Clinical trials: sponsored by BioMarin Pharmaceutical
- Sponsor BioMarin Pharmaceutical
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency
- Trial name
- ENERGY (NCT05734196)
- Testing
- INZ-701
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Inozyme Pharma
Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
- Trial name
- NCT06046820
- Testing
- INZ-701, Control Arm (Conventional Therapy)
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 27 people
- Sponsor
- Inozyme Pharma
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy 1
- Trial name
- NCT07473973
- Testing
- INZ-701
- Where
- 8 sites in Brazil, France and 6 more
- Volunteers
- 2 people
- Sponsor
- Inozyme Pharma
Noonan Syndrome
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment.
- Trial name
- NCT06668805
- Testing
- Vosoritide Injection
- Where
- 38 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
- Trial name
- CANOPY-HCH-EXT (NCT07073014)
- Testing
- Vosoritide
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 140 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria (PKU)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
- Trial name
- PEGASUS (NCT05270837)
- Testing
- Pegvaliase, Diet Only
- Where
- 16 sites in United States, Germany
- Volunteers
- 55 people
- Sponsor
- BioMarin Pharmaceutical
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Cerliponase Alfa Observational Study in the US
This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with…
- Trial name
- NCT04476862
- Testing
- Cerliponase Alfa, Administration Kit
- Where
- 16 sites in United States
- Volunteers
- 35 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
VIrtual STudy in Achondroplasia for the US (VISTA)
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™.
- Trial name
- VISTA (NCT06168201)
- Where
- 8 sites in United States
- Volunteers
- 170 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Idiopathic Short Stature
A Study to Assess Growth in Children With Idiopathic Short Stature
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.
- Trial name
- NCT06309979
- Where
- 38 sites in United States
- Volunteers
- 300 people
- Sponsor
- BioMarin Pharmaceutical
Idiopathic Short Stature
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children…
- Trial name
- NCT06382155
- Testing
- Vosoritide Injection, Human Growth Hormone
- Where
- 47 sites in United States, Australia and 4 more
- Volunteers
- 100 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to < 36 months over a 52-week period.
- Trial name
- NCT07126262
- Testing
- Vosoritide
- Where
- 26 sites in United States, Australia and 5 more
- Volunteers
- 60 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria (PKU)
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the…
- Trial name
- PALace (NCT05813678)
- Testing
- Pegvaliase
- Where
- 26 sites in United States, Germany and 1 more
- Volunteers
- 450 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria (PKU)
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the…
- Trial name
- NCT06305234
- Testing
- Pegvaliase
- Where
- 11 sites in United States
- Volunteers
- 200 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria, Maternal
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and…
- Trial name
- PALomino (NCT05579548)
- Testing
- Pegvaliase
- Where
- 4 sites in United States, Canada and 2 more
- Volunteers
- 50 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
- Trial name
- NCT04554940
- Testing
- vosoritide
- Where
- 3 sites in Australia, United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia.
- Trial name
- ACH (NCT02724228)
- Testing
- BMN 111
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 30 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical