[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100506586":3},{"organization":4,"armGroups":7,"interventions":23,"overallOfficials":36,"centralContacts":37,"locations":43,"responsibleParty":94,"collaborators":96,"id":102,"slug":103,"hasResults":104,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":108,"eligibilityCriteria":109,"healthyVolunteers":110,"sex":111,"minAge":112,"maxAge":36,"enrollmentInfo":113,"targetDuration":36,"studyType":116,"phases":117,"briefSummary":120,"conditions":121,"keywords":123,"overallStatus":70,"whyStopped":36,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":133},{"fullName":5,"class":6},"ADARx Pharmaceuticals, Inc.","INDUSTRY",[8,14,19],{"label":9,"type":10,"description":11,"interventionNames":12},"Phase 1 - Active ADX-038 administered to HP","EXPERIMENTAL","For each cohort in Phase 1 (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-038): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.",[13],"Drug: ADX-038",{"label":15,"type":16,"description":11,"interventionNames":17},"Phase 1- Placebo administered to HP","PLACEBO_COMPARATOR",[18],"Drug: Placebo",{"label":20,"type":10,"description":21,"interventionNames":22},"Phase 2a - ADX-038 administered to PNH participants","This will be initiated at the dose level determined by the Safety Review Committee from SAD in HPs. The treatment of PNH participants is an open-label study.",[13],[24,31],{"type":25,"name":26,"description":27,"armGroupLabels":28,"otherNames":29},"DRUG","ADX-038","siRNA duplex oligonucleotide",[9,20],[30],"siRNA",{"type":25,"name":32,"description":33,"armGroupLabels":34,"otherNames":35},"Placebo","Saline",[15],[33],null,[38],{"name":39,"role":40,"phone":41,"phoneExt":36,"email":42},"Stephanie Leyva","CONTACT","877-232-7974","info@adarx.com",[44,58,68,83],{"facility":45,"status":46,"city":47,"state":48,"zip":49,"country":50,"countryCode":51,"cosmosGeoPoint":52,"geoPoint":57,"contacts":36},"Nucleus Network Brisbane","COMPLETED","Brisbane","Queensland","4006","Australia","AU",{"type":53,"coordinates":54},"Point",[55,56],153.02809,-27.46794,{"lat":56,"lon":55},{"facility":59,"status":46,"city":60,"state":61,"zip":62,"country":50,"countryCode":51,"cosmosGeoPoint":63,"geoPoint":67,"contacts":36},"Peter MacCallum Cancer Centre","Melbourne","Victoria","3000",{"type":53,"coordinates":64},[65,66],144.96332,-37.814,{"lat":66,"lon":65},{"facility":69,"status":70,"city":71,"state":61,"zip":72,"country":50,"countryCode":51,"cosmosGeoPoint":73,"geoPoint":77,"contacts":78},"Royal Melbourne Hospital","RECRUITING","Parkville","3052",{"type":53,"coordinates":74},[75,76],144.95,-37.78333,{"lat":76,"lon":75},[79],{"name":80,"role":40,"phone":81,"phoneExt":36,"email":82},"Jeff Szer, MD","+61385595000","PCCTU.HaemA@petermac.org",{"facility":84,"status":46,"city":85,"state":85,"zip":86,"country":87,"countryCode":88,"cosmosGeoPoint":89,"geoPoint":93,"contacts":36},"Richmond Pharmacology Ltd","London","SE1 1YR","United Kingdom","UK",{"type":53,"coordinates":90},[91,92],-0.12574,51.50853,{"lat":92,"lon":91},{"type":95,"investigatorFullName":36,"investigatorTitle":36,"investigatorAffiliation":36,"oldNameTitle":36,"oldOrganization":36},"SPONSOR",[97,100],{"name":98,"class":99},"ADARx Australia Pty Ltd","UNKNOWN",{"name":101,"class":6},"Novotech (Australia) Pty Limited","100506586","phase-1-safety-tolerability-pk-and-pd-of-adx-038-in-healthy-participants-and-paroxysmal-nocturnal-hemoglobinuria-pnh-patients-100506586",false,"NCT05876312","Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients","A Phase 1, Randomized, Double Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Participants Followed by a Phase 2a Open Label Study in Participants With PNH and Residual Anemia to Evaluate the Safety, Tolerability, PK and PD of ADX-038","PNH","Phase 1 Key Inclusion Criteria\n\n* 18 to 55 years of age\n* Participants who are healthy as determined by medical evaluation\n* History of recent meningococcal, pneumococcal and Haemophilus influenzae type B vaccinations or willing to be vaccinated\n* Screening tests negative for illicit drug, nicotine, and alcohol use\n\nPhase 1 Key Exclusion Criteria\n\n* History of any significant medical conditions, except for completely excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia without evidence of recurrence within the prior 3 months\n* Any viral, bacterial, parasitic, or fungal infection within the prior 30 days\n* Frequent respiratory, nasopharyngeal or ear infections (more than 5 infections per year)\n* History of environmental exposure or sick contact that increase the risk of meningococcal, pneumococcal and\u002For Haemophilus influenza type B infections\n* Complement deficiency or immunodeficiency syndrome\n* Major surgery or significant traumatic injury within the prior 3 months\n* History of anaphylaxis or hypersensitivity reactions\n* History of penicillin allergy\n* History of splenectomy\n* History of alcohol abuse or illicit drug use\n* Donated plasma within the prior 7 days\n* Donated blood or loss more than 400 milliliters of blood (excluding blood volume drawn at screening) within the prior 90 days\n* Screening estimated creatinine clearance of less than 60 milliliters per minute\n* Screening hematology, serum chemistry, or coagulation parameters that are outside the normal range\n* Screening vital signs that are abnormal per protocol specification\n* Screening electrocardiogram findings that are clinically significant\n* Pregnant or lactating females\n* Use of prescription (except for contraceptives and study-related prophylactic antibiotics) or over-the counter medications (except for paracetamol or ibuprofen) or vitamins\u002Fsupplements within the prior 7 days\n* Use of medications that may reduce the effectiveness of hormonal contraceptives within the prior 28 days\n* Use of an investigational therapeutics within the prior 30 days or within the expected washout (at least 5 half-lives)\n* Unwilling or unable to adhere to study-related prophylactic antibiotics requirements\n\nPhase 2a Key Inclusion Criteria\n\n* at least 18 years of age\n* Diagnosis of paroxysmal nocturnal hemoglobinuria based on documented clone size\n* Hemoglobin concentration of less than 12 gram per deciliter\n* History of recent meningococcal, pneumococcal and Haemophilus influenzae type b vaccinations or willing to be vaccinated\n* On a stable anti-C5 regimen for greater than or equal to 12 weeks prior to Day 1\n\nPhase 2a Key Exclusion Criteria\n\n* Any viral, bacterial, parasitic, or fungal infection within the prior 14 days\n* HIV, active hepatitis C or hepatitis B infection\n* History of meningococcal or tuberculosis infection\n* History of malignancy in the past 5 years, except for completely excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia with no evidence of recurrence within the prior 3 months\n* Complement deficiency syndrome\n* History of hematopoietic stem cell transplantation\n* History of splenectomy\n* Inflammatory bowel disease, systemic lupus erythematosus, rheumatoid arthritis, or chronic liver disease\n* Clinically significant and uncontrolled medical conditions including, but not limited to, thromboembolic disease, acute coronary syndrome, and diabetes\n* Pregnant or lactating females\n* Use of an investigational therapeutics within the prior 30 days or within the expected washout period (at lest 5 half-lives)\n* Abstain from alcohol consumption for 48 hrs before day of dosing and restrict to no more than an average of 14 standard drinks per week",true,"ALL","18 Years",{"count":114,"type":115},50,"ESTIMATED","INTERVENTIONAL",[118,119],"PHASE1","PHASE2","The first-in-human Phase 1\u002FPhase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).",[122],"Paroxysmal Nocturnal Hemoglobinuria (PNH)",[30,108],"2026-06-12",{"date":126,"type":127},"2026-06-16","ACTUAL",{"date":129,"type":127},"2023-08-07",{"date":131,"type":115},"2028-07-31",{"name":5,"class":6},4]