatory requirements, and intellectual property considerations, are still under evaluation. A final determination will be made following further internal review and alignment with applicable data protection laws, institutional policies, and ethical guidelines. If IPD sharing is approved in the future, details regarding data accessibility, anonymization methods, and request procedures will be specified in an updated plan.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Third Affiliated Hospital, Sun Yat-Sen University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}