Clinical trials: Birth Defect
Atopic Dermatitis
A First-in-Human PoC Study With BEN2293 in Patients With Mild to Moderate Atopic Dermatitis
A randomised, adaptive design, double-blind, placebo-controlled, first-in-human, two-part study to investigate the safety, tolerability, PK and preliminary efficacy of multiple topical doses of BEN2293 in patients with mild to…
- Trial name
- NCT04737304
- Where
- United Kingdom
- Volunteers
- 123 people
- Sponsor
- BenevolentAI Bio
Hemophilia A
Study Evaluating The Safety Of Xyntha In Usual Care Settings
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year…
- Trial name
- NCT00765726
- Testing
- Moroctocog alfa(AF-CC), Blood draw for laboratory testing
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 12 people
- Sponsor
- Pfizer
Huntington's Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
This study tested the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of ISIS 443139 administered intrathecally to adult participants with early manifest Huntington's Disease.
- Trial name
- NCT02519036
- Testing
- ISIS 443139 10 mg, ISIS 443139 30 mg, ISIS 443139 60 mg
- Where
- 9 sites in Canada, Germany and 1 more
- Volunteers
- 46 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Cystic Fibrosis
Study Investigating a Delayed-Release Pancrelipase in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
This study will assess the effect of pancrelipase delayed release 24,000 unit capsules on fat and nitrogen absorption in subjects with PEI due to Cystic Fibrosis.
- Trial name
- NCT00510484
- Testing
- Pancrelipase Delayed Release
- Where
- 23 sites in United States, Hungary and 3 more
- Volunteers
- 35 people
- Sponsor
- Solvay Pharmaceuticals
Atopic Dermatitis
A Study to Evaluate the Safety and Efficacy of CBP-201 in Chinese Adult Subjects With Moderate to Severe AD
This study is a single-arm, open-label, multi-center clinical study designed to assess the safety and efficacy of CBP-201 in eligible subjects with moderate to severe Atopic Dermatitis.
- Trial name
- NCT05905133
- Testing
- CBP-201
- Where
- 38 sites in China
- Volunteers
- 360 people
- Sponsor
- Connect Biopharm LLC
Hypophosphatasia (HPP)
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
- Trial name
- NCT01203826
- Testing
- Asfotase Alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Rosacea, Vascular Diseases
Vbeam Pro Pulse Dye Laser for the Treatment of Vascular Conditions
This is a clinical study to evaluate the safety and performance of the Vbeam Pro Laser System for the treatment of vascular indications, including rosacea and port wine stain birthmarks.
- Conditions
- Rosacea, Vascular Diseases, Port-Wine Stain
- Trial name
- NCT07290426
- Testing
- Vbeam Pro Laser Treatment
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Candela Corporation
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
Sickle Cell Disease, Vaso-occlusive Crisis
A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Trial name
- NCT05348915
- Testing
- Inclacumab
- Where
- 50 sites in United States, Brazil and 8 more
- Volunteers
- 242 people
- Sponsor
- Pfizer
Primary Hypercholesterolaemia, Mixed Hyperlipidaemia
Lipid Efficacy/Tolerability Study (0524A-020)
A 28-week clinical trial in patients with primary hypercholesterolemia or mixed hyperlipidemia to study the effects of MK0524A on lipids and tolerability. There will be 9 scheduled clinic visits, and 3 treatment groups.
- Trial name
- NCT00269204
- Testing
- niacin (+) laropiprant, ER-niacin
- Volunteers
- 1,620 people
- Sponsor
- Merck Sharp & Dohme LLC
Atopic Dermatitis
Topical NF-kappaB Decoy in the Treatment of Atopic Dermatitis
The purpose of this study is to determine whether this topical NF-kappaB Decoy candidate is safe in persons with atopic dermatitis. Preliminary evidence of efficacy (whether it is working) will also be evaluated.
- Trial name
- NCT00125333
- Testing
- NF-kappaB Decoy
- Where
- 9 sites in United States
- Volunteers
- 75 people
- Sponsor
- Anesiva, Inc.
Spinocerebellar Degeneration
Phase II Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
To evaluate the efficacy, safety and pharmacokinetics profile of KPS-0373 in patients with SCD
- Trial name
- NCT00863538
- Testing
- KPS-0373
- Where
- 3 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Alport Syndrome
A Study of ELX-02 in Patients With Alport Syndrome
This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
- Trial name
- NCT05448755
- Testing
- ELX-02
- Where
- 4 sites in Australia, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Rheumatoid Arthritis, Osteoarthritis
A Multi-centre Study to Assess the Long-term Performance of the DePuy PROXIMA™ Hip in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the DePuy PROXIMA™ hip in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Avascular Necrosis
- Trial name
- NCT00208455
- Testing
- DePuy Proxima™ Hip
- Where
- 4 sites in Germany, Italy and 2 more
- Volunteers
- 184 people
- Sponsor
- DePuy International
Phenylketonuria (PKU)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
- Trial name
- PEGASUS (NCT05270837)
- Testing
- Pegvaliase, Diet Only
- Where
- 16 sites in United States, Germany
- Volunteers
- 55 people
- Sponsor
- BioMarin Pharmaceutical
Rheumatoid Arthritis, Osteoarthritis
An Electronic Data Capture Study to Assess the Long-term Performance of the DePuy PROXIMA™ Hip in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the DePuy PROXIMA™ hip in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Avascular Necrosis
- Trial name
- NCT01134445
- Testing
- DePuy Proxima™ Hip
- Where
- 4 sites in France, Portugal and 2 more
- Volunteers
- 42 people
- Sponsor
- DePuy International
Urothelial Carcinoma
Derazantinib and Atezolizumab in Patients With Urothelial Cancer
The purpose of this study was to evaluate efficacy of derazantinib monotherapy or derazantinib-atezolizumab in combination in patients with advanced urothelial cancer harboring fibroblast growth factor receptor (FGFR) genetic…
- Trial name
- FIDES-02 (NCT04045613)
- Testing
- Derazantinib 300 mg once daily monotherapy, Derazantinib 200 mg once daily + atezolizumab 1200 mg, Derazantinib 300 mg once daily+ atezolizumab 1200 mg
- Where
- 66 sites in United States, Australia and 12 more
- Volunteers
- 95 people
- Sponsor
- Basilea Pharmaceutica
Hemophilia A, Hemophilia B
Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
- Trial name
- NCT02740413
- Volunteers
- 86 people
- Sponsor
- Pfizer
Sarcoma, Neuroblastoma
To Evaluate the Safety, Activity and Pharmacokinetics of Marqibo in Children and Adolescents With Refractory Cancer
Background: Marqibo(Registered Trademark) is a new anticancer drug. It combines Vincristine sulfate, which is a widely used anticancer drug, and packages it into a tiny fat bubble known as a liposome.
- Conditions
- Sarcoma, Neuroblastoma, Wilms Tumor, Leukemia
- Trial name
- NCT01222780
- Testing
- Marqibo
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Acrotech Biopharma Inc.
Chronic Hepatitis C Infection
Open-Labeled Study of PSI-7977 and RBV With and Without PEG-IFN in Treatment-Naïve Patients With HCV GT2 or GT3
This study is to assess the safety and tolerability of sofosbuvir (SOF) 400 mg with and without ribavirin (RBV) and/or with and without pegylated interferon alfa-2a (PEG) in subjects with genotype 1, 2 or 3 hepatitis C (HCV)…
- Trial name
- NCT01260350
- Testing
- SOF, RBV, PEG
- Where
- 2 sites in New Zealand
- Volunteers
- 292 people
- Sponsor
- Gilead Sciences