Clinical trials: Effect Neonatal
Pregnancy, Hyperlipidemias
Bempedoic Acid Pregnancy Surveillance Program
Bempedoic acid pregnancy surveillance program
- Trial name
- NCT05103254
- Testing
- Bempedoic Acid, Bempedoic Acid / Ezetimibe
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Esperion Therapeutics, Inc.
Hypophosphatasia
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
The goal of this clinical trial is to compare the effectiveness of two doses of ilofotase alfa, an enzyme replacement treatment, in patients with hypophosphatasia (HPP).
- Trial name
- NCT05890794
- Testing
- Ilofotase Alfa, 0.8 mg/kg, Ilofotase Alfa, 3.2 mg/kg
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- AM-Pharma
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.
Aspergillosis, Cystic Fibrosis
The Effect of PC945 on Aspergillus Fumigatus Lung Infection in Patients With Cystic Fibrosis
This study tests the effects of an experimental drug PC945 in people with cystic fibrosis whose lungs are infected by the fungus Aspergillus fumigatus.
- Trial name
- NCT03870841
- Testing
- PC945
- Where
- 2 sites in United Kingdom
- Volunteers
- 4 people
- Sponsor
- Pulmocide Ltd
Lactose Intolerance
Effect of a Standard Meal on the Pharmacokinetic Profile of RP-G28 in Healthy Adult Male and Female Subjects
Randomized, open-label, 2-period, 2-sequence, crossover study to evaluate the effect of a standard meal on the pharmacokinetic (PK) profile of orally administered RP-G28.
- Trial name
- RP (NCT03563846)
- Testing
- RP-G28
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Ritter Pharmaceuticals, Inc.
Atopic Dermatitis
Effects of Lactobacillus Reuteri Plus Vitamin D3 in Children With Atopic Dermatitis
The purpose of this study is to evaluate in a randomized, double-blinded, controlled trial, whether a new food supplement containing Lactobacillus reuteri DSM 17938 and vitamin D3 (Reuterin® D3) may improve the SCORAD in…
- Trial name
- NCT02945683
- Testing
- Reuterin D3
- Where
- Italy
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Noos S.r.l.
Colic
The Effect of Probiotics on Symptoms of Infantile Colic
The aim of this study is to evaluate the effects of a probiotic formulation on the daily crying patterns of infants. It is hypothesized that participants given the probiotic formulation will show a significant reduction in daily…
- Trial name
- PROCOLIN (NCT05198700)
- Testing
- Probiotic Formulation
- Where
- 5 sites in Spain
- Sponsor
- Lallemand Health Solutions
Retinitis Pigmentosa (RP), Usher Syndrome
Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance
Retinitis pigmentosa and similar degenerative diseases of the retina lead to progressive loss of vision. TES therapy with the CE-marked OkuStim® System is a treatment approved in the EU for slowing the progression of the disease.
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome, Cone Rod Dystrophy
- Trial name
- TES-STEP (NCT07548944)
- Testing
- Transcorneal Electrical Stimulation using the OkuStim System
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Okuvision GmbH
Alpha-Mannosidosis
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.
- Trial name
- LAMPO(0-3) (NCT06184503)
- Testing
- Velmanase Alfa
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Mucopolysaccharidosis IVA, Morquio A Syndrome
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01609062
- Testing
- BMN 110
- Where
- 11 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
Sapropterin dihydrochloride (subsequently referred to as sapropterin) (Kuvan®) was approved by the FDA for the treatment of hyperphenylalaninemia in 2007.
- Trial name
- NCT00789568
- Testing
- sapropterin dihydrochloride, Moxifloxacin
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell Disease
A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)
The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12 weeks to patients with…
- Trial name
- STRONG SCD (NCT03285178)
- Testing
- IW-1701
- Where
- 37 sites in United States, Lebanon and 1 more
- Volunteers
- 88 people
- Sponsor
- Cyclerion Therapeutics
Alzheimer's Disease, Huntington's Disease
A Phase 1, Randomized, Open-Label, Two-Way Crossover Study To Evaluate The Steady-State Effect Of Dimebon (PF 01913539) On The Single-Dose Pharmacokinetics And Pharmacodynamics Of Warfarin In Healthy Subjects
This study will evaluate the potential drug-drug interaction of Dimebon with the FDA-recommended CYP2C9 substrate warfarin in healthy subjects.
- Trial name
- NCT00827034
- Testing
- Warfarin, Dimebon
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Pfizer
Gout
Phase 2a RDEA3170 and Allopurinol Combination Study in Gout Subjects
This is a Phase 2a, randomized, open-label, multicenter study to assess the pharmacodynamic (PD) effects of RDEA3170 administered in combination with allopurinol compared with allopurinol administered alone in adult subjects with…
- Trial name
- NCT02498652
- Testing
- RDEA3170 2.5 mg, allopurinol 300 mg
- Where
- 4 sites in United States
- Volunteers
- 41 people
- Sponsor
- Ardea Biosciences, Inc.
Gastroesophageal Reflux Disease
A Study to Evaluate the Effect of Lansoprazole on Hospitalized Neonates With Gastroesophageal Reflux Disease
The purpose of this study is to understand how quickly lansoprazole, once daily (QD), improves feeding in premature babies or babies less than 28 days of age.
- Trial name
- NCT00174928
- Testing
- Lansoprazole
- Volunteers
- 24 people
- Sponsor
- Takeda
Alpha 1-Antitrypsin Deficiency
Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin…
- Trial name
- SEQUOIA (NCT03945292)
- Testing
- Fazisiran Injection
- Where
- 21 sites in United States, Germany and 4 more
- Volunteers
- 40 people
- Sponsor
- Arrowhead Pharmaceuticals
Homozygous Familial Hypercholesterolemia
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
The available medications used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can retard the progression of cardiovascular disease, however, they are unlikely to regress…
- Trial name
- MODE (NCT01412034)
- Testing
- CER-001
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 23 people
- Sponsor
- Cerenis Therapeutics, SA
Advanced Solid Tumors, Advanced Breast Cancer
Phase 1/2 Study of ETX-636 in Participants With Advanced Solid Tumors
Phase 1/2, open-label study of ETX-636 in participants with advanced solid tumors
- Trial name
- NCT06993844
- Testing
- ETX-636 dose escalation, ETX-636 dose escalation in combination with fulvestrant, ETX-636 dose expansion in combination with fulvestrant
- Where
- 15 sites in United States, China
- Volunteers
- 233 people
- Sponsor
- Ensem Therapeutics
Atopic Dermatitis (Eczema)
The Effects of an L-histidine-Based Supplement, Eczema Topical, and Unscented Soap on Children With Eczema
The purpose of this study is to examine the effects of 2 dietary supplements containing the amino acid, L-histidine, alongside a plant-biotech topical product, and an unscented bar soap on 2 age groups of children with mild to…
- Trial name
- NCT07670676
- Testing
- Powder Supplement + Topical Oil + Unscented Soap, Gummy Supplement + Topical Lotion + Unscented Soap
- Where
- Poland
- Volunteers
- 40 people
- Sponsor
- Codex Labs Corporation
Gout, Hyperuricemia
A Randomized, Double-Blind, Dose-Response Study of the Safety and Uric Acid Effects of Oral Ulodesine Added to Allopurinol in Subjects With Gout and Concomitant Moderate Renal Insufficiency
To evaluate the overall safety and tolerability of ulodesine when combined with allopurinol in subjects with moderate renal insufficiency.
- Conditions
- Gout, Hyperuricemia, Arthritis, Joint Disease
- Trial name
- NCT01407874
- Testing
- Ulodesine (BCX4208) 5 mg, Ulodesine (BCX4208) 10 mg
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- BioCryst Pharmaceuticals