Ignite Creation Date:
2025-12-24 @ 11:57 PM
Ignite Modification Date:
2026-01-03 @ 6:18 AM
Study NCT ID:
NCT04152551
Status:
RECRUITING
Last Update Posted:
2025-12-15
First Post:
2019-10-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Effects of Bisphosphonates on OI-Related Hearing Loss
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010013', 'term': 'Osteogenesis Imperfecta'}, {'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'D009085', 'term': 'Mucopolysaccharidosis IV'}, {'id': 'C565842', 'term': 'Brittle Bone Disorder'}], 'ancestors': [{'id': 'D010009', 'term': 'Osteochondrodysplasias'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D003095', 'term': 'Collagen Diseases'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D006311', 'term': 'Hearing Disorders'}, {'id': 'D004427', 'term': 'Ear Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D012678', 'term': 'Sensation Disorders'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000068296', 'term': 'Risedronic Acid'}], 'ancestors': [{'id': 'D004164', 'term': 'Diphosphonates'}, {'id': 'D063065', 'term': 'Organophosphonates'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D011725', 'term': 'Pyridines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': '25 adults with type 1 OI and diagnosed hearing loss will be treated with oral risedronate.\n\n25 adults with type 1 OI will be enrolled into the control arm.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2019-11-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2026-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-09', 'studyFirstSubmitDate': '2019-10-18', 'studyFirstSubmitQcDate': '2019-11-01', 'lastUpdatePostDateStruct': {'date': '2025-12-15', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2019-11-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Pure Tone Averages', 'timeFrame': 'Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months', 'description': 'Average hearing thresholds at 250, 500, 1000, 2000, 3000, 4000, 8000 Hertz'}], 'secondaryOutcomes': [{'measure': 'Speech Recognition Scores', 'timeFrame': 'Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months', 'description': 'Lowest volume participant can hear and understand speech (decibels)'}, {'measure': 'Word Recognition Scores', 'timeFrame': 'Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months', 'description': 'Percent of words participants correctly repeat in word recognition test (%)'}, {'measure': 'Hearing Handicap Inventory Raw Score', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Score 0-40. Lower score is better. Adults (self-reported)'}, {'measure': 'Tinnitus Handicap Inventory Score', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Score 0-100. Lower score is better. Adults (self-reported).'}, {'measure': 'Dizziness Handicap Inventory Score', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Score 0-100. Lower score is better. Adults (self-reported). Incidence and impact of vertigo in study population.'}, {'measure': 'SF-36 Scale and Summary Scores', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Score 0-100. Higher score is better. Adults (self-reported) quality-of-life survey.'}, {'measure': 'Pediatric Outcomes Data Collection Instrument (PODCI) Score', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Score 0-100. Lower score is better. Children (ages 6-10 years), parent-reported. Assessment of overall health and functioning.'}, {'measure': 'Adolescent Outcomes Questionnaire Score', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Score 0-100. Lower score is better. Children (ages 11-17 years), parent- or self-reported. Assessment of overall health and functioning.'}, {'measure': 'DEXA Z-score', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Higher score is better. Relative Bone Density'}, {'measure': 'DEXA Bone Mineral Density', 'timeFrame': 'Yearly (Baseline, 12, 24, 36, 48, 60 months)', 'description': 'Higher score is better. Bone Mineral Density (grams/centimeter\\^2)'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['hearing loss', 'bisphosphonate', 'skeletal dysplasia', 'brittle bone disorder', 'osteogenesis imperfecta', 'OI'], 'conditions': ['Osteogenesis Imperfecta']}, 'referencesModule': {'references': [{'pmid': '24715559', 'type': 'BACKGROUND', 'citation': 'Van Dijk FS, Sillence DO. Osteogenesis imperfecta: clinical diagnosis, nomenclature and severity assessment. Am J Med Genet A. 2014 Jun;164A(6):1470-81. doi: 10.1002/ajmg.a.36545. Epub 2014 Apr 8.'}, {'pmid': '22567374', 'type': 'BACKGROUND', 'citation': 'Pillion JP, Vernick D, Shapiro J. Hearing loss in osteogenesis imperfecta: characteristics and treatment considerations. Genet Res Int. 2011;2011:983942. doi: 10.4061/2011/983942. Epub 2011 Dec 14.'}, {'pmid': '7285446', 'type': 'BACKGROUND', 'citation': 'Sillence D. Osteogenesis imperfecta: an expanding panorama of variants. Clin Orthop Relat Res. 1981 Sep;(159):11-25.'}, {'pmid': '19358256', 'type': 'BACKGROUND', 'citation': 'Swinnen FK, De Leenheer EM, Coucke PJ, Cremers CW, Dhooge IJ. Audiometric, surgical, and genetic findings in 15 ears of patients with osteogenesis imperfecta. Laryngoscope. 2009 Jun;119(6):1171-9. doi: 10.1002/lary.20155.'}, {'pmid': '3918222', 'type': 'BACKGROUND', 'citation': 'Berger G, Hawke M, Johnson A, Proops D. Histopathology of the temporal bone in osteogenesis imperfecta congenita: a report of 5 cases. Laryngoscope. 1985 Feb;95(2):193-9. doi: 10.1288/00005537-198502000-00014.'}, {'pmid': '24751750', 'type': 'BACKGROUND', 'citation': 'Vincent R, Wegner I, Stegeman I, Grolman W. Stapedotomy in osteogenesis imperfecta: a prospective study of 32 consecutive cases. Otol Neurotol. 2014 Dec;35(10):1785-9. doi: 10.1097/MAO.0000000000000372.'}, {'pmid': '10562298', 'type': 'BACKGROUND', 'citation': 'Plotkin LI, Weinstein RS, Parfitt AM, Roberson PK, Manolagas SC, Bellido T. Prevention of osteocyte and osteoblast apoptosis by bisphosphonates and calcitonin. J Clin Invest. 1999 Nov;104(10):1363-74. doi: 10.1172/JCI6800.'}, {'pmid': '10321934', 'type': 'BACKGROUND', 'citation': 'Rogers MJ, Frith JC, Luckman SP, Coxon FP, Benford HL, Monkkonen J, Auriola S, Chilton KM, Russell RG. Molecular mechanisms of action of bisphosphonates. Bone. 1999 May;24(5 Suppl):73S-79S. doi: 10.1016/s8756-3282(99)00070-8. No abstract available.'}, {'pmid': '18775204', 'type': 'BACKGROUND', 'citation': 'Drake MT, Clarke BL, Khosla S. Bisphosphonates: mechanism of action and role in clinical practice. Mayo Clin Proc. 2008 Sep;83(9):1032-45. doi: 10.4065/83.9.1032.'}, {'pmid': '16564235', 'type': 'BACKGROUND', 'citation': 'Kanzaki S, Ito M, Takada Y, Ogawa K, Matsuo K. Resorption of auditory ossicles and hearing loss in mice lacking osteoprotegerin. Bone. 2006 Aug;39(2):414-9. doi: 10.1016/j.bone.2006.01.155. Epub 2006 Mar 24.'}, {'pmid': '22935809', 'type': 'BACKGROUND', 'citation': 'Quesnel AM, Seton M, Merchant SN, Halpin C, McKenna MJ. Third-generation bisphosphonates for treatment of sensorineural hearing loss in otosclerosis. Otol Neurotol. 2012 Oct;33(8):1308-14. doi: 10.1097/MAO.0b013e318268d1b3.'}, {'pmid': '27153329', 'type': 'BACKGROUND', 'citation': 'Kang WS, Nguyen K, McKenna CE, Sewell WF, McKenna MJ, Jung DH. Measurement of Ototoxicity Following Intracochlear Bisphosphonate Delivery. Otol Neurotol. 2016 Jul;37(6):621-6. doi: 10.1097/MAO.0000000000001042.'}, {'pmid': '22708940', 'type': 'BACKGROUND', 'citation': 'Ting TH, Zacharin MR. Hearing in bisphosphonate-treated children with osteogenesis imperfecta: our experience in thirty six young patients. Clin Otolaryngol. 2012 Jun;37(3):229-33. doi: 10.1111/j.1749-4486.2012.02476.x. No abstract available.'}, {'pmid': '24754836', 'type': 'BACKGROUND', 'citation': 'Patel RM, Nagamani SC, Cuthbertson D, Campeau PM, Krischer JP, Shapiro JR, Steiner RD, Smith PA, Bober MB, Byers PH, Pepin M, Durigova M, Glorieux FH, Rauch F, Lee BH, Hart T, Sutton VR. A cross-sectional multicenter study of osteogenesis imperfecta in North America - results from the linked clinical research centers. Clin Genet. 2015 Feb;87(2):133-40. doi: 10.1111/cge.12409. Epub 2014 May 30.'}]}, 'descriptionModule': {'briefSummary': 'Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in their 20s. Bisphosphonates have been successfully used to treat otosclerosis, a common cause of hearing loss similar to OI-related hearing loss. As many individuals with OI-related hearing loss also present with otosclerosis and because of their mechanistic similarities, the investigators propose studying the effects of bisphosphonate treatment on individuals diagnosed with both OI type I and hearing loss, thereby determining its effectiveness as a potential treatment for hearing loss.\n\nThe investigators will enroll 50 individuals diagnosed with type I OI and age 18-100. 25 adults will be enrolled into the treatment arm and receive bisphosphonate treatment (must have at least mild hearing loss), while 25 adults will be enrolled into the control arm. The investigators will enroll 25 children (6-17 years of age) diagnosed with OI who are currently receiving bisphosphonate treatment as part of their care for orthopedic symptoms. The investigators will also observe 25 children (6-17 years of age) diagnosed with OI who are NOT currently receiving bisphosphonate treatment. The study duration is 63 months (approximately 5 years). Enrollment is anticipated to begin in November 2019.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '100 Years', 'minimumAge': '6 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria (Adult Treatment Arm):\n\n* Diagnosis of OI type I\n* Diagnosis of at least mild hearing loss (\\>20dB pure tone average) by audiogram testing\n* 18+\n* Vitamin D level \\> 30\n\nInclusion Criteria (Adult Control Arm):\n\n* Diagnosis of OI type I\n\nInclusion Criteria (Child Observational Bisphosphonate Arm)\n\n* Diagnosis of OI\n* Age 6-17 years\n* Currently receiving bisphosphonate treatment as standard of care\n\nInclusion Criteria (Child Observational No Treatment Arm)\n\n* Diagnosis of OI\n* Age 6-17 years\n* NOT receiving bisphosphonate treatment and will not receive bisphosphonate treatment for the duration of the study\n\nExclusion Criteria (ALL ARMS):\n\n* Family history of hearing-loss (not related to OI or occupational hearing loss)\n* Pregnancy'}, 'identificationModule': {'nctId': 'NCT04152551', 'briefTitle': 'Effects of Bisphosphonates on OI-Related Hearing Loss', 'organization': {'class': 'OTHER', 'fullName': 'Hospital for Special Surgery, New York'}, 'officialTitle': 'Effects of Bisphosphonates on OI-Related Hearing Loss: A Pilot Study', 'orgStudyIdInfo': {'id': '#2018-0700'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Adult Treatment Arm', 'description': 'Intervention treatment arm. Adults (18+ years) with type 1 OI. Must have at least mild hearing loss. Will receive Risedronate (35mg, 0-2x/week as clinically indicated) for duration of study. Changes in hearing, quality of life, and bone density will be monitored.', 'interventionNames': ['Drug: Risedronate Oral Tablet']}, {'type': 'NO_INTERVENTION', 'label': 'Child (Bisphosphonate Arm)', 'description': 'Observational (no investigational intervention) arm. Children (6-17 years) with any type of OI who are already receiving bisphosphonate treatment as standard of care treatment for orthopedic symptoms. Changes in hearing, quality of life, and bone density will be observed for the duration of the study.'}, {'type': 'NO_INTERVENTION', 'label': 'Child (Control Arm)', 'description': 'Observational arm. Children (6-17 years) with any type of OI who are not receiving bisphosphonate treatment. Changes in hearing, quality of life, and bone density will be observed for the duration of the study.'}, {'type': 'NO_INTERVENTION', 'label': 'Adult Control Arm', 'description': 'Observational arm. Adults (18+ years) with type 1 OI. Changes in hearing, quality of life, and bone density will be monitored.'}], 'interventions': [{'name': 'Risedronate Oral Tablet', 'type': 'DRUG', 'otherNames': ['actonel'], 'description': 'Oral bisphosphonate', 'armGroupLabels': ['Adult Treatment Arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10021', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Su Htwe', 'role': 'CONTACT', 'email': 'htwes@hss.edu', 'phone': '(212) 774-2355'}], 'facility': 'Hospital for Special Surgery', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}], 'centralContacts': [{'name': 'Su Htwe', 'role': 'CONTACT', 'email': 'htwes@hss.edu', 'phone': '(212)774-2355'}, {'name': 'Erin Carter, MS, CGC', 'role': 'CONTACT', 'phone': '(212) 774-7332'}], 'overallOfficials': [{'name': 'Cathleen Raggio, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital for Special Surgery, New York'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hospital for Special Surgery, New York', 'class': 'OTHER'}, 'collaborators': [{'name': 'The New York Community Trust', 'class': 'OTHER'}, {'name': 'East River Medical Imaging', 'class': 'UNKNOWN'}, {'name': 'Northwell Health', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}