Clinical trials: Fetal Developmental Abnormality
Non-inflammatory Degenerative Joint Disease, Osteoarthritis
Study to Evaluate the Safety and Effectiveness of REAL INTELLIGENCE™ CORI™ in Unicondylar Knee Arthroplasty (UKA) Procedures
This is a prospective study to demonstrate the safety and effectiveness of CORI and to register CORI in China mainland.
- Conditions
- Non-inflammatory Degenerative Joint Disease, Osteoarthritis, Avascular Necrosis, Requires Correction of Functional Deformity
- Trial name
- CORI RCT UKA (NCT04797897)
- Testing
- CORI, Conventional
- Where
- 3 sites in Australia, Hong Kong and 1 more
- Volunteers
- 4 people
- Sponsor
- Smith & Nephew, Inc.
Recessive Dystrophic Epidermolysis Bullosa
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with…
- Trial name
- DEFI-RDEB (NCT04213261)
- Testing
- FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- Castle Creek Biosciences, LLC.
Homozygous Familial Hypercholesterolaemia (HoFH)
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein…
- Trial name
- NCT04681170
- Testing
- Lomitapide
- Where
- 12 sites in Germany, Israel and 4 more
- Volunteers
- 46 people
- Sponsor
- Amryt Pharma
Atopic Dermatitis
Evaluating the Effects of Tradipitant vs. Placebo in Atopic Dermatitis (EPIONE2)
This is a multicenter, randomized, double-blind, placebo-controlled study to be conducted in the United States in subjects with atopic dermatitis.
- Trial name
- NCT04140695
- Testing
- Tradipitant
- Where
- 51 sites in United States
- Volunteers
- 87 people
- Sponsor
- Vanda Pharmaceuticals
Familial Partial Lipodystrophy
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of fasting triglycerides in participants with familial partial lipodystrophy.
- Trial name
- NCT03514420
- Testing
- AKCEA-ANGPTL3-LRx
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Akcea Therapeutics
Hypophosphatemic Rickets, X Linked Dominant
Calcitonin for Treating X-linked Hypophosphatemia
X-linked hypophosphatemia (XLH) is the most common form of inherited rickets in the United States. It also causes bone disease in adults.
- Trial name
- NCT01652573
- Testing
- nasal salmon calcitonin
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Yale University
Neurofibromatosis Type 1, Sarcoma
Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00304083
- Testing
- filgrastim, doxorubicin hydrochloride, etoposide
- Where
- 17 sites in United States
- Volunteers
- 48 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
Congenital Hyperinsulinism (CHI), Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)
Phase II Safety and Efficacy Study of 18FDOPA PET-CT in Children With Hyperinsulinemic Hypoglycemia
Children with congenital hyperinsulinism (CHI) have low blood sugar, and some of these children may require surgery to remove part or all of their pancreas.
- Trial name
- 18FDOPA (NCT01468454)
- Testing
- 18 F-DOPA
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- Children's Hospital of Philadelphia
Lymphatic Malformations, Lymphatic Diseases
Sildenafil for the Treatment of Lymphatic Malformations
A Phase 2 study to evaluate safety and efficacy of sildenafil taken orally to improve or resolve lymphatic malformations in children.
- Trial name
- NCT02335242
- Testing
- Sildenafil 20 mg tablets
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Stanford University
Angiofibromas, Tuberous Sclerosis
Topical Rapamycin to Erase Angiofibromas in TSC
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous…
- Trial name
- Treatment (NCT01526356)
- Testing
- Rapamycin
- Where
- 10 sites in United States, Australia
- Volunteers
- 179 people
- Sponsor
- The University of Texas Health Science Center, Houston
Glioblastoma
A Study of DSP-7888 Dosing Emulsion in Combination With Bevacizumab in Patients With Recurrent or Progressive Glioblastoma Following Initial Therapy
This is an event driven, adaptive design, a randomized, active-controlled, multicenter, open-label, parallel groups, Phase 3 study of DSP-7888 Dosing Emulsion plus Bevacizumab versus Bevacizumab alone in patients with recurrent…
- Trial name
- NCT03149003
- Testing
- DSP-7888 Dosing Emulsion, Bevacizumab
- Where
- 61 sites in United States, Canada and 3 more
- Volunteers
- 221 people
- Sponsor
- Sumitomo Pharma America, Inc.
Growth Hormone Deficiency, Panhypopituitarism
Growth Hormone and Endothelial Function in Children
Objective: This study is designed to determine whether growth hormone treatment in children 8 to 18 years of age alters function of the lining of the arteries.
- Conditions
- Growth Hormone Deficiency, Panhypopituitarism, Short Stature
- Trial name
- NCT00373386
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ohio State University
Lennox Gastaut Syndrome (LGS)
A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome
The aims of the study are: to learn if soticlestat, when given as add-on therapy, reduces the number of major motor drop seizures in children, teenagers, and adults with Lennox-Gastaut Syndrome.
- Trial name
- NCT04938427
- Testing
- Soticlestat
- Where
- 98 sites in United States, Australia and 16 more
- Volunteers
- 270 people
- Sponsor
- Takeda
Tourette Syndrome
Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment.
- Trial name
- NCT02679079
- Testing
- NBI-98854
- Where
- 44 sites in United States
- Volunteers
- 98 people
- Sponsor
- Neurocrine Biosciences
Neonatal Abstinence Syndrome
Weighted Blankets With Infants With NAS
Infants with neonatal abstinence syndrome (NAS) who are cared for in a Neonatal Intensive Care Unit (NICU) often display symptoms including hyperactivity, irritability, jitteriness, poor feeding, and poor sleep patterns.
- Trial name
- NCT03113656
- Testing
- Weighted Blanket, Non-Weighted Blanket
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- TriHealth 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
Neonatal Abstinence Syndrome
Donor Human Milk in Neonatal Abstinence Syndrome
This study is designed to develop pilot data on the acceptability and benefit of donor human milk for infants undergoing pharmacologic treatment for NAS.
- Trial name
- DHM&NAS (NCT02182973)
- Testing
- donor human milk
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Neolac Inc dba Medolac Laboratories
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT00483379
- Testing
- alglucosidase alfa
- Where
- 11 sites in United States, Australia and 1 more
- Volunteers
- 13 people
- Sponsor
- Genzyme, a Sanofi Company
Anemia, Sickle Cell
Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA.
- Trial name
- NCT00890396
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 163 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
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