Clinical trials: Skeletal Dysplasia
Genetic Disorder, Achondroplasia
Special Survey for Long Term Application
This study is conducted in Japan. The aim of this study is to assess the incidence rate of adverse drug reactions (ADRs) when using somatropin (Norditropin®) for treatment of for achondroplasia without epiphyseal line closure…
- Trial name
- NCT01516229
- Testing
- somatropin
- Where
- 2 sites in Japan
- Volunteers
- 395 people
- Sponsor
- Novo Nordisk A/S
Puberty, Precocious, McCune-Albright Syndrome
Faslodex in McCune-Albright Syndrome
The purpose of this study is to evaluate the safety, effectiveness and pharmacokinetics of a study drug called Faslodex (fulvestrant) in the treatment of progressive precocious puberty (PPP) (early puberty) in girls with…
- Trial name
- FMAS (NCT00278915)
- Testing
- Fulvestrant
- Where
- 16 sites in United States, France and 4 more
- Volunteers
- 30 people
- Sponsor
- AstraZeneca
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Rhizomelic Chondrodysplasia Punctata
A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics
PPI-1011 is being developed as a docosahexaenoic acid (DHA) containing plasmalogen precursor with good long-term stability, specifically for the treatment of rhizomelic chondrodysplasia punctata (RCDP), which is an ultra rare…
- Trial name
- NCT05969977
- Testing
- PPI-1011
- Where
- Canada
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- MED-LIFE DISCOVERIES LP
Genetic Disorder, Achondroplasia
A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin
This study is conducted in Japan. The aim of the study is to evaluate the efficacy of somatropin (Norditropin®) on adult height (cm) in patients with achondroplasia / hypochondroplasia enrolled in the GH-1941 study (NCT01516229).
- Trial name
- NCT01435629
- Testing
- somatropin
- Where
- Japan
- Volunteers
- 81 people
- Sponsor
- Novo Nordisk A/S
Rheumatoid Arthritis, Osteoarthritis
A Single Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Ceramic-on-ceramic Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Pinnacle™ Cup with a ceramic-on-ceramic 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
- NCT00872222
- Testing
- Pinnacle™ Acetabular System
- Where
- Czechia
- Volunteers
- 98 people
- Sponsor
- DePuy International
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
Bone Diseases, Bone Cysts
Safety and Efficacy of Non-Setting Paste in Bone Defect Reconstruction
This study aims to evaluate the safety and efficacy of a novel bone substitute material for the treatment of bone defects in patients requiring defect filling following the excision of benign bone tumors, cysts, or similar…
- Conditions
- Bone Diseases, Bone Cysts, Bone Neoplasm, Enchondromatosis
- Trial name
- NCT07379008
- Testing
- Non-setting paste
- Where
- Poland
- Volunteers
- 29 people
- Sponsor
- Biovico Sp. z o.o.
Achondroplasia
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
The purpose of this study is to measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose.
- Trial name
- NCT01590446
- Testing
- BMN 111, Normal Saline
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Rheumatoid Arthritis, Osteoarthritis
A Study to Assess the Long-term Performance of SmartSet® HV and SmartSet® GHV Bone Cements in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of artificial hip joints implanted with two different bone cements, SmartSet® HV and SmartSet® GHV, in the treatment of patients with hip joint disease requiring a total hip…
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872066
- Testing
- SmartSet® HV bone cement, SmartSet® GHV bone cement
- Where
- Slovakia
- Volunteers
- 243 people
- Sponsor
- DePuy International
Osteogenesis Imperfecta
A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers
To understand if AGA2115 is safe and well tolerated in healthy adult volunteers.
- Trial name
- NCT06086613
- Testing
- AGA2115
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Angitia Incorporated Limited
Osteoarthritis (OA) of the Hip, Avascular Necrosis of Bone of Hip
MDR Vivacit-E Elevated Study
The main objective of this study is to confirm long-term safety, performance, and clinical benefits of the Vivacit-E Vitamin E HXLPE Elevated Liners when used in primary total hip arthroplasty.
- Conditions
- Osteoarthritis (OA) of the Hip, Avascular Necrosis of Bone of Hip, Protrusio Acetabuli, Traumatic Arthropathy-Hip
- Trial name
- NCT07150221
- Testing
- Vivacit-E Elevated Liners
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Zimmer Biomet
Achondroplasia
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
This is a multicenter, non-randomized, open-label, phase I/II study in children with ACH. This study will start with a dose escalation of ABSK061 in children with ACH to evaluate the safety, tolerability, PK, and efficacy.
- Trial name
- NCT07297875
- Testing
- ABSK061
- Where
- 7 sites in China
- Volunteers
- 110 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Achondroplasia
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with…
- Trial name
- ACH (NCT02055157)
- Testing
- BMN 111
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 35 people
- Sponsor
- BioMarin Pharmaceutical
Non-inflammatory Degenerative Joint Disease (NIDJD), Avascular Necrosis
MDR - Longevity IT Oblique and Offset Liners
The objective of this retrospective enrollment/prospective follow-up consecutive series PMCF study is to collect data confirming safety, performance, and clinical benefits of the Longevity IT Oblique and Offset liners when used…
- Conditions
- Non-inflammatory Degenerative Joint Disease (NIDJD), Avascular Necrosis, Protrusio Acetabuli, Traumatic Arthritis
- Trial name
- NCT05980234
- Testing
- Total Hip Arthroplasty with Longevity IT Oblique Liners, Total Hip Arthroplasty with Longevity IT Offset Liners
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Zimmer Biomet
SEDC, Hypochondrogenesis
A Natural History Study in Children With a Type II Collagen Disorder With Short Stature
There are relatively few data available on type II collagen disorders, and evidence is lacking on the disease course in relation to symptoms and development of complications, the level of actual disease burden over time as well…
- Conditions
- SEDC, Hypochondrogenesis, Semd, Strudwick Type, Kniest Dysplasia
- Trial name
- ROCKET (NCT05408715)
- Testing
- Natural History Study
- Where
- 2 sites in France, Spain
- Volunteers
- 60 people
- Sponsor
- Innoskel
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
- Trial name
- NCT05353192
- Testing
- Recombinant human growth hormone
- Where
- China
- Volunteers
- 39 people
- Sponsor
- Changchun GeneScience Pharmaceutical 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 Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH.
- Trial name
- NCT03875534
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 260 people
- Sponsor
- Ascendis Pharma A/S
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