Ignite Creation Date:
2025-12-25 @ 3:19 AM
Ignite Modification Date:
2025-12-31 @ 6:31 PM
Study NCT ID:
NCT06668805
Status:
RECRUITING
Last Update Posted:
2025-08-06
First Post:
2024-10-30
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009634', 'term': 'Noonan Syndrome'}, {'id': 'D014424', 'term': 'Turner Syndrome'}, {'id': 'D004392', 'term': 'Dwarfism'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D001847', 'term': 'Bone Diseases'}], 'ancestors': [{'id': 'D019465', 'term': 'Craniofacial Abnormalities'}, {'id': 'D009139', 'term': 'Musculoskeletal Abnormalities'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D006059', 'term': 'Gonadal Dysgenesis'}, {'id': 'D012734', 'term': 'Disorders of Sex Development'}, {'id': 'D014564', 'term': 'Urogenital Abnormalities'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D058533', 'term': 'Sex Chromosome Disorders of Sex Development'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D025064', 'term': 'Sex Chromosome Disorders'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D006058', 'term': 'Gonadal Disorders'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000632572', 'term': 'vosoritide'}, {'id': 'D019382', 'term': 'Human Growth Hormone'}], 'ancestors': [{'id': 'D013006', 'term': 'Growth Hormone'}, {'id': 'D010908', 'term': 'Pituitary Hormones, Anterior'}, {'id': 'D010907', 'term': 'Pituitary Hormones'}, {'id': 'D036361', 'term': 'Peptide Hormones'}, {'id': 'D006728', 'term': 'Hormones'}, {'id': 'D006730', 'term': 'Hormones, Hormone Substitutes, and Hormone Antagonists'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 72}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-11-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2041-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-01', 'studyFirstSubmitDate': '2024-10-30', 'studyFirstSubmitQcDate': '2024-10-30', 'lastUpdatePostDateStruct': {'date': '2025-08-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-10-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change from baseline in Annualized Growth Velocity (AGV)', 'timeFrame': 'At 6 months'}], 'secondaryOutcomes': [{'measure': 'Incidence of treatment-emergent adverse events', 'timeFrame': 'Until the end of the study, up to 15 years'}, {'measure': 'Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Incidence of cardiac conditions requiring discontinuation of study treatment', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in height', 'timeFrame': 'At 6 months and at 24 months'}, {'measure': 'Change from baseline in height Z-score', 'timeFrame': 'At 6 months and at 24 months'}, {'measure': 'Change from baseline in 12-month interval AGV', 'timeFrame': '24 months'}, {'measure': 'Change from baseline in upper to lower body segment ratio', 'timeFrame': 'At 12 and 24 months'}, {'measure': 'Change from baseline in arm span to height ratio', 'timeFrame': 'At 12 and 24 months'}, {'measure': 'Change from baseline in height up to Final Adult Height (FAH)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in height Z-score up to FAH', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': '12-month interval AGV summarized by age and sex up to FAH', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Tanner stage over the course of the study', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Time vosoritide is present at maximum concentration (Tmax)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Maximum vosoritide observed plasma concentration (Cmax)', 'timeFrame': 'Every 6 months through he end of the study, up to15 years'}, {'measure': 'Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Elimination half-life of vosoritide (t½)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Apparent clearance of vosoritide (CL/F)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Apparent volume of distribution of vosoritide (Vz/F)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Change from pre-dose in urine cyclic guanine monophosphate (cGMP)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in serum collagen X marker (CXM)', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in bone age/chronological age', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in total body (less head) BMD Z-score', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in lumbar spine bone mineral density (BMD) Z-score', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in total body (less head) bone mineral content (BMC)', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Change from baseline in lumbar spine BMC', 'timeFrame': 'Every 12 months through the end of the study, up to 15 years'}, {'measure': 'Change in bone morphology based on whole length lower extremity X-rays', 'timeFrame': 'Every 6 months through the end of the study, up to 15 years'}, {'measure': 'Incidence of bone-related events of special interest (fracture, slipped capital femoral epiphysis and avascular necrosis or osteonecrosis)', 'timeFrame': 'Throughout study'}, {'measure': 'Change from baseline in the physical domain score and total score of the QoLISSY', 'timeFrame': 'At 24 months'}, {'measure': 'Change from baseline in the physical and social domain scores and total score of the PedsQL', 'timeFrame': 'At 24 months'}, {'measure': 'Change from baseline in PGI-S and CaGI-S item scores', 'timeFrame': 'At 24 months'}, {'measure': 'PGI-C and CaGI-C item scores', 'timeFrame': 'At 24 months'}, {'measure': 'Change from baseline in PROMIS-SF Physical Activity score', 'timeFrame': 'At 24 months'}, {'measure': 'Change from baseline in KABC-II NVI scores', 'timeFrame': 'At 24 months'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Short Stature', 'Musculoskeletal Diseases', 'Bone Diseases', 'Developmental Endocrine System Diseases Natriuretic Peptide, C-Type'], 'conditions': ['Short Stature Homeobox- Containing Gene SHOX Deficiency', 'Noonan Syndrome', 'Turner Syndrome']}, 'descriptionModule': {'briefSummary': 'The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide versus hGH on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.', 'detailedDescription': 'This is a Phase 2, randomized, active-controlled, multicenter, basket study of vosoritide in children with Turner syndrome, short stature homeobox-containing gene (SHOX) deficiency, or Noonan syndrome who have an inadequate response to human growth hormone (hGH) treatment. The study is intended to characterize the short-term efficacy and safety of 3 dosing regimens of vosoritide versus hGH. The efficacy and safety of the vosoritide therapeutic dose will be further evaluated, with a comparison to hGH after 2 years of treatment, and an analysis of the impact of vosoritide on final adult height (FAH).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '3 Years', 'genderBased': True, 'genderDescription': 'Participants must be ≥ 3 years old, and \\<11 years old (female) or \\<12 years old (males) at the time of signing the informed consent form (ICF).', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Participants must be ≥ 3 years old, and \\< 11 years old (females) or \\< 12 years old (males), at the time of signing the informed consent form\n2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.\n3. A height assessment corresponding to a height Z-score of \\> -2.00 SDs and ≤ -1.75 SDs (up to 20% of participants)/≤ -2.00 SDs (at least 80% of participants) in reference to the general population of the same age and sex.\n4. Tanner Stage 1, at time of signing the ICF.\n5. Have been receiving continuous hGH for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment and be receiving a dose of ≥ 0.35 mg/kg weekly, with no weight-based dosing changes in the last 6 months and none planned in the future.\n6. Are willing to continue on hGH at their current dose for the Baseline Growth Phase, and for 2 years post randomization if randomized to the hGH arm.\n7. Inadequate response to prior hGH treatment.\n\nExclusion Criteria:\n\n1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.\n2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.\n3. Bone age advanced beyond chronological age by more than 2 years.\n4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,\n5. Have an unstable condition likely to require surgical intervention during the study.\n6. Evidence of decreased growth velocity (AGV \\< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays.\n7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.\n8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.'}, 'identificationModule': {'nctId': 'NCT06668805', 'briefTitle': 'A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With an Inadequate Response to Human Growth Hormone. (CANOPY NS, TS, SHOX-D-2)', 'orgStudyIdInfo': {'id': '111-211'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Vosoritide Dose 1 - Low Dose', 'description': 'Drug: Vosoritide - Dose 1 Injection\n\n• Experimental Drug Lyophilized powder for reconstitution', 'interventionNames': ['Drug: Vosoritide Injection']}, {'type': 'EXPERIMENTAL', 'label': 'Vosoritide Dose 2 - Medium Dose', 'description': 'Drug: Vosoritide - Dose 2 Injection\n\n• Experimental Drug Lyophilized powder for reconstitution', 'interventionNames': ['Drug: Vosoritide Injection']}, {'type': 'EXPERIMENTAL', 'label': 'Vosoritide Dose 3- High Dose', 'description': 'Drug: Vosoritide Dose 3 Injection\n\n• Experimental Drug Lyophilized powder for reconstitution', 'interventionNames': ['Drug: Vosoritide Injection']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Human Growth Hormone', 'description': 'Drug: Human Growth Hormone', 'interventionNames': ['Drug: Human Growth Hormone']}], 'interventions': [{'name': 'Vosoritide Injection', 'type': 'DRUG', 'description': 'Modified recombinant human C-type natriuretic peptide Vosoritide', 'armGroupLabels': ['Vosoritide Dose 1 - Low Dose', 'Vosoritide Dose 2 - Medium Dose', 'Vosoritide Dose 3- High Dose']}, {'name': 'Human Growth Hormone', 'type': 'DRUG', 'description': 'Commercial product containing somatotropin.', 'armGroupLabels': ['Human Growth Hormone']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Shanlee Davis', 'role': 'CONTACT', 'email': 'shanlee.davis@childrenscolorado.org', 'phone': '720-777-5251'}], 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '19803', 'city': 'Wilmington', 'state': 'Delaware', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Chijioke Ikomi', 'role': 'CONTACT', 'email': 'chijioke.ikomi@nemours.org', 'phone': '302-651-5965'}], 'facility': "Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)", 'geoPoint': {'lat': 39.74595, 'lon': -75.54659}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Andrew Dauber', 'role': 'CONTACT', 'email': 'adauber@childrensnational.org'}], 'facility': "Children's National Medical Center", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '33155', 'city': 'Miami', 'state': 'Florida', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Ana Maria Rodriguez Barreto', 'role': 'CONTACT', 'email': 'anamaria.rodriguezbarreto@nicklaushealth.org'}], 'facility': "Nicklaus Children's Hospital", 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '83712', 'city': 'Boise', 'state': 'Idaho', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Daniel Flynn', 'role': 'CONTACT', 'email': 'flynnd@slhs.org', 'phone': '2083817340'}], 'facility': "St. Luke's Children's Endocrinology and Diabetes", 'geoPoint': {'lat': 43.6135, 'lon': -116.20345}}, {'zip': '40506', 'city': 'Lexington', 'state': 'Kentucky', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Yuri Zarate', 'role': 'CONTACT', 'email': 'yuri.zarate@uky.edu', 'phone': '859-257-1000'}], 'facility': "Kentucky Children's Hospital", 'geoPoint': {'lat': 37.98869, 'lon': -84.47772}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Shilpa Mehta', 'role': 'CONTACT', 'email': 'shilpa_mehta@bchphysicians.org'}], 'facility': 'New York Medical College', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55454', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Kyriakie Sarafoglou', 'role': 'CONTACT', 'email': 'saraf010@umn.edu'}], 'facility': 'M Health Fairview Pediatric Specialty Clinic - Explorer', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Cassie Mintz', 'role': 'CONTACT', 'email': 'cassie.mintz@mssm.edu'}], 'facility': 'Icahn School of Medicine at Mount Sinai', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Laurie Cohen', 'role': 'CONTACT', 'email': 'lacohen@montefiore.org', 'phone': '718-920-4664'}], 'facility': "Children's Hospital at Montefiore", 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}, {'zip': '28203', 'city': 'Charlotte', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Edwin Ferren', 'role': 'CONTACT', 'email': 'edwin.ferren@atriumhealth.org'}], 'facility': 'Atrium Health Carolinas Medical Center', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '75235', 'city': 'Dallas', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Nadia Merchant', 'role': 'CONTACT', 'email': 'nadia.merchant@utsouthwestern.edu', 'phone': '214-456-5959'}], 'facility': "Children's Medical Center Dallas", 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'David Rodriguez Buritica', 'role': 'CONTACT', 'email': 'david.f.rodriguezburitica@uth.tmc.edu'}], 'facility': 'University of Texas Health Science Center at Houston (UT Health)', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '13385', 'city': 'Marseille', 'state': 'Bouches-du-Rhône', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Rachel Reynaud', 'role': 'CONTACT', 'email': 'rachel.reynaud@ap-hm.fr'}], 'facility': 'Hôpital de la Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'zip': '49 933', 'city': 'Angers', 'state': 'Maine-et-Loire', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Regis Coutant', 'role': 'CONTACT', 'email': 'recoutant@chu-angers.fr'}], 'facility': 'Angers University Hospital Center', 'geoPoint': {'lat': 47.47156, 'lon': -0.55202}}, {'city': 'Toulouse', 'state': 'Occitanie', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Thomas Edouard', 'role': 'CONTACT', 'email': 'edouard.t@chu-toulouse.fr'}], 'facility': 'CHU de Toulouse - Hôpital des Enfants', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'zip': '94270', 'city': 'Le Kremlin-Bicêtre', 'state': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Agnes Linglart', 'role': 'CONTACT', 'email': 'agnes.linglart@aphp.fr'}], 'facility': 'South Paris University Hospitals - Bicetre Hospital', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'zip': '75019', 'city': 'Paris', 'state': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Jean-Claude Carel', 'role': 'CONTACT', 'email': 'jean-claude.carel@rdb.aphp.fr'}], 'facility': 'Hôpital Robert-Debré', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '16147', 'city': 'Genova', 'state': 'Genoa', 'status': 'NOT_YET_RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Mohamad Maghnie', 'role': 'CONTACT', 'email': 'mohamadmaghnie@gaslini.org'}], 'facility': 'IRCCS Istituto Giannina Gaslini', 'geoPoint': {'lat': 45.21604, 'lon': 11.87211}}, {'zip': '33011', 'city': 'Oviedo', 'state': 'Principality of Asturias', 'status': 'NOT_YET_RECRUITING', 'country': 'Spain', 'contacts': [{'name': 'Isolina Riano Galan', 'role': 'CONTACT', 'email': 'isolinariano@gmail.com'}], 'facility': 'Central University Hospital of Asturias', 'geoPoint': {'lat': 43.36029, 'lon': -5.84476}}], 'centralContacts': [{'name': 'Trial Specialist', 'role': 'CONTACT', 'email': 'medinfo@bmrn.com', 'phone': '1-800-983-4587'}, {'name': 'Study Manager', 'role': 'CONTACT', 'email': 'Medinfo@bmrn.com', 'phone': '1-800-983-4587'}], 'overallOfficials': [{'name': 'Medical Director, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'BioMarin Pharmaceutical'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}