Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 17, 2026, 1:35 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 134 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: BioMarin Pharmaceutical Clear all
Recruiting No phase listed Observational

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

NCT05579548
View directory record Official record
Conditions
Phenylketonuria, Maternal
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Female only
Enrollment
50 participants
Timeline
2022 – 2032
U.S. locations
1
States / cities
Wilmington, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 17, 2026, 1:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

NCT02354664
View directory record Official record
Conditions
Pompe Disease
Interventions
Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test
Other
Lead sponsor
University of Florida
Other
Eligibility
20 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 24, 2024 · Synced Oct 17, 2026, 1:35 AM EDT
Recruiting No phase listed Observational

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

NCT06212947
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 15 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2043
U.S. locations
16
States / cities
Phoenix, Arizona • Los Angeles, California • Oakland, California + 13 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2025 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

NCT00104247
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2014 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 17, 2025 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 3 Interventional Results available

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

NCT00838435
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 6 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced Oct 17, 2026, 1:35 AM EDT
Completed No phase listed Observational

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

NCT02294877
View directory record Official record
Conditions
Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Interventions
Vimizim® (elosulfase alfa)
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
418 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
26
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 22 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2024 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

NCT02485899
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190, Intracerebroventricular (ICV) access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 16 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 24, 2022 · Synced Oct 17, 2026, 1:35 AM EDT
Completed No phase listed Observational

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

NCT01603095
View directory record Official record
Conditions
Achondroplasia
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 17 Years
Enrollment
363 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Wilmington, Delaware + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

Sapropterin as a Treatment for Autistic Disorder

NCT00850070
View directory record Official record
Conditions
Autistic Disorder
Interventions
sapropterin, Placebo
Drug
Lead sponsor
The Children's Health Council
Other
Eligibility
3 Years to 6 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated May 1, 2018 · Synced Oct 17, 2026, 1:35 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

NCT00214773
View directory record Official record
Conditions
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
237 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2020
U.S. locations
23
States / cities
Irvine, California • Los Angeles, California • Oakland, California + 19 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2021 · Synced Oct 17, 2026, 1:35 AM EDT
Withdrawn Phase 2Phase 3 Interventional

Trial of Kuvan in Lesch-Nyhan Disease

NCT00935753
View directory record Official record
Conditions
Behavioral Manifestations of Lesch-Nyhan Disease
Interventions
sapropterin
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2019 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01415427
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA
Interventions
BMN 110 - Weekly, BMN 110 - Every Other Week
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
10
States / cities
Phoenix, Arizona • Oakland, California • Orange, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2021 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

NCT00532844
View directory record Official record
Conditions
Endothelial Dysfunction
Interventions
Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 75 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Hackensack, New Jersey
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 1 Interventional

Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies

NCT02392793
View directory record Official record
Conditions
Childhood Solid Tumors
Interventions
Talazoparib, Irinotecan, Temozolomide, Filgrastim, Peg-filgrastim
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Months to 25 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 9, 2021 · Synced Oct 17, 2026, 1:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

PKU Skin Stripping

NCT04368624
View directory record Official record
Conditions
Phenylketonurias
Interventions
Skin Tape, Blood Draw
Other · Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
86 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Oct 17, 2026, 1:35 AM EDT
Not listed No phase listed Observational

Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

NCT02365376
View directory record Official record
Conditions
Rare Genetic Disorders
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 18, 2015 · Synced Oct 17, 2026, 1:35 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase, Diet Only
Drug · Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
12 Years to 17 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 17, 2026, 1:35 AM EDT
Terminated Phase 2 Interventional Results available

Extension Study for Patients Who Have Participated in a BMN 701 Study

NCT01435772
View directory record Official record
Conditions
Pompe Disease
Interventions
BMN 701
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
13 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
4
States / cities
San Diego, California • Gainesville, Florida • Tampa, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated May 22, 2018 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

NCT01590446
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111, Normal Saline
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
22 Years to 45 Years · Male only
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 11, 2012 · Synced Oct 17, 2026, 1:35 AM EDT
Completed Phase 3 Interventional Results available

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

NCT00067470
View directory record Official record
Conditions
Mucopolysaccharidosis VI
Interventions
Placebo, N-acetylgalactosamine 4-sulfatase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
7 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Novato, California
Source: ClinicalTrials.gov public record
Updated Dec 24, 2009 · Synced Oct 17, 2026, 1:35 AM EDT
Active, not recruiting Phase 1 Interventional

The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication

NCT04800692
View directory record Official record
Conditions
Claudication, Intermittent, Peripheral Artery Disease, Peripheral Vascular Diseases
Interventions
Tetrahydrobiopterin 10 mg/kg, Tetrahydrobiopterin 20 mg/kg, L-Ascorbate, L-Arginine
Drug
Lead sponsor
Louis Messina
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 17, 2026, 1:35 AM EDT
Enrolling by invitation Phase 3 Interventional

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

NCT07073014
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Vosoritide
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
10
States / cities
Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 1:35 AM EDT
Recruiting No phase listed Observational

A Study to Assess Growth in Children With Idiopathic Short Stature

NCT06309979
View directory record Official record
Conditions
Idiopathic Short Stature
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
2 Years to 16 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2040
U.S. locations
38
States / cities
Phoenix, Arizona • Los Angeles, California • Orange, California + 33 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 17, 2026, 1:35 AM EDT