Clinical trials: Tolerability
Pompe Disease (Late-onset)
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease.
- Trial name
- NCT06391736
- Testing
- GC301
- Where
- China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Homocystinuria
Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition").
- Trial name
- NCT03406611
- Testing
- Pegtibatinase
- Where
- 12 sites in United States, France and 1 more
- Volunteers
- 39 people
- Sponsor
- Travere Therapeutics, Inc.
Group B Streptococcus Infections
Trial To Evaluate The Safety, Tolerability, And Immunogenicity Of A Multivalent Group B Streptococcus Vaccine In Healthy Nonpregnant Women And Pregnant Women And Their Infants
Phase 1/2, randomized, placebo-controlled, observer-blinded study will evaluate the safety, tolerability and immunogenicity of the investigational multivalent group B streptococcus vaccine administered at one dose level (various…
- Trial name
- NCT03765073
- Testing
- Multivalent Group B streptococcus vaccine
- Where
- 35 sites in United States, South Africa and 1 more
- Volunteers
- 1,208 people, healthy volunteers welcome
- Sponsor
- Pfizer
Pediatric Growth Hormone Deficiency
A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)
This is a two-part clinical study for children with growth hormone deficiency. In the first part, participants will be randomly assigned to receive different doses of an oral treatment (GS3-007a dry suspension) or a placebo for…
- Trial name
- NCT07264595
- Testing
- GS3-007a dry suspension, rhGH injection
- Where
- China
- Volunteers
- 88 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer
Cow Milk Protein Allergy
CMPA-GO Study: Growth and Safety Outcomes in Infants With CMPA Fed a Novel Extensively Hydrolyzed Formula
CMPA-GO Study: Growth and safety Outcomes in infants with CMPA fed a novel extensively hydrolyzed formula
- Trial name
- CMPA-GO (NCT06830681)
- Testing
- New generation eHF, Commercial eHF
- Where
- Spain
- Volunteers
- 4 people
- Sponsor
- Société des Produits Nestlé (SPN)
Sickle Cell Disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512.
- Trial name
- NCT06546670
- Testing
- ITU512
- Where
- 14 sites in United States, Brazil and 1 more
- Volunteers
- 161 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Uncomplicated Plasmodium Falciparum Malaria
Efficacy, Safety and Tolerability of KLU156 in Adults and Children With Uncomplicated P. Falciparum Malaria
This study aims to confirm the efficacy, safety and tolerability of KLU156, a fixed dose combination of ganaplacide (KAF156) and a solid dispersion formulation of lumefantrine (lumefantrine-SDF), when administered once daily for…
- Trial name
- KALUMA (NCT05842954)
- Testing
- KLU156, Coartem
- Where
- 33 sites in Burkina Faso, Côte d’Ivoire and 10 more
- Volunteers
- 1,720 people
- Sponsor
- Novartis Pharmaceuticals
Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic
A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)
Macrophage Activation Syndrome (MAS) is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on the background of systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset…
- Conditions
- Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic, Arthritis, Juvenile, Adult Onset Still Disease
- Trial name
- NCT03311854
- Testing
- Emapalumab
- Where
- 5 sites in United States, France and 3 more
- Volunteers
- 14 people
- Sponsor
- Swedish Orphan Biovitrum
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)
This is a study of the safety and tolerability of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related…
- Trial name
- NCT04980872
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 60 people
- Sponsor
- Merck Sharp & Dohme LLC
Tourette's Disorder
Safety and Tolerability Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to obtain long term efficacy, safety and tolerability data of once weekly aripiprazole in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01416441
- Testing
- Aripiprazole
- Where
- 80 sites in United States, Bulgaria and 8 more
- Volunteers
- 170 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Leishmaniasis, Visceral
A Study Evaluating Sitamaquine Compared With Amphotericin B In The Treatment Of Visceral Leishmaniasis.
Sitamaquine is an 8-aminoquinoline which is being developed as an oral treatment for visceral leishmaniasis (VL). Pre-clinical and subsequent clinical investigations have demonstrated oral efficacy against Leishmania donovani.
- Trial name
- NCT00381394
- Testing
- sitamaquine
- Where
- 3 sites in India
- Volunteers
- 61 people
- Sponsor
- GlaxoSmithKline
Congenital Adrenal Hyperplasia
Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy
This study is an open-label, randomised, titration-blinded, parallel arm, multicenter study to compare twice daily Chronocort® with standard care in participants with Congenital Adrenal Hyperplasia (CAH).
- Trial name
- RESTORE (NCT03532022)
- Testing
- Chronocort®, Standard Care
- Where
- United States
- Sponsor
- Neurocrine UK Limited
Escherichia Coli Diarrhea
Enterotoxigenic Escherichia Coli (ETEC) ETVAX Vaccine Trial in Bangladesh
The purpose of this study is to determine if the ETEC vaccine ETVAX with and without dmLT adjuvant is safe and immunogenic in adults, children, toddlers and infants in Bangladesh.
- Trial name
- ETVAX/dmLT (NCT02531802)
- Testing
- ETVAX, dmLT, Bicarbonate Buffer
- Where
- Bangladesh
- Volunteers
- 475 people, healthy volunteers welcome
- Sponsor
- PATH
COVID-19
Study of Remdesivir in Participants Below 18 Years Old With COVID-19
The goals of this clinical study are to learn more about the study drug, remdesivir, and how safe it is in participants less than 18 years old with coronavirus disease 2019 (COVID-19).
- Trial name
- CARAVAN (NCT04431453)
- Testing
- Remdesivir
- Where
- 32 sites in United States, Italy and 2 more
- Volunteers
- 59 people
- Sponsor
- Gilead Sciences
Pulmonary Arterial Hypertension
A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension
The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to (>=)…
- Trial name
- NCT03492177
- Testing
- selexipag (Uptravi)
- Where
- 34 sites in United States, Belarus and 14 more
- Volunteers
- 63 people
- Sponsor
- Actelion
Papillomavirus Infections
A Study of the Safety, Tolerability, and Immunogenicity of a 9-valent Human Papillomavirus Vaccine ([9vHPV]; V503) Administered to 9- to 15-Year-Old Japanese Girls (V503-008).
This study will evaluate the safety, tolerability, and immunogenicity of V503 in Japanese girls between the ages of 9 and 15 and will determine whether V503 induces an acceptable immune response to all human papillomavirus (HPV)…
- Trial name
- NCT01254643
- Testing
- V503
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Asthma
Plasma Levels of MK0633 in Children Ages 6-12 (0633-023)
To evaluate the safety, tolerability and plasma levels of single oral doses of MK0633 in pediatric asthma patients ages 6 to less than 12 years.
- Trial name
- NCT00751413
- Testing
- Comparator: MK0633
- Volunteers
- 22 people
- Sponsor
- Merck Sharp & Dohme LLC
Neurogenic Detrusor Overactivity, Overactive Bladder
A Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Mirabegron OCAS (Oral Controlled Absorption System) in Pediatric Subjects With Neurogenic Detrusor Overactivity or Overactive Bladder
The purpose of this study is to evaluate the pharmacokinetics as well as the safety and tolerability of mirabegron OCAS tablets after single-dose administration at different dose levels in children and adolescents with NDO or OAB.
- Conditions
- Neurogenic Detrusor Overactivity, Overactive Bladder, Pharmacokinetics of Mirabegron
- Trial name
- NCT02211846
- Testing
- Mirabegron
- Where
- 13 sites in Belgium, Denmark and 3 more
- Volunteers
- 34 people
- Sponsor
- Astellas Pharma Inc
Asthma
Extension Study to Evaluate the Safety and Tolerability of Tezepelumab in Adults and Adolescents With Severe, Uncontrolled Asthma
Subjects who completed either D5180C00007 or D5180C00009 will be offered the opportunity to consent for the Multicentre, Double-blind, Randomized, Placebo Controlled, Parallel Group, Phase 3, Safety Extension Study to Evaluate…
- Trial name
- DESTINATION (NCT03706079)
- Testing
- Tezepelumab
- Where
- 180 sites in United States, Argentina and 16 more
- Volunteers
- 951 people
- Sponsor
- AstraZeneca
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