Clinical trials: Congenital Anomalies
Atopic Dermatitis
Evaluation of the Efficacy of Topialyse Baume barrière on Atopic Dermatis
The purpose of this study is to evaluate the efficacy and tolerability of a new SVR care product and to evaluate the reduction of the topical corticosteroids' consumption with this emollient care.
- Trial name
- TOPIA (NCT05006300)
- Testing
- Topialyse Baume Barrière
- Sponsor
- SVR Group
Thalassemia, Iron Overload
Evaluation of the Quality of Life in Patients With Chronic Iron Overload Due to Hemoglobinopathies in Greece.
This observational clinical study aims to evaluate the HRQoL of thalassemia patients with iron overload in Greece, who are under treatment with deferasirox based on standard clinical practice.
- Trial name
- FEROUSA (NCT07352878)
- Volunteers
- 150 people
- Sponsor
- Elpen Pharmaceutical Co. Inc.
Turner's Syndrome
An Open-Label, Multi-centre, Phase III Study of Local Tolerability of ZOMACTON 10MG
Children with growth failure due to inadequate secretion of growth hormone or growth retardation due to Turner's syndrome are individually dosed and treated for 12 weeks
- Trial name
- NCT00250250
- Testing
- ZOMACTON
- Where
- 6 sites in Czechia, France and 1 more
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- Ferring Pharmaceuticals
Usher Syndromes
Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.
- Trial name
- SLO RP (NCT04355689)
- Testing
- NPI-001
- Where
- 4 sites in Australia
- Volunteers
- 49 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Sickle Cell Disease
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
- Trial name
- GBT_HOPE (NCT03036813)
- Testing
- voxelotor
- Where
- 62 sites in United States, Canada and 10 more
- Volunteers
- 449 people
- Sponsor
- Pfizer
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
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of CMK389 in Patients With Moderate to Severe Atopic Dermatitis.
The main purpose of this phase 2 study was to assess the efficacy and safety of CMK389 in patients with atopic dermatitis.
- Trial name
- NCT04836858
- Testing
- CMK389
- Where
- 18 sites in Czechia, France and 4 more
- Volunteers
- 71 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
- Trial name
- REALIZE (NCT01524289)
- Testing
- Anacetrapib
- Volunteers
- 306 people
- Sponsor
- Merck Sharp & Dohme LLC
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen
Skin Abnormalities
A Pilot Evaluation Exploring New Adhesive Materials and Their Ability to Adhere to Abdominal and Peristomal Skin
The study investigates different adhesive materials on healthy and peristomal skin
- Trial name
- NCT05641168
- Testing
- Adhesive material, Standard adhesive material
- Where
- Denmark
- Volunteers
- 114 people, healthy volunteers welcome
- Sponsor
- Coloplast A/S
Cardiopulmonary Failure, Myocardial Infarction (MI)
COR-INSIGHT: Optimizing Cardiovascular and Cardiopulmonary Outcomes with AI-Driven Multiplexed Indications Using COR ECG Wearable
The COR-INSIGHT trial aims to evaluate the effectiveness of Peerbridge COR advanced ambulatory ECG wearables (COR 1.0 and COR 2.0) in accurately and non-invasively detecting cardiovascular and cardiopulmonary conditions using…
- Conditions
- Cardiopulmonary Failure, Myocardial Infarction (MI), Heart Decompensation, Heart Failure
- Trial name
- COR-INSIGHT (NCT06763549)
- Testing
- SUBPROTOCOL A, SUBPROTOCOL B, SUBPROTOCOL C
- Where
- United States
- Volunteers
- 15,000 people, healthy volunteers welcome
- Sponsor
- Peerbridge Health, Inc
Cystic Fibrosis
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT05844449
- Testing
- VNZ/TEZ/D-IVA
- Where
- 37 sites in United States, Australia and 8 more
- Volunteers
- 174 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- NCT07263685
- Testing
- No intervention
- Where
- Saudi Arabia
- Volunteers
- 50 people
- Sponsor
- Takeda
Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
A Natural History Study of Aspartylglucosaminuria
Aspartylglucosaminuria (AGU) is a rare neurodegenerative lysosomal storage disease (LSD) characterized by developmental delay, psychomotor regression, worsening intellectual disability, gait disturbance and, ultimately, premature…
- Conditions
- Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency, Lysosomal Storage Diseases
- Trial name
- AGU (NCT03853876)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Neurogene Inc.
Hereditary Hemorrhagic Telangiectasia
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers evaluate the efficacy, safety, tolerability and PD of…
- Trial name
- InsigHHT (NCT06659640)
- Testing
- ALN-6400
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Hemophilia A
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Primary objective To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives To characterize additional pharmacokinetic…
- Trial name
- NCT05042440
- Testing
- Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegol
- Where
- Bulgaria
- Volunteers
- 13 people
- Sponsor
- Sanofi
Mesial Temporal Lobe Epilepsy
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).
- Trial name
- GenTLE (NCT06063850)
- Testing
- AAV9-hSyn1-miGRIK2
- Where
- 18 sites in United States
- Volunteers
- 12 people
- Sponsor
- UniQure Biopharma B.V.
Embolic Stroke of Undetermined Source, Transient Ischemic Attack
RoBotic TCD Ultrasound BubbLe Study Compared to Transthoracic Echocardiography for Detection of Right to Left Shunt
This study is a multi-center, prospective, single-arm, non-significant risk (NSR) device study in which up to 150 evaluable subjects with suspicion of embolic stroke of undetermined source (ESUS) will be evaluated with NB-IS TCD…
- Conditions
- Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Right-To-Left Atrial Shunt, Patent Foramen Ovale
- Trial name
- BUBL (NCT04604015)
- Testing
- NeuralBot Investigational System, Transthoracic Echocardiography (TTE)
- Where
- 7 sites in United States
- Volunteers
- 154 people
- Sponsor
- NeuraSignal Inc dba NovaSignal
Foramen Ovale, Patent
The Paradigm II Trial: PFX Closure System in Subjects With Cryptogenic Stroke, Transient Ischemic Attack, Migraine or Decompression Illness
The primary objective of this study is to demonstrate the safety and efficacy of the PFX Closure System when utilized for patent foramen ovale (PFO) in patients suffering from cryptogenic stroke (undetermined cause of stroke)…
- Trial name
- NCT00196040
- Testing
- PFX Closure System
- Where
- Germany
- Volunteers
- 60 people
- Sponsor
- Cierra
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