Clinical trials: Defect, Congenital
Atopic Dermatitis
Clinical Relevance of Scratching and Sleep in Atopic Dermatitis
Atopic Dermatitis (AD), otherwise known as (atopic) eczema, is a chronic relapsing inflammatory skin disease. For difficult-to-treat AD, treatment options are limited.
- Trial name
- NCT05467046
- Testing
- Smartwatch system
- Where
- Netherlands
- Volunteers
- 16 people
- Sponsor
- Philips Clinical & Medical Affairs Global
X-Linked Adrenoleukodystrophy
A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice.
- Trial name
- NCT03278899
- Where
- 3 sites in United States
- Volunteers
- 100 people
- Sponsor
- NeuroVia, Inc.
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
A Phase 1 Study of Single and Multiple Ascending Doses of GV-100
This is a first-in-human, multi-part clinical study designed to evaluate the safety, tolerability, pharmacokinetics (PK), food effect, and drug-drug interaction (DDI) potential of GV-100 following oral administration in healthy…
- Trial name
- NCT07788014
- Testing
- GV-100
- Where
- Australia
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Gilva Therapeutics Inc.
Arterio-Venous Fistula, Kidney Failure, Chronic
VALUE- Vascular Access Laminate eUropean Experience. A Post Market Surveillance Study to Assess the Safety and Effectiveness of VasQ
This is a prospective single arm study enrolling patients who are scheduled for creation of a new Brachiocephalic or Radiocephalic AV fistula.
- Conditions
- Arterio-Venous Fistula, Kidney Failure, Chronic, Kidney Diseases, Renal Failure Chronic
- Trial name
- VALUE (NCT03246984)
- Testing
- VasQ
- Where
- 11 sites in France, Germany and 2 more
- Volunteers
- 80 people
- Sponsor
- Laminate Medical Technologies
Hernia, Inguinal, Flat Foot
The Efficacy and Safety of Chloroprocaine 1% and 2% in Pediatric Population
Randomized, multi-center, double-blind, two-armed, parallel active groups, prospective trial to evaluate the efficacy and safety of local anesthetic Chloroprocaine at two different concentrations ( at 1% and 2%) in a pediatric…
- Trial name
- NCT03918798
- Testing
- Chloroprocaine 1% Injectable Solution, Chloroprocaine 2% Injectable Solution
- Where
- 9 sites in Italy, Spain
- Volunteers
- 164 people
- Sponsor
- Sintetica SA
Alström Syndrome
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with…
- Trial name
- NCT03184584
- Testing
- PBI-4050
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Liminal BioSciences Ltd.
Prader-Willi Syndrome
A Study of Pitolisant in Patients With Prader-Willi Syndrome
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
- Trial name
- NCT06366464
- Testing
- Pitolisant tablet
- Where
- 57 sites in United States, Australia and 11 more
- Volunteers
- 134 people
- Sponsor
- Harmony Biosciences Management, Inc.
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1
Study of CAD-1883 for Spinocerebellar Ataxia
This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3
- Trial name
- Synchrony-1 (NCT04301284)
- Testing
- CAD-1883
- Where
- 6 sites in United States
- Sponsor
- Cadent Therapeutics
Huntington Disease
Safety Evaluation of Cellavita HD Administered Intravenously in Participants With Huntington's Disease
Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a first-in-human, non-randomized, phase I study in which participants with Huntington's Disease will receive three intravenous injections and will be followed…
- Trial name
- SAVE-DH (NCT02728115)
- Testing
- Cellavita HD Lower Dose, Cellavita HD Higher dose
- Where
- Brazil
- Volunteers
- 6 people
- Sponsor
- Azidus Brasil
Hemophilia A
BAX 855 Continuation
To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.
- Trial name
- NCT01945593
- Testing
- BAX855
- Where
- 89 sites in United States, Australia and 21 more
- Volunteers
- 218 people
- Sponsor
- Baxalta now part of Shire
Dermatitis, Atopic
Skin Irritation Study of GSK2894512 Cream
GSK2894512 is a novel anti-inflammatory agent that is currently under development for the topical treatment of atopic dermatitis and chronic plaque psoriasis.
- Trial name
- NCT02637206
- Testing
- GSK2894512 Cream, Vehicle Cream, Empty Patch
- Where
- Japan
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Hemophilia A
Study Evaluating ReFacto AF in Severe Hemophilia A
To characterize the safety and efficacy of ReFacto AF in treating acute bleeding episodes during prophylaxis treatment, including neoantigenicity.
- Trial name
- NCT00037544
- Testing
- ReFacto AF
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Atopic Dermatitis
Steroid-Sparing Effect With Pimecrolimus in Pediatric Patients With Atopic Dermatitis
This study will investigate the steroid sparing effect of pimecrolimus cream 1% in pediatric atopic dermatitis
- Trial name
- NCT00351052
- Testing
- Pimecrolimus
- Where
- Germany
- Volunteers
- 184 people
- Sponsor
- Novartis
Dermatitis, Atopic
Clinical Effect, Safety and Tolerability of GSK1070806 in Atopic Dermatitis
This study will evaluate efficacy and safety of GSK1070806 in moderate to severe atopic dermatitis (AtD) participants.
- Trial name
- NCT04975438
- Testing
- GSK1070806
- Where
- 11 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- GlaxoSmithKline
Alagille Syndrome
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
- Trial name
- NCT06850038
- Where
- 10 sites in United States
- Volunteers
- 30 people
- Sponsor
- Ipsen
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.
- Conditions
- Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00364858
- Testing
- Cerezyme
- Where
- 26 sites in United States, Brazil and 5 more
- Volunteers
- 95 people
- Sponsor
- Genzyme, a Sanofi Company
Migraine Disorders, Heart Septal Defects, Atrial
ESCAPE Migraine Trial
The purpose of this two arm controlled double-blind study is to determine the safety and effectiveness of PFO closure (closing a hole in the wall of the heart) in reducing the frequency of migraine headaches, in patients who…
- Trial name
- NCT00267371
- Testing
- PFO Closure with Premere investigational device., Medical management/current medications per standard of care by personal physician.
- Where
- 37 sites in United States
- Volunteers
- 168 people
- Sponsor
- Abbott Medical Devices
Atopic Dermatitis, Skin Diseases
Study to Assess the Efficacy and Safety of Orismilast in Atopic Dermatitis (ADESOS)
This study investigates 3 different doses of orismilast modified release compared to placebo in adult patients with moderate-to-severe atopic dermatitis.
- Trial name
- NCT05469464
- Testing
- Orismilast modified release tablets
- Where
- 48 sites in United States, Germany and 2 more
- Volunteers
- 235 people
- Sponsor
- UNION therapeutics
Healthy Volunteers
Single and Multiple Dose Study to Evaluate Safety and Pharmacokinetics of BMS-986533 in Healthy Participants and Assessments of Food and pH Effects on Relative Bioavailability, and Drug-Drug Interaction Potential in Healthy Participants
The purpose of this study is to evaluate the safety and pharmacokinetics of BMS-986533 in healthy participants receiving single and multiple doses, to assess food and pH effects on the relative bioavailability of BMS-986533, and…
- Trial name
- NCT07629999
- Testing
- BMS-986533, Famotidine, Dabigatran Etexilate
- Where
- United States
- Volunteers
- 136 people, healthy volunteers welcome
- Sponsor
- Bristol-Myers Squibb