Ignite Creation Date:
2025-12-24 @ 11:03 PM
Ignite Modification Date:
2025-12-25 @ 8:34 PM
Study NCT ID:
NCT07181369
Status:
RECRUITING
Last Update Posted:
2025-12-04
First Post:
2025-09-12
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Phase 1 Study of GTX-B001 in Healthy Subjects and Patients With Cold Urticaria
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000096703', 'term': 'Cold Urticaria'}, {'id': 'D014581', 'term': 'Urticaria'}, {'id': 'D012871', 'term': 'Skin Diseases'}], 'ancestors': [{'id': 'D017445', 'term': 'Skin Diseases, Vascular'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D006969', 'term': 'Hypersensitivity, Immediate'}, {'id': 'D006967', 'term': 'Hypersensitivity'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077330', 'term': 'Saline Solution'}], 'ancestors': [{'id': 'D000077324', 'term': 'Crystalloid Solutions'}, {'id': 'D007552', 'term': 'Isotonic Solutions'}, {'id': 'D012996', 'term': 'Solutions'}, {'id': 'D004364', 'term': 'Pharmaceutical Preparations'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Single Ascending Dose'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 72}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-10-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-27', 'studyFirstSubmitDate': '2025-09-12', 'studyFirstSubmitQcDate': '2025-09-12', 'lastUpdatePostDateStruct': {'date': '2025-12-04', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-09-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2027-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'GTX-B001 pharmacodynamics (Parts A and B)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Change from baseline in blood biomarkers such as serum tryptase'}, {'measure': 'GTX-B001 pharmacodynamics in skin (Part B)', 'timeFrame': 'Day 1 to Day 29', 'description': 'Change from baseline in mast cell density and mast cell activation markers upon cold provocation, assessed from skin punch biopsy at baseline and three times post-treatment.'}, {'measure': 'Evaluation of immunogenicity (Parts A and B)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Incidence of anti-drug antibodies'}, {'measure': 'Disease control (Part B) - Critical Temperature Threshold (CTT)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Change from baseline and percentage of responders in Critical Temperature Threshold (CTT) over time as determined by provocation testing using the TempTest®. The CTT determines the highest temperature that induces symptoms.'}, {'measure': 'Disease control (Part B) - Urticaria Control Test (UCT)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Change from baseline and percentage of responders for the Urticaria Control Test (UCT). Total score ranges from 0 to 16. A score of 12 or higher indicates well-controlled chronic urticaria, while a score below 12 suggests poorly controlled disease.'}, {'measure': 'Disease control (Part B) - Cold Urticaria Activity Score (Cold UAS)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Change from baseline in Cold Urticaria Activity Score (Cold UAS). The total score ranges from 0 to 6, the higher the score the higher the disease activity.'}, {'measure': 'Disease control (Part B) - Cold Urticaria Quality of Life Questionnaire (ColdU-QoL)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Change from baseline in Cold Urticaria Quality of Life Questionnaire (ColdU-QoL). The total score ranges from 0 to 100, the higher the score the higher the impairment.'}], 'primaryOutcomes': [{'measure': 'Safety as assessed by the incidence and severity of adverse events (Parts A and B)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Safety and tolerability of a single intravenous infusion of GTX-B001 as assessed by the percentage of subjects at each dose level with Grade 2 or higher adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE).'}], 'secondaryOutcomes': [{'measure': 'GTX-B001 pharmacokinetics (Parts A and B)', 'timeFrame': 'Day 1 to Day 85', 'description': 'Serum concentrations of GTX-B001 at specified visits'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Urticaria', 'Skin diseases', 'Mast cells', 'Bispecific antibody', 'GTX-B001', 'Infusion', 'Single Ascending Dose', 'Healthy subjects', 'Safety', 'Pharmacokinetics', 'Pharmacodynamics', 'Cold urticaria', 'Intravenous'], 'conditions': ['Healthy Subjects', 'Cold Urticaria']}, 'descriptionModule': {'briefSummary': 'This is a first-in-human study aimed at determining the safety of a single dose of GTX-B001 in healthy participants and in people with cold urticaria.', 'detailedDescription': 'GTX-B001 is a humanized bispecific antibody than binds with one arm to a protein called c-Kit and with the second arm to a protein called CD203c, both expressed on mast cells.\n\nThis study is a randomized, double-bind, placebo-controlled phase 1 study evaluating the safety, pharmacokinetics and pharmacodynamics of a single dose of GTX-B001 in healthy participants (Part A) and in patients with cold uricaria who remain symptomatic despite treatment with antihistamines (Part B). In part B, the preliminary efficacy of GTX-B001 on the signs and symptoms of cold urticaria will also be evaluated.\n\n48 healthy participants are estimated to be enrolled in part A in five ascending cohorts, while 24 patients with cold urticaria are estimated to be enrolled in Part B in two ascending cohorts.\n\nPotential participants will be screened for up to 4 weeks prior to enrollment. GTX-B001 will be administered intravenously on Day 1 to randomized participants who will be followed for 12 weeks post-treatment.\n\nParticipants will be required to attend a total of 9 visits including the screening visit.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': "Key inclusion Criteria (Part A):\n\n* The subject is between 18 and 55 years of age (both inclusive) at the date of screening.\n* The subject is in good general health based on the medical history, physical examination and the results of vital signs, electrocardiogram and clinical laboratory, as judged by the investigator.\n\nKey inclusion Criteria (Part B):\n\n* The subject is between 18 and 75 years of age (both inclusive) at the date of screening\n* A history of signs and/or symptoms of ColdU experienced ≥ 3 months prior to screening.\n* A positive cold stimulation test during screening and baseline, using the TempTest® device.\n* Inadequate control of ColdU by second generation H1-antihistamine (sgAH) as defined as the presence of itch and hives following cold contact within the last 6 weeks prior to screening in spite of use of a sgAH.\n* A Urticaria Control Test (UCT) score of \\< 12 at screening.\n\nKey exclusion Criteria (Parts A \\& B):\n\n* Presence of any clinically significant illness, such as cardiovascular, neurologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, or endocrine disease or disorder that would interfere with the patient's safety or the interpretation of trial results in the judgment of the investigator.\n* Treatment with an investigational drug or intervention for any condition within three months of screening, or concurrent participation in another clinical trial in which an investigational drug is administered.\n* Treatment with immunomodulating therapy (e.g., intravenous or intramuscular immunoglobulins, systemic corticosteroids, cyclosporine, methotrexate, dapsone, cyclophosphamide, tacrolimus, mycophenolate mofetil, hydroxychloroquine, infliximab, adalimumab, etanercept, etc.) within 4 weeks (or 5 half-lives, whichever is longer) prior to screening.\n* Use of any monoclonal antibody or therapeutic protein within 4 weeks (or 5 half-lives, whichever is longer) prior to screening.\n* History of immunoglobulin E (IgE)-mediated anaphylaxis, autoimmune disorders requiring more than topical medication, or asthma requiring the use of inhaled medication within the past 5 years.\n\nAdditionally for Part B:\n\n* Disease with symptoms of urticaria or angioedema other than Chronic Urticaria (CU), for example urticaria vasculitis, urticaria pigmentosa, erythema multiforme, mastocytosis, hereditary urticaria, or acquired/drug-induced urticaria. Patients with concomitant Chronic Spontaneous Urticaria (CSU) or other forms of Chronic Inducible Urticaria (CIndU) are eligible provided that symptoms at screening are consistent with ColdU and that ColdU is the dominant form of CU.\n* Any other skin disease associated with chronic itching that might influence in the investigators' opinion of the study evaluations and results, e.g., atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, or psoriasis.\n* Receipt of any biologic therapy for urticaria control (e.g. omalizumab, dupilumab, ligelizumab, etc.) within three months of screening (whether on-label or off-label)."}, 'identificationModule': {'nctId': 'NCT07181369', 'acronym': 'CHILL-MC', 'briefTitle': 'A Phase 1 Study of GTX-B001 in Healthy Subjects and Patients With Cold Urticaria', 'organization': {'class': 'INDUSTRY', 'fullName': 'Granular Therapeutics Limited'}, 'officialTitle': 'A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Tolerability, Pharmacokinetic, Pharmacodynamic, and Preliminary Efficacy Trial of GTX-B001 in Healthy Volunteers (Part A) and Patients With Cold Induced Urticaria (Part B)', 'orgStudyIdInfo': {'id': 'GRN-GTXB001-001'}, 'secondaryIdInfos': [{'id': '2025-522126-13-00', 'type': 'CTIS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part A (HV) - GTX-B001', 'description': 'Healthy participants will receive a single dose of GTX-B001', 'interventionNames': ['Drug: GTX-B001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part A (HV) - Normal Saline', 'description': 'Healthy participants assigned to receive placebo will receive a single dose of normal saline', 'interventionNames': ['Drug: Normal Saline']}, {'type': 'EXPERIMENTAL', 'label': 'Part B (ColdU) - GTX-B001', 'description': 'Patients with Cold urticaria will receive a single dose of GTX-B001', 'interventionNames': ['Drug: GTX-B001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part B (ColdU) - Normal Saline', 'description': 'Patients with Cold urticaria assigned to receive placebo will receive a single dose of normal saline', 'interventionNames': ['Drug: Normal Saline']}], 'interventions': [{'name': 'GTX-B001', 'type': 'DRUG', 'otherNames': ['ALY-301'], 'description': 'Single infusion of one of up to five dosages of GTX-B001', 'armGroupLabels': ['Part A (HV) - GTX-B001', 'Part B (ColdU) - GTX-B001']}, {'name': 'Normal Saline', 'type': 'DRUG', 'description': 'Single infusion of normal saline', 'armGroupLabels': ['Part A (HV) - Normal Saline', 'Part B (ColdU) - Normal Saline']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Berlin', 'status': 'RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Principal Investigator', 'role': 'CONTACT', 'email': 'info@charite-research.org', 'phone': '+49 30 450 539 200'}], 'facility': 'Charité Research Organization gmbh', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Berlin', 'status': 'NOT_YET_RECRUITING', 'country': 'Germany', 'contacts': [{'name': 'Principal Investigator', 'role': 'CONTACT', 'email': 'study-coordination-berlin@itmp.fraunhofer.de', 'phone': '+49 30 450 518 219'}], 'facility': 'Fraunhofer Institute For Translational Medicine And Pharmacology', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Granular Therapeutics Limited', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}