Clinical trials: Skeletal Dysplasia
Osteogenesis Imperfecta
Pediatric Safety Follow-up Study of Prior Treatment With Romosozumab for Osteogenesis Imperfecta
The primary objective of this trial is to evaluate the safety of romosozumab in participants with osteogenesis imperfecta (OI) that have completed Study 20200105, regardless of whether they received investigational product…
- Trial name
- NCT07366086
- Testing
- Vitamin D, Calcium
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 71 people
- Sponsor
- Amgen
Osteogenesis Imperfecta
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
The primary objective of the study is to evaluate the effect of setrusumab vs intravenous bisphosphonates (IV-BP) on reduction in fracture rate, including morphometric vertebral fractures in pediatric participants.
- Trial name
- Cosmic (NCT05768854)
- Testing
- Bisphosphonate, Setrusumab
- Where
- 20 sites in United States, Brazil and 5 more
- Volunteers
- 69 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Achondroplasia
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
This trial will evaluate the efficacy of KK8398 on annualized height velocity after 52 weeks of repeated administration of KK8398 to patients with achondroplasia
- Trial name
- NCT06926491
- Testing
- KK8398
- Where
- 9 sites in Japan
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Achondroplasia
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.
- Trial name
- NCT03583697
- Testing
- BMN 111
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.
- Trial name
- NCT04085523
- Testing
- TransCon CNP
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 57 people
- Sponsor
- Ascendis Pharma A/S
Achondroplasia
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height.
- Trial name
- NCT03989947
- Testing
- Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 73 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.
- Trial name
- NCT05813314
- Testing
- BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Exostoses, Multiple Hereditary
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple…
- Trial name
- MO-Ped (NCT03442985)
- Testing
- Palovarotene 2.5 mg, Palovarotene 5.0 mg
- Where
- 31 sites in United States, Australia and 10 more
- Volunteers
- 193 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Osteoarthritis, Post-traumatic Arthritis
A Randomised Single Centre Study to Compare the Long-term Wear Characteristics of Marathon™ and Enduron™ Polyethylene Cup Liners in Primary Total Hip Replacement
The purpose of this study is to compare the performance and compare the wear characteristics of two polyethylene cup liners, Marathon™ and Enduron™, in the treatment of patients with hip joint disease requiring a total hip…
- Conditions
- Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders, Avascular Necrosis
- Trial name
- NCT00208442
- Testing
- Marathon™, Enduron
- Where
- New Zealand
- Volunteers
- 122 people
- Sponsor
- DePuy International
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia.
- Trial name
- teACH (NCT06732895)
- Testing
- Navepegritide
- Where
- 5 sites in Canada, Denmark and 3 more
- Volunteers
- 25 people
- Sponsor
- Ascendis Pharma A/S
Achondroplasia
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin.
- Trial name
- NCT01603095
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 363 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
Achondroplasia
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to…
- Trial name
- NCT05116046
- Testing
- Recifercept
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Pfizer
Achondroplasia
A 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
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
Study of Infigratinib in Children With Achondroplasia
This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase…
- Trial name
- NCT04265651
- Testing
- Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 84 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Rheumatoid Arthritis, Osteoarthritis
A Multi-centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Polyethylene-on-metal Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Pinnacle™ Cup with a polyethylene-on-metal bearing combination in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00208429
- Testing
- Pinnacle Acetabular System
- Sponsor
- DePuy International
Achondroplasia
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
Approximately 63 participants will be randomized to one of three doses to receive Recifercept either Low Dose Medium Dose High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2…
- Trial name
- NCT04638153
- Testing
- Recifercept
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 60 people
- Sponsor
- Pfizer
Osteoarthritis, Post-traumatic Arthritis
A Randomised Single Centre Study to Compare the Long-term Performance of 4 Designs of the DePuy Ultima LX Stem in Primary Total Hip Replacement
The purpose of this study is to compare the performance of 4 designs of the DePuy Ultima LX hip stem in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Osteoarthritis, Post-traumatic Arthritis, Collagen Disorder, Avascular Necrosis
- Trial name
- NCT00208351
- Testing
- Ultima LX Collared Stem - Non-Polished/Blasted Finished, Ultima LX Collared Stem - Polished Finished, Ultima LX Collarless Stem - Non-Polished/Blasted Finished
- Where
- United Kingdom
- Volunteers
- 164 people
- Sponsor
- DePuy International
Achondroplasia
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia
Purpose of the study: The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China.
- Trial name
- NCT05246033
- Testing
- TransCon CNP
- Where
- 5 sites in China
- Volunteers
- 24 people
- Sponsor
- Visen Pharmaceuticals (Shanghai) Co., Ltd.