Clinical trials: Genetics
Cystic Fibrosis
Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults With Cystic Fibrosis.
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-032 in healthy adult subjects (Phase 1) and of two doses in Adults with Cystic Fibrosis (Phase 1b).
- Trial name
- NCT05712538
- Testing
- ARCT-032
- Where
- New Zealand
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Arcturus Therapeutics, Inc.
Acute Gouty Arthritis
Long-Term Safety and Tolerability of Canakinumab Prefilled Syringes in Frequently Flaring Acute Gouty Arthritis Patients
This is a 36 week open-label extension of the canakinumab pre-filled syringe study for safety and tolerability in patients who have frequent flares of acute gouty arthritis.
- Trial name
- NCT01431638
- Testing
- Canakinumab 150mg in prefilled syringe
- Where
- 68 sites in United States, Canada and 2 more
- Volunteers
- 397 people
- Sponsor
- Novartis Pharmaceuticals
Hunter Syndrome
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome.
- Trial name
- NCT05058391
- Testing
- Elaprase
- Where
- 5 sites in India
- Volunteers
- 5 people
- Sponsor
- Takeda
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
Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
This multicenter, open-label, non-randomized study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at a dose of 6 milligrams per kilogram (mg/kg) every 4 weeks in participants…
- Trial name
- HAVEN 4 (NCT03020160)
- Testing
- Emicizumab
- Where
- 17 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- Hoffmann-La Roche
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen
Haemophilia A
Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
Primary efficacy endpoint: To study whether there is a difference in the length of the bleeding free periods between infusion of FVIII that is reconstituted with 22.1, 12.6, or 4.2 mg of pegylated liposomes per kg bwt compared…
- Trial name
- NCT00245297
- Testing
- Kogenate FS, reconstituted in a suspension of liposomes
- Where
- 3 sites in Russia
- Volunteers
- 16 people
- Sponsor
- Recoly N.V.
Fabry Disease
Treatment Protocol of Replagal for Patients With Fabry Disease
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week.
- Trial name
- NCT01031173
- Testing
- agalsidase alfa
- Where
- 25 sites in United States
- Sponsor
- Shire
GM1 Gangliosidosis
A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis
LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system.
- Trial name
- NCT04273269
- Testing
- LYS-GM101
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 5 people
- Sponsor
- LYSOGENE
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Atopic Dermatitis
Human Testing of an Over-the-Counter (OTC) Cream in Children With Eczema
This three week study will be conducted to determine the safety and efficacy of an over-the-counter (OTC) cream in children with mild to moderate atopic dermatitis.
- Trial name
- NCT01326910
- Testing
- Colloidal Oatmeal, Topical Cream
- Where
- 2 sites in United States
- Volunteers
- 90 people
- Sponsor
- Johnson & Johnson Consumer Products Company Division of Johnson & Johnson Consumer Companies, Inc.
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
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
Hereditary Corneal Dystrophy, Corneal Abnormality
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Trial name
- CLARITY (NCT07750678)
- Testing
- GEB-101 Injection
- Where
- 2 sites in United States, China
- Volunteers
- 30 people
- Sponsor
- GenEditBio Limited
Pulmonary Alveolar Proteinosis, Bronchiectasis
Inhaled Molgramostim (rhGM-CSF) in Healthy Adult Subjects
This is a randomized, double-blind, placebo-controlled, single ascending (SAD), and multiple ascending dose (MAD) study conducted at a single clinical site within the UK.
- Conditions
- Pulmonary Alveolar Proteinosis, Bronchiectasis, Cystic Fibrosis, Acute Respiratory Distress Syndrome
- Trial name
- NCT02468908
- Testing
- Molgramostim
- Where
- United Kingdom
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Savara Inc.
Endocrine Disease, Adrenal Insufficiency
Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH
The purpose of this study is to gather safety and effectiveness information about a new formulation of Hydrocortisone (Chronocort®) used to treat patients with a disease called congenital adrenal hyperplasia (CAH).
- Conditions
- Endocrine Disease, Adrenal Insufficiency, Congenital Adrenal Hyperplasia
- Trial name
- NCT01735617
- Testing
- Hydrocortisone Modified Release Capsules
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Neurocrine UK Limited
Ataxia Telangiectasia
International Ataxia Rating Scale in Younger Patients
The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.
- Trial name
- NCT01942850
- Where
- 5 sites in United States, India and 2 more
- Volunteers
- 63 people
- Sponsor
- Quince Therapeutics S.p.A.
Down Syndrome, Edwards Syndrome
Non-Invasive Screening for Fetal Aneuploidy
The purpose of this study is to determine if a laboratory test developed by the Sequenom Center for Molecular Medicine (SCMM) that uses a new marker found in the mother's blood can better identify pregnancies that have a child…
- Trial name
- NCT00847990
- Testing
- Maternal blood screening test for fetal aneuploidy
- Where
- 25 sites in United States
- Volunteers
- 5,000 people
- Sponsor
- Sequenom, Inc.
Adenomatous Polyposis Coli
A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis
The purpose of this study is to determine the effect of JNJ-64251330 in participants with Familial Adenomatous Polyposis (FAP) on colorectal polyp burden (sum of the polyp diameters).
- Trial name
- NCT05014360
- Testing
- JNJ-64251330
- Where
- 16 sites in United States, France and 5 more
- Volunteers
- 42 people
- Sponsor
- Janssen Research & Development, LLC
Myotonic Dystrophy 1
Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
The purpose of this study is to determine whether MYODM (formulated composition containing Theobroma cacao supplemented with caffeine (caffeine/theobromine ratio1/1.85, w/w) is effective in the treatment of excessive daytime…
- Trial name
- NCT04634682
- Testing
- MYODM
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Myogem Health Company, S.L.