Clinical trials: Congenital Anomaly
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
Hemophilia A
Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A
This study is to describe the safety and efficacy of Xyntha® during the usual care setting.
- Trial name
- NCT01790828
- Testing
- Xyntha : coagulation factor IIIV (recombinant)
- Where
- 4 sites in South Korea
- Volunteers
- 42 people
- Sponsor
- Pfizer
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets, PAS-004 Capsule
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
Hereditary Angioedema (HAE)
Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study
This protocol for post study access allows pediatric participants (2-11 years old at the time of consent) with HAE who have completed study CSL312_3003 (NCT05819775) to continue treatment with CSL312 for routine prevention of HAE…
- Trial name
- NCT07159464
- Testing
- CSL312
- Sponsor
- CSL Behring
Atopic Dermatitis
Study of Abrocitinib Compared With Dupilumab in Adults With Moderate to Severe Atopic Dermatitis on Background Topical Therapy
This is a randomized, double-blind, double-dummy, active-controlled, multi-center study to assess the efficacy and safety of abrocitinib 200 mg (2 x 100 mg tablets) administered orally QD compared with dupilumab 300 mg…
- Trial name
- NCT04345367
- Testing
- Abrocitinib 200 mg, Dupilumab 300 mg
- Where
- 151 sites in United States, Australia and 13 more
- Volunteers
- 727 people
- Sponsor
- Pfizer
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Facioscapulohumeral Muscular Dystrophy, FSHD
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
Dermatitis, Atopic
Drug Drug Interaction Study Evaluating the Effect of Fluvoxamine or Fluconazole on PK and Safety of PF-04965842.
A Phase 1, Open Label, Parallel-Cohort, Randomized, Fixed Sequence Study To Evaluate The Pharmacokinetic Drug Drug Interaction Between PF-04965842 and Fkuvoxamine (cohort 1) or Fluconazole (Cohort 2) in healthy subjects.
- Trial name
- NCT03634345
- Testing
- Period 1 - Day 1: PF-04965842 administered, Period 2: Cohort 1: Fluvoxamine & PF-04965842, Period 2: Cohort 2: Fluconazole & PF-04965842
- Where
- Belgium
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
Thyroid Eye Disease, Graves' Orbitopathy
Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis With Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study
The overall objective is to investigate the efficacy, tolerability, and safety of teprotumumab (a fully human monoclonal antibody [mAb] inhibitor of the insulin-like growth factor-1 receptor [IGF-1R]) administered once every 3…
- Trial name
- OPTIC (NCT03298867)
- Testing
- Teprotumumab
- Where
- 14 sites in United States, Germany and 1 more
- Volunteers
- 83 people
- Sponsor
- Amgen
Cystic Fibrosis Lung
4D-710 in Adult Patients With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Trial name
- CF (NCT05248230)
- Testing
- 4D-710
- Where
- 20 sites in United States
- Volunteers
- 30 people
- Sponsor
- 4D Molecular Therapeutics
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
A Study on Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in Children, Teenagers and Adults in Portugal
The main aim of this study is to learn about the percentage of persons diagnosed with Dravet Syndrome (DS) and Lennox-Gastaut-Syndrome (LGS) in 2022 and of persons newly diagnosed in 2021 and 2022 compared to the overall…
- Trial name
- DRALEGA-PT (NCT06395792)
- Testing
- No Intervention
- Sponsor
- Takeda
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