Clinical trials: Congenital Deformity
Fragile X Syndrome
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have…
- Trial name
- NCT01348087
- Testing
- AFQ056
- Where
- 28 sites in United States, Australia and 8 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
A Study to Evaluate the Safety of Lebrikizumab Compared to Topical Corticosteroids in Adult Patients With Atopic Dermatitis
The primary objective for this study is to evaluate the safety of lebrikizumab compared with Topical Corticosteroids (TCS) alone in patients with persistent moderate to severe Atopic Dermatitis (AD) that is inadequately…
- Trial name
- NCT02465606
- Testing
- Lebrikizumab, Topical Corticosteroid Creams (Triamcinolone acetonide 0.1% and Hydrocortisone 2.5%)
- Where
- 19 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency
The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects with A1PI deficiency and emphysema/ chronic…
- Trial name
- NCT02722304
- Testing
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 7 people
- Sponsor
- Baxalta now part of Shire
Fabry Disease
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks.
- Trial name
- NCT04974749
- Testing
- REPLAGAL
- Where
- 6 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Preterm Labor, Preterm Birth
MIRACLE of LIFE Study
The goal of this observational study is to develop and validate cell-free RNA-based biomarkers for predicting a variety of adverse pregnancy outcomes in a pregnant person population. The main question it aims to answer are: 1.
- Conditions
- Preterm Labor, Preterm Birth, Preterm Birth Complication, Preterm Premature Rupture of Membrane
- Trial name
- MoL (NCT06074601)
- Testing
- Mirvie Predictive Test for Adverse Pregnancy Outcomes
- Where
- 10 sites in United States
- Volunteers
- 10,000 people
- Sponsor
- Mirvie
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
Arthritis, Osteoarthritis
Tanezumab In Osteoarthritis Of The Knee (2)
The purpose of this study is to test the efficacy and safety of 2 doses of tanezumab compared with naproxen and placebo in patients with osteoarthritis.
- Trial name
- NCT00830063
- Testing
- tanezumab 10 mg, tanezumab 5 mg, naproxen
- Where
- 94 sites in United States
- Volunteers
- 832 people
- Sponsor
- Pfizer
Exocrine Pancreatic Insufficiency
Phase 1 Study to Assess Safety and Efficacy of ANG003
Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency.
- Trial name
- NCT06052293
- Testing
- ANG003
- Where
- 20 sites in United States
- Volunteers
- 57 people
- Sponsor
- Anagram Therapeutics, Inc.
Chronic Stable Angina
Pharmacokinetics of AUX-001 40mg Once-daily in Healthy Subjects Under Fasting and Fed Conditions
Exploratory, single-dose, open-label, pharmacokinetic study to establish uptake, plasma levels safety and tolerability of orally administered AUX-001 on an empty stomach (i.e, fasting) as well as after a meal (i.e.
- Trial name
- NCT06249581
- Testing
- AUX-001 40mg once-daily
- Where
- Portugal
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Auxilius Pharma sp.z.o.o.
Acute Noninfectious Posterior, Intermediate, or Pan Uveitis
Study of FTY720 in Patients With Uveitis
This study will assess the safety, tolerability, and efficacy of FTY720 in patients with acute, noninfectious intermediate, posterior and pan uveitis
- Trial name
- NCT01791192
- Testing
- FTY720, Oral Corticosteroid
- Sponsor
- Novartis Pharmaceuticals
Haemophilia A
Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities
This is a study on Italian patients with haemophilia A in prophylaxis treatment with Turoctocog alfa under routine clinical conditions.
- Trial name
- THIRd (NCT04584892)
- Testing
- Turoctocog alfa
- Where
- 10 sites in Italy
- Volunteers
- 18 people
- Sponsor
- Novo Nordisk A/S
Cystic Fibrosis
Indirect Comparison of Tobramycin Solution for Inhalation Versus Aztreonam Lysine for Inhalation in the Treatment of Cystic Fibrosis
Matching-adjusted indirect comparison of tobramycin solution (TIS) versus aztreonam lysine (AZLI) using TIS patient level clinical trial data and AZLI aggregated clinical trial data from published literature.
- Trial name
- NCT01460836
- Testing
- Tobramycin solution for inhalation, Aztreonam lysine for inhalation
- Sponsor
- Novartis Pharmaceuticals
Lax Skin
Evaluation of Facial and Neck Skin Laxity With SofWave Treatment in Adults Initiating Tirzepatide for Weight Loss
Open-label, randomized double-arm prospective, single-center, self-controlled clinical study.
- Trial name
- NCT07685834
- Testing
- SUPERB
- Where
- Canada
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Staphylococcus Aureus, Atopic Dermatitis
Exploratory Study to Evaluate the Application of NLAC Cream in Adults With Atopic Dermatitis
Atopic dermatitis (AD) is the most common chronic inflammatory skin disorder, with a lifetime prevalence of 15-25% in children and 1-3% in adults worldwide.
- Trial name
- NCT05092464
- Testing
- Natural Lactic Acid-enriched Cream
- Volunteers
- 10 people
- Sponsor
- Microbio Co Ltd
Angelman Syndrome
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
This is a phase I, multicenter, non-randomized, adaptive, open-label, multiple ascending, intra-participant, dose-escalation study with a long-term extension (LTE) part and an optional open-label extension (OOE) part.
- Trial name
- NCT04428281
- Testing
- RO7248824
- Where
- 12 sites in United States, Italy and 2 more
- Volunteers
- 74 people
- Sponsor
- Hoffmann-La Roche
Lennox Gastaut Syndrome, Dravet Syndrome
Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution
This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- NCT05044819
- Testing
- Cannabidiol
- Where
- 26 sites in United States
- Volunteers
- 154 people
- Sponsor
- Jazz Pharmaceuticals
Cardiovascular Abnormalities
Magnevist® Injection Enhanced MRA Compared to Non Contrast MRA for the Detection of Structural Abnormalities of the Aortic Arch and Cerebral Branches
The purpose of this study is to look at the safety (what are the side effects) and efficacy (how well does it work) of Magnevist (the study drug) used for MRI of the aortic arch and cerebral branches.
- Trial name
- NCT00310609
- Testing
- Gadopentetate dimeglumine (Magnevist)
- Volunteers
- 123 people
- Sponsor
- Bayer
Primary Immunodeficiency
A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency
The purpose of the study is to determine whether biweekly (every 2 weeks) administration of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) produces a steady-state area under the…
- Trial name
- NCT04566692
- Testing
- IGSC 20%
- Where
- 12 sites in United States
- Volunteers
- 33 people
- Sponsor
- Grifols Therapeutics LLC
Pompe Disease
A Global Prospective Observational Registry of Patients With Pompe Disease
This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD).
- Trial name
- NCT06121011
- Testing
- Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa
- Where
- 41 sites in United States, Austria and 10 more
- Volunteers
- 500 people
- Sponsor
- Amicus Therapeutics
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
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