Clinical trials: Aetiology
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)/preliminary efficacy of…
- Trial name
- Unite-CNM (NCT04033159)
- Testing
- DYN101
- Where
- 8 sites in Belgium, Denmark and 4 more
- Volunteers
- 14 people
- Sponsor
- Dynacure
Prophylaxis of Avian Influenza
Immunogenicity, Safety and Tolerability of Two Doses of a Pre-pandemic Influenza Vaccine in Participants Aged 6 Months to 17 Years
Evaluate the immune response and reactogenicity of H5N1 vaccination in subjects aged 6 months to 17 years compared to seasonal flu vaccination
- Trial name
- NCT00537524
- Testing
- H5N1 Influenza Vaccine
- Where
- Finland
- Volunteers
- 471 people, healthy volunteers welcome
- Sponsor
- Novartis Vaccines
Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children
GB06 Phase III Trial for Pediatric Growth Hormone Deficiency
This study aims to evaluate the efficacy and safety of GB06 (a biosimilar of Norditropin®FlexProTM from Novo Nordisk) for the treatment of growth disorders caused by growth hormone deficiency (GHD) in children.
- Conditions
- Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children, Growth Hormone Deficiency, Pediatric
- Trial name
- NCT07553351
- Testing
- GB06, Norditropin®FlexProTM
- Where
- China
- Volunteers
- 192 people
- Sponsor
- Kexing Biopharm Co., Ltd.
Influenza Caused by the Novel Influenza A (H1N1) Virus
A Clinical Trial of CSL's 2009 H1N1 Influenza Vaccine (CSL425) in Healthy Children
The purpose of this study is to determine whether CSL425 is a safe and effective vaccine for eliciting an immune response to H1N1 influenza in healthy children.
- Trial name
- NCT00940108
- Testing
- CSL425
- Where
- 5 sites in Australia
- Volunteers
- 370 people, healthy volunteers welcome
- Sponsor
- Seqirus
X-Linked Retinitis Pigmentosa
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
- Trial name
- HORIZON (NCT03316560)
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Beacon Therapeutics
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
The purpose of this study is to characterize the disease progression of confirmed OPA1 mutation-associated autosomal dominant optic atrophy (ADOA) by evaluating the changes in ocular structural and functional outcomes.
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Trial name
- NCT06140329
- Where
- 7 sites in United States, Australia and 4 more
- Volunteers
- 1 people
- Sponsor
- PYC Therapeutics
Muscular Atrophy, Sprains
Study to Assess the Efficacy and Safety of Ibuprofen 50 mg/g Gel Compared to Profenid 25mg/g Gel
The aim of this study is to determine that ibuprofen 50mg/g gel is effective and safety treating patients with muscle aches, joint pains or due to sprains, bruises, tendinitis or myofascial compared to Profenid 25mg/g gel.
- Conditions
- Muscular Atrophy, Sprains, Tendonitis
- Trial name
- NCT01373697
- Testing
- Ibuprofen, Profenid
- Where
- Brazil
- Volunteers
- 144 people
- Sponsor
- Laboratório Teuto Brasileiro S/A
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.
- Trial name
- NCT06275620
- Testing
- AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity
A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants
The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants…
- Conditions
- Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity, Intraventricular Hemorrhage, Retinopathy of Prematurity (ROP)
- Trial name
- NCT03253263
- Testing
- OHB-607
- Where
- 62 sites in United States, Australia and 11 more
- Volunteers
- 295 people
- Sponsor
- OHB Neonatology Ltd.
Rotavirus Infection, Adenoviridae Infection
Study of Nitazoxanide Suspension in the Treatment of Diarrhea Caused by Enteric Viruses in Children
The purpose of this study is to determine the effect of nitazoxanide suspension in treating diarrhea caused by enteric viruses in children less than 12 years of age.
- Conditions
- Rotavirus Infection, Adenoviridae Infection, Norovirus Infection
- Trial name
- NCT00302640
- Testing
- Alinia (nitazoxanide)
- Volunteers
- 50 people
- Sponsor
- Romark Laboratories L.C.
Individual Patient Expanded Access for AB-PA01, an Investigational Anti-Pseudomonas Aeruginosa Bacteriophage Therapeutic
The purpose of the expanded access program is to allow physicians to provide treatment with investigational drug, AB-PA01, for patients with serious or immediately life-threatening Pseudomonas aeruginosa infections, for which no…
- Trial name
- NCT03395743
- Testing
- AB-PA01
- Sponsor
- Armata Pharmaceuticals, Inc.
Urinary Bladder Neurogenic
Pharmacokinetics and Safety Study With Alfuzosin in Children and Adolescents With Elevated Detrusor Leak Point Pressure of Neuropathic Etiology
The primary objective is to investigate the pharmacokinetics (PK) of 2 doses of alfuzosin (given as a solution or tablets depending on age) in children and adolescents 2 to 16 years of age with elevated detrusor Leak Point…
- Trial name
- ALFACHIP (NCT00629720)
- Testing
- alfuzosin (SL770499)
- Where
- 2 sites in United States, Serbia
- Volunteers
- 29 people
- Sponsor
- Sanofi
Methylmalonic Acidemia (MMA)
A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene
Methylmalonic Acidemia (MMA) is a severe and rare condition that affects how the body turns food into energy. In people with MMA, the body is missing or has a very low activity of a specific protein (an enzyme called…
- Trial name
- NCT07432880
- Where
- 8 sites in United States, Italy and 2 more
- Volunteers
- 30 people
- Sponsor
- Genespire Srl
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Double-stranded DNA Virus
Study to Assess Brincidofovir Treatment of Serious Diseases or Conditions Caused by Double-stranded DNA Viruses
This was a multicenter, open-label study of oral brincidofovir (BCV) treatment of serious disease or conditions caused by double-stranded DNA (dsDNA) virus(es).
- Trial name
- NCT01143181
- Testing
- Brincidofovir
- Where
- 37 sites in United States
- Volunteers
- 210 people
- Sponsor
- Jazz Pharmaceuticals
Restless Legs Syndrome
A Study of Rotigotine Patch in Adolescent Subjects With Restless Legs Syndrome of Unknown Cause
The purpose of this study is to demonstrate the efficacy of rotigotine against placebo in adolescent subjects with idiopathic Restless Legs Syndrome (RLS) over a 12-week maintenance period and to investigate the safety and…
- Trial name
- NCT03728933
- Testing
- Rotigotine 1 milligram/24 hours, Rotigotine 2 milligram/24 hours
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- UCB Biopharma SRL
Viral Infection, Bacterial Infections
A Controlled, Blinded Study to Validate the Diagnostic Accuracy and Assess the Clinical Utility of a Host-response Based Diagnostic Tool for Distinguishing Between Bacterial and Viral Etiologies in Pediatric Patients Presenting to the ED With Suspicion of Acute Infection
To externally validate the diagnostic accuracy and assess the clinical utility of a host-response based diagnostic tool called ImmunoXpert™, for differentiating between bacterial and viral etiologies in pediatric patients >3…
- Conditions
- Viral Infection, Bacterial Infections, Antibiotic Misuse, Host Response Diagnostics
- Trial name
- ROSETTA (NCT04254991)
- Testing
- ImmunoXpert™
- Volunteers
- 525 people
- Sponsor
- MeMed Diagnostics Ltd.
Diphtheria, Pertussis
Study of Quinvaxem for Vaccination Against Diphtheria, Pertussis, Tetanus, Hepatitis B and Diseases Caused by Haemophilus Influenzae Type B
The aim of this study was to evaluate the immunogenicity and safety of the Quinvaxem vaccine (a liquid combination vaccine against diphtheria, tetanus, B. pertussis, hepatitis B and H. influenzae Type B).
- Conditions
- Diphtheria, Pertussis, Tetanus, Hepatitis B
- Trial name
- NCT01362517
- Testing
- Quinvaxem
- Where
- Vietnam
- Volunteers
- 131 people, healthy volunteers welcome
- Sponsor
- Crucell Holland BV
Prevention of Infectious Disease Caused by SARS-CoV-2
VAXZEVRIA Japan Post-Marketing Surveillance (PMS) for the Long-term Safety of VAXZEVRIA
This survey will be conducted for the subjects who participate in the priority survey (cohort survey) conducted by the Scientific Research Group of the Ministry of Health, Labour and Welfare at the initial stage of administration…
- Trial name
- NCT05135455
- Where
- 6 sites in Japan
- Volunteers
- 467 people
- Sponsor
- AstraZeneca
Leber Congenital Amaurosis 10, Blindness
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
- Trial name
- BRIGHTEN (NCT04855045)
- Testing
- sepofarsen
- Where
- 9 sites in Belgium, Brazil and 5 more
- Volunteers
- 15 people
- Sponsor
- ProQR Therapeutics