Clinical trials: Effect Neonatal
Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia
Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.
This is a Phase 1, open-label, 3-period study to determine radiprodil's potential to act as a perpetrator of cytochrome P-450 (CYP) metabolic pathways and transporter pathways.
- Conditions
- Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia, Other Neurological Disorders
- Trial name
- NCT06975605
- Testing
- Radiprodil + co-administered drugs
- Where
- Australia
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- GRIN Therapeutics, Inc.
Cystic Fibrosis
Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The purpose of the study is to investigate the effects of miglustat on CFTR function in cystic fibrosis…
- Trial name
- NCT00537602
- Testing
- miglustat
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- Actelion
Duchenne Muscular Dystrophin (DMD)
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
Duchenne muscular dystrophin (DMD) is an X-linked, fatal muscle-wasting disease caused by mutations in the DMD gene encoding the dystrophin proteins, with symptom onset before age of 6 years in boys.
- Trial name
- MUSCLE (NCT06594094)
- Testing
- HG302
- Where
- China
- Volunteers
- 4 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Coronary Artery Disease, Atherosclerosis
Effect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery Disease
This study is a phase 1, intravenous, open-label, single-dose escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ACP-501 (recombinant human Lecithin Cholesterol Acyl Transferase…
- Trial name
- NCT01554800
- Testing
- ACP-501
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- MedImmune LLC
Gaucher Disease
A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Primary Objective: To study the effect of mild and moderate hepatic impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects…
- Trial name
- NCT02536911
- Testing
- eliglustat
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Pain
An Exploratory Study to Assess the Effects of JNJ-39439335 on the Relief of Pain Using a Thermal-Grill Experimental Model
This exploratory study will use a thermal-grill experimental model to assess the effects of JNJ-39439335 (50 mg, single oral dose) on the relief of pain.
- Trial name
- NCT01006304
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Coronary Heart Disease (CHD)
Effect of Evolocumab in Patients at High Cardiovascular Risk Without Prior Myocardial Infarction or Stroke
This study will assess the effect of lowering low-density lipoprotein cholesterol (LDL-C) with evolocumab on major cardiovascular events in adults without a prior myocardial infarction (MI) or stroke who are at high risk of a…
- Trial name
- VESALIUS-CV (NCT03872401)
- Testing
- Evolocumab
- Where
- 852 sites in United States, Argentina and 31 more
- Volunteers
- 12,301 people
- Sponsor
- Amgen
Cystic Fibrosis, Bronchiectasis
Effect of HFCWO Vests on Spirometry Measurements
The impact of high-frequency chest wall oscillation therapy on spirometry values (Forced Expiratory Volume, Forced Vital Capacity, Peak Expiratory Flow, Forced Expiratory Flow and Tidal Volume is investigated during use of…
- Trial name
- NCT03628456
- Testing
- International Biophysics AffloVest, Hill-Rom Monarch
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- International Biophysics Corporation
Sickle Cell Disease
Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.
The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease
- Trial name
- HESTIA5 (NCT04293172)
- Testing
- Brilinta
- Sponsor
- AstraZeneca
Atopic Dermatitis, Dermatitis Atopic
Dupilumab Skin BArrier Function and LIpidomics STudy in Atopic Dermatitis in China
This is a 16-week, open label, exploratory study designed to investigate dupilumab's effect on skin barrier function as measured by transepidermal water loss (TEWL) before and after skin tape stripping.
- Trial name
- BALISTAD-CN (NCT05624112)
- Testing
- Dupilumab
- Where
- China
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Sanofi
Skin Depressions, Skin Elasticity
A Study to Evaluate the Effect of Juvederm Volift With Lidocaine on Aging Hands in Participants Age 35 and Older
This study will evaluate the effect of Juvederm Volift with Lidocaine, an injectable gel implant, to treat aging hands of participants 35 and older.
- Conditions
- Skin Depressions, Skin Elasticity, Skin Laxity
- Trial name
- NCT04390581
- Testing
- Juvéderm® VOLIFT with Lidocaine
- Where
- France
- Volunteers
- 75 people
- Sponsor
- Allergan
Healthy Volunteers, Cryopyrin Associated Periodic Syndrome
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
This is a first in human (FIH), single-centre, double -blind, randomised, cross-over, SAD followed by a MAD study of IZD334 conducted in healthy adult participants as well as an open-label cohort in adult patients with CAPS.
- Trial name
- NCT04086602
- Testing
- IZD334
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Inflazome UK Ltd
Gut Microbiota Modulation
STUDY OF THE EFFECTS OF L. CASEI DG® (ENTEROLACTIS BABY) IN HEALTHY PRETERM AND FULL-TERM INFANTS.
The goal of this clinical trial was to learn whether giving Enterolactis Baby®, a probiotic food supplement containing Lactobacillus casei DG® (under the new taxonomical denomination: Lacticaseibacillus paracasei DG, CNCM…
- Trial name
- NCT07882537
- Testing
- Enterolactis Baby® drops
- Where
- Italy
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Alfasigma S.p.A.
GM2 Gangliosidosis, Tay-Sachs Disease
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff…
- Conditions
- GM2 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease
- Trial name
- NCT03759665
- Testing
- IB1001
- Where
- 8 sites in United States, Germany and 2 more
- Volunteers
- 30 people
- Sponsor
- IntraBio Inc
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Dermatitis, Atopic
Effect of Moisturising Creams on Skin Moisture in Atopic Dermatitis
The aim of this study is evaluate the effect of three moisturising creams on Stratum corneum (SC) hydratation and barrier function.
- Trial name
- NCT00846235
- Testing
- Aqualan L, Aqualan Plus, Aqualan L + glycerol ad 20%
- Where
- Finland
- Volunteers
- 24 people
- Sponsor
- Orion Corporation, Orion Pharma
Atopic Dermatitis
Epiceram™ Device Versus Mid-Strength Topical Steroid (Fluticasone Propionate 0.05%) for Treatment of Atopic Dermatitis
Novel therapies for AD that avoid immunosuppression and potential carcinogensis are needed. EpiCeram™ , a topical cream, represents a novel class of therapy for skin disorders that does not contain corticosteroids or other…
- Trial name
- NCT00616538
- Testing
- Epiceram(r), Fluticasone Propionate 0.05%
- Volunteers
- 121 people
- Sponsor
- Ceragenix Pharmaceuticals
Primary Immunodeficiency Diseases (PID)
A Study About How TAK-881 is Processed by the Body and Side Effects in People With Primary Immunodeficiency Diseases
The main aim of this study is to evaluate the PK, safety, tolerability and immunogenicity of subcutaneous (SC) administration of TAK-881 in adult and pediatric participants with PIDD and compare them to HYQVIA in participants 16…
- Trial name
- NCT05755035
- Testing
- TAK-881, HYQVIA
- Where
- 32 sites in United States, Czechia and 6 more
- Volunteers
- 65 people
- Sponsor
- Takeda
Fragile X Syndrome
Open Label Study Investigating Safety and Efficacy of NPL2009 50 mg - 150 mg on Prepulse Inhibition Tests and Continuous Performance Tasks, Adults With Fragile X Syndrome
This is an open label exploratory study to investigate the safety and effects of a single dose of NPL-2009(50 mg - 150 mg) on Prepulse Inhibition (PPI) Tests and Continuous Performance Tasks (CPT) in adults with Fragile X Syndrome
- Trial name
- NCT00637221
- Testing
- NPL-2009
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Neuropharm
Atopic Dermatitis
A Clinical Trial of BMX-010 in Adult Subjects With Atopic Dermatitis
Phase 2 clinical trial conducted in 2 parts: Part 1 - Pharmacokinetics and Part 2 - Randomized and Placebo Controlled (subject and clinical assessors will be blinded). Study Product will be applied to AD BID days 1-28.
- Trial name
- NCT05491447
- Testing
- BMX-010
- Where
- 19 sites in United States
- Volunteers
- 103 people
- Sponsor
- BioMimetix JV, LLC