Clinical trials: Skeletal Dysplasia
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.
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period…
- Trial name
- ApproaCH (NCT05598320)
- Testing
- Navepegritide (TransCon CNP)
- Where
- 10 sites in United States, Australia and 5 more
- Volunteers
- 84 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
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
Biliary Tract Cancer, Cholangiocarcinoma
Gemcitabine Plus Cisplatin With or Without Bintrafusp Alfa (M7824) in Participants With 1L BTC
Study consisted of an open-label, safety run-in part and a randomized, double-blind, placebo-controlled Phase 2/3 part. In the Phase 2/3 part, the study was evaluated whether bintrafusp alfa in combination with the current…
- Conditions
- Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
- Trial name
- NCT04066491
- Testing
- M7824, Gemcitabine, Cisplatin
- Where
- 99 sites in United States, Argentina and 13 more
- Volunteers
- 309 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Cervical Cancer
Bintrafusp Alfa Combination Therapy in Participants With Cervical Cancer (INTR@PID 046)
This study was to evaluate the safety and tolerability of bintrafusp alfa in combination with other anti-cancer therapies in participants with locally advanced or advanced cervical cancer.
- Trial name
- NCT04551950
- Testing
- M7824, Carboplatin, Paclitaxel
- Where
- 13 sites in United States, Japan and 1 more
- Volunteers
- 25 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
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
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries.
- Trial name
- NCT03794609
- Where
- 57 sites in United States, Australia and 12 more
- Volunteers
- 315 people
- Sponsor
- Pfizer
Fibrous Dysplasia Of Bone
Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia
Background: Fibrous dysplasia (FD) is a disorder that affects bone growth. Affected bone tissue is weakened, and people with FD are prone to deformities, fractures, and other problems.
- Trial name
- NCT05509595
- Testing
- Burosumab
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Hip Osteoarthritis, Hip Impingement Syndrome
Losartan to Improve Hip Microfracture
Phase I/IIA randomized, double-blind, placebo-controlled clinical trial to assess safety and efficacy of oral losartan for improving cartilage repair following microfracture to treat focal cartilage defects during hip arthroscopy.
- Conditions
- Hip Osteoarthritis, Hip Impingement Syndrome, Cartilage Damage, Fibrosis
- Trial name
- NCT04212650
- Testing
- Losartan
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Steadman Philippon Research Institute
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
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
Osteopetrosis
Reduced Intensity AlloTransplant For Osteopetrosis
We believe that hematopoietic stem cell transplantation (HSCT) will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone architecture, and the anemia and…
- Trial name
- NCT00638820
- Testing
- Stem Cell or Umbilical Cord Blood Transplantation, Campath, Busulfan, Clofarabine, Total Lymphoid Irradiation
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Osteogenesis Imperfecta
Post-market Observational Study on JTIN Telescopic Nail in Osteogenesis Imperfecta Pediatric Patients
This study has been planned as part of the Orthofix Srl post-market active surveillance plan for the collection of data on both the clinical performance and the safety profile of the JTIN.
- Trial name
- JTIN (NCT05612139)
- Testing
- JTIN implantation surgery
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Orthofix s.r.l.
Achondroplasia
A Study to Learn About Recifercept in Patients With Achondroplasia
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs.
- Trial name
- NCT05659719
- Testing
- Recifercept
- Where
- United States
- Volunteers
- 248 people
- Sponsor
- Pfizer
Hepatocellular Carcinoma
A Study of LY2157299 in Participants With Advanced Hepatocellular Carcinoma
The main purpose of this study is to evaluate the safety and effectiveness of the study drug known as LY2157299 in participants with hepatocellular carcinoma.
- Trial name
- NCT02178358
- Testing
- LY2157299, Sorafenib
- Where
- 16 sites in China, Hong Kong and 3 more
- Volunteers
- 132 people
- Sponsor
- Eli Lilly and Company
Osteogenesis Imperfecta
Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta
The purpose of this study is to determine the effectiveness of teriparatide (FORTEO), which is human parathyroid hormone 1-34, for increasing bone mass and improving bone structure in adults affected with Osteogenesis Imperfecta…
- Trial name
- OI (NCT00131469)
- Testing
- Teriparatide (FORTEO)
- Where
- 3 sites in United States
- Volunteers
- 79 people
- Sponsor
- Oregon Health and Science University
Osteogenesis Imperfecta (OI)
Open-label Extension of Study 20130173 of Denosumab in Children and Young Adults With Osteogenesis Imperfecta
To evaluate long-term safety of denosumab in children/young adults with pediatric osteogenesis imperfecta (OI) who completed the prior study 20130173 (NCT02352753).
- Trial name
- NCT03638128
- Testing
- Denosumab, Alternative osteoporosis medications
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 75 people
- Sponsor
- Amgen
Osteogenesis Imperfecta
Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Children with Osteogenesis Imperfecta (OI) have bone pain, low bone mass and fractures. There are no approved drugs for the treatment of OI in children, even though some intravenous (IV) bisphosphonates are used off-label in some…
- Trial name
- NCT00106028
- Testing
- risedronate sodium (Actonel)
- Where
- 20 sites in United States, Australia and 11 more
- Volunteers
- 143 people
- Sponsor
- Warner Chilcott
Fibrosis, Focal Glomerulosclerosis
Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)
This study will examine the effectiveness of the drug pirfenidone in treating focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein…
- Conditions
- Fibrosis, Focal Glomerulosclerosis, Kidney Failure, Nephrotic Syndrome
- Trial name
- NCT00001959
- Testing
- Pirfenidone
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Osteoporosis, Cerebral Palsy
Prevention of Post Operative Bone Loss in Children
Hypothesis: one-dose pamidronate will prevent post-operative bone loss in children at risk for low bone density Plan: children with chronic disease such as CP, spina bifida, etc.
- Conditions
- Osteoporosis, Cerebral Palsy, Spina Bifida, Osteopenia
- Trial name
- NCT00655681
- Testing
- pamidronate, saline
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of New Mexico