Clinical trials: Physiological Effects of Drugs
- Condition Physiological Effects of Drugs
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Eosinophilic Esophagitis
Safety Study of CC-93538 in Adult and Adolescent Participants With Eosinophilic Esophagitis
This study is an open-label, uncontrolled study design to evaluate the long-term safety and tolerability of treatment with CC-93538.
- Trial name
- NCT04991935
- Testing
- CC-93538
- Where
- 167 sites in United States, Argentina and 13 more
- Volunteers
- 367 people
- Sponsor
- Celgene
Multiple Sclerosis
Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)
The aim of this retrospective study is to review and describe safety, tolerability and efficacy of Rebif® (subcutaneous interferon [IFN]-beta-1a) in children and adolescents, using information already recorded in medical records.
- Trial name
- REPLAY (NCT01207648)
- Testing
- Rebif®
- Where
- 18 sites in United States, Argentina and 6 more
- Volunteers
- 307 people
- Sponsor
- EMD Serono
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Eosinophilic Esophagitis
A Study to Evaluate the Efficacy and Safety of CC-93538 in Adult and Adolescent Participants With Eosinophilic Esophagitis
Study CC-93538-EE-001 is a Phase 3, multicenter, multinational, randomized, double-blind, placebo-controlled induction and maintenance study to evaluate the efficacy and safety of CC- 93538 in adult and adolescent participants…
- Trial name
- NCT04753697
- Testing
- CC-93538
- Where
- 212 sites in United States, Argentina and 13 more
- Volunteers
- 430 people
- Sponsor
- Celgene
Diabetes Mellitus, Type 1, Diabetes Mellitus
Diamyd Administered Into Lymph Nodes in Combination With Vitamin D in Type 1 Diabetes
The objective of DIAGNODE-2 is to evaluate the efficacy of Diamyd compared to Placebo, upon administration directly into a lymph node in combination with an oral vitamin D/Placebo regimen, in terms of preserving endogenous…
- Conditions
- Diabetes Mellitus, Type 1, Diabetes Mellitus, Autoimmune Diseases, Metabolic Disease
- Trial name
- NCT03345004
- Testing
- Diamyd, Vitamin D
- Where
- 18 sites in Czechia, Netherlands and 2 more
- Volunteers
- 109 people
- Sponsor
- Diamyd Medical AB
Infantile Hemangioma
Study in Infants With Infantile Hemangioma to Compare Propranolol Gel to Placebo
There is an unsatisfied medical need for a first-line treatment of localized uncomplicated proliferating Infantile Hemangioma with a good benefit/risk profile.
- Trial name
- NCT01512173
- Testing
- propranolol gel
- Where
- 17 sites in France, Poland and 1 more
- Volunteers
- 82 people
- Sponsor
- Pierre Fabre Dermatology
Hereditary Angioedema
Safety and Pharmacokinetics of GNR-038 in Healthy Volunteers
It is an open-label dose-escalating study in sequential cohorts to assess safety and pharmacokinetics of GNR-038.
- Trial name
- NCT04557319
- Testing
- GNR-038, 25 МЕ/kg, GNR-038, 50 МЕ/kg, GNR-038, 100 МЕ/kg
- Where
- Russia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.
- Trial name
- NCT03583697
- Testing
- BMN 111
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
- Trial name
- CANOPY-HCH-3 (NCT06455059)
- Testing
- Vosoritide
- Where
- 23 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- BioMarin Pharmaceutical