Clinical trials: Newborn Diseases
Wilson Disease
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
- Trial name
- NCT02763215
- Testing
- Standard of Care Medications
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 64 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Gout
Phase 3, Febuxostat, Allopurinol and Placebo-Controlled Study in Gout Subjects.
The purpose of this study is to compare febuxostat, allopurinol and placebo, once daily (QD), in subjects with gout.
- Trial name
- APEX (NCT00174915)
- Testing
- Febuxostat, Allopurinol
- Volunteers
- 1,072 people
- Sponsor
- Takeda
Sickle Cell Disease
Study of SANGUINATE™ Versus Hydroxyurea in Sickle Cell Disease (SCD) Patients
The purpose of this study is to compare the safety of SANGUINATE™ versus Hydroxyurea in patients suffering from Sickle Cell Disease.
- Trial name
- NCT01848925
- Testing
- SANGUINATE™, Hydroxyurea
- Where
- 4 sites in Colombia, Panama
- Volunteers
- 24 people
- Sponsor
- Prolong Pharmaceuticals
Fuchs Endothelial Corneal Dystrophy
A Multicenter Study Assessing the Efficacy and Safety of STN1010904 Ophthalmic Suspension 0.03% and 0.1% Compared With Vehicle in Subjects With Fuchs Endothelial Corneal Dystrophy (FECD)
This is a Phase IIa study to assess efficacy and safety of STN1010904 ophthalmic suspension (0.03%, and 0.1 %), twice daily dosing when compared to Placebo in subjects diagnosed with Fuchs Endothelial Corneal Dystrophy (FECD).
- Trial name
- PHANTOM (NCT05376176)
- Testing
- STN1010904 ophthalmic suspension 0.03% BID, STN1010904 ophthalmic suspension 0.1% BID
- Where
- 15 sites in United States, France and 1 more
- Volunteers
- 86 people
- Sponsor
- Santen Inc.
Cystic Fibrosis
Efficacy and Safety of Inhaled Nitric Oxide (NO) in Cystic Fibrosis (CF) Patients
Prospective, randomized, placebo controlled, phase II clinical study of subjects crossing over from an approved inhaled antibiotic to inhaled nitric oxide as compared to a placebo control arm.
- Trial name
- NCT02498535
- Testing
- Nitric Oxide 160 ppm
- Where
- 7 sites in United States, Canada
- Volunteers
- 49 people
- Sponsor
- Novoteris, LLC
Arthrosis, Traumatic Arthritis
The Medacta International SMS Post-Marketing Surveillance Study
This is a Post-Marketing Surveillance of SMS femoral stem prosthesis.
- Conditions
- Arthrosis, Traumatic Arthritis, Rheumatoid Polyarthritis, Congenital Hip Dysplasia
- Trial name
- NCT02748408
- Testing
- SMS femoral stem
- Where
- 4 sites in Austria, Italy and 2 more
- Volunteers
- 550 people
- Sponsor
- Medacta International SA
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
IBS, Constipation Predominant
The Beneficial Effects on the Intestinal Function of a Food Supplement Based on an Extract of Green Tea and a Pool of Probiotics in Subjects With Irritable Bowel Syndrome With Predominance of Constipation
The study will aim to evaluate the effectiveness of the use of a food supplement, based on a green tea extract (Camellia sinensis (L.) Kuntze - folium) and a pool of probiotics (L. plantarum, L. rhamnosus and B. animalis subsp.
- Trial name
- NCT07094035
- Testing
- food supplement based on a mixture of green tea extract and a pool of probiotics.
- Where
- Italy
- Volunteers
- 64 people
- Sponsor
- Adamas Biotech S.r.l.
Hereditary Angioedema, Angioneurotic Edema
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening.
- Trial name
- NCT00225147
- Testing
- Recombinant Human C1 Inhibitor
- Where
- Netherlands
- Volunteers
- 77 people
- Sponsor
- Pharming Technologies B.V.
Huntington's Disease
A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod
The primary objective of this study is to assess the efficacy of laquinimod as treatment in participants with HD after 52 weeks using the Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS or TMS).
- Trial name
- LEGATO-HD (NCT02215616)
- Testing
- Laquinimod
- Where
- 53 sites in United States, Canada and 8 more
- Volunteers
- 352 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Wilson Disease
Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)
The goal of this clinical trial is to learn if GC310 (AAV5-ATP7B) gene therapy can treat Wilson's Disease (WD) in patients over the age of 18 years old.
- Trial name
- NCT07173933
- Testing
- GC310
- Where
- China
- Volunteers
- 15 people
- Sponsor
- GeneCradle Inc
Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension, Interstitial Lung Disease
A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)
This study evaluates the effect of ROC-101 in adults with either Pulmonary Arterial Hypertension (PAH) or Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH).
- Trial name
- ROCSTAR (NCT07175038)
- Testing
- ROC-101
- Where
- 24 sites in United States, Australia and 7 more
- Volunteers
- 40 people
- Sponsor
- AllRock Bio, Inc.
Noonan Syndrome
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested…
- Trial name
- NCT00351221
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Insmed Incorporated
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Atopic Dermatitis
Efficacy and Tolerability of a Probiotic Ointment
This study evaluates the tolerability and efficacy of a probiotic ointment compared with a comparator lotion in children with atopic dermatitis.
- Trial name
- NCT07701447
- Testing
- Probiotic Ointment, Comparator product
- Volunteers
- 92 people
- Sponsor
- BioGaia AB
Cystic Fibrosis
Cystic Fibrosis Remote Monitoring System
The objective of this study is to determine if interactive technology (i.e. virtual coaching) that facilitates vest therapy goal setting and provides positive reinforcement will increase adherence to vest therapy for airway…
- Trial name
- NCT04453358
- Testing
- InCourage-Connect, InCourage
- Volunteers
- 35 people
- Sponsor
- Koronis Biomedical Technologies
Cardiomyopathy, Dilated, Heart Failure
The Persistence of Autoantibody Neutralisation by BC 007 in Patients With Chronic HFrEF and Autoantibodies Against the Beta1-Adrenergic Receptor
Chronic heart failure (CHF) is one of the major causes of death in Western societies. Evidence has accumulated that functionally active autoantibodies directed against the beta1 adrenergic receptor (β1 AAb) are of…
- Conditions
- Cardiomyopathy, Dilated, Heart Failure, Autoantibodies
- Trial name
- NCT04192214
- Testing
- BC 007
- Where
- 3 sites in Serbia
- Volunteers
- 30 people
- Sponsor
- Berlin Cures GmbH
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).
- Trial name
- NCT04227106
- Testing
- EB-101
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Abeona Therapeutics, Inc
Hemophilia A
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A.
- Trial name
- Leopold II (NCT01233258)
- Testing
- rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose
- Where
- 47 sites in United States, Argentina and 16 more
- Volunteers
- 80 people
- Sponsor
- Bayer
Fed
Bioavailability Study of Desloratadine 5 mg Tablet of Dr. Reddy's Under Fed Conditions
The purpose of this study is to compare the rate and extent of absorption of Dr. Reddy's Desloratadine 5 mg tablet to that of Clarinex® 5 mg tablet in healthy subjects under fed conditions.
- Trial name
- NCT01314352
- Testing
- Desloratadine
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
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