Clinical trials: Patients
Junctional Epidermolysis Bullosa
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17)
Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying…
- Trial name
- HOLOGENE17 (NCT03490331)
- Testing
- Transplantation surgery of genetically corrected cultured epidermal autograft (ATMP)
- Where
- Austria
- Volunteers
- 1 people
- Sponsor
- Holostem s.r.l.
Duchenne Muscular Dystrophy
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies.
- Trial name
- NCT02525302
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Akashi Therapeutics
Hemophilia A
Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives.
- Trial name
- PARkER (NCT03405337)
- Testing
- FVIII products, Conventional FVIII replacement therapies
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- Bayer
Ion Beam Radiation Therapy for Tumor Patients
Ion Therapy Patient Registry
The purpose of the study is the prospective and standardized data collection of patients treated with ion therapy at MedAustron.
- Trial name
- REGI-MA-002015 (NCT03049072)
- Testing
- Protons and Carbon Ions
- Where
- Austria
- Volunteers
- 2,800 people
- Sponsor
- EBG MedAustron GmbH
Gastroesophageal Reflux Disease (GERD)
Safety of Daily Dose Esomeprazole for GERD for Pediatric and Adolescent Patients
This study is a phase III, multi-centre, randomized, double-blind study to assess the safety and tolerability of once daily treatment with esomeprazole 20 or 40 mg in pediatric and adolescent patients 12 to 17 years of age…
- Trial name
- NCT00241501
- Testing
- Esomeprazole (Nexium)
- Where
- 40 sites in United States, Canada and 2 more
- Volunteers
- 140 people
- Sponsor
- AstraZeneca
Spinal Muscular Atrophy
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
- Trial name
- SMNRx (NCT01494701)
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 28 people
- Sponsor
- Biogen
Safety Issues, HPV Infection
BS-FU01 Follow-Up Study of FluBHPVE6E7 Study Subjects
This is a follow-up study for patients treated with FluBHPVE6E7 in previous interventional studies.
- Trial name
- NCT04528407
- Testing
- Observation without intervention
- Where
- Austria
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- BlueSky Immunotherapies GmbH
Different Classes of the Antihypertensive Treatment
Egypt Hypertension Study
This study is an epidemiological non-interventional trial that aims to evaluate standard daily practice in managing patients with arterial hypertension.
- Trial name
- NCT02604771
- Where
- Egypt
- Volunteers
- 1,500 people
- Sponsor
- AstraZeneca
Adrenal Insufficiency
Extension Study for Patients Entered Into Study Infacort 003
A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia…
- Trial name
- NCT02733367
- Testing
- Infacort®
- Where
- Germany
- Volunteers
- 18 people
- Sponsor
- Neurocrine UK Limited
Obesity, Genetic Obesity
EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
The protocol describes a randomized, double-blind, placebo-controlled trial with independent sub-studies of setmelanotide in patients with obesity and at least one of the specific gene variants in the Melanocortin-4 Receptor…
- Trial name
- NCT05093634
- Testing
- Setmelanotide
- Where
- 56 sites in United States, Canada and 8 more
- Volunteers
- 296 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Choroidal Neovascularization (CNV)
Assess the Efficacy/Safety of Intravitreal Ranibizumab in Patients With Vision Loss Due to Choroidal Neovascularization.
This study was conducted to evaluate the efficacy and safety of 0.5 mg ranibizumab in adult and adolescent patients with visual impairment due to choridal neovascularization (CNV).
- Trial name
- NCT01840410
- Testing
- Ranibizumab
- Where
- 59 sites in Australia, Canada and 19 more
- Volunteers
- 183 people
- Sponsor
- Novartis Pharmaceuticals
Hallux Rigidus, Unspecified Foot
Radiological Stage and Efficacy of an Intra-articular Injection of Hyaluronic Acid for the Treatment of Hallux Rigidus
The hallux rigidus is the most frequent type of arthrosis at the age of 50 and beyond. For the moderate forms of the disease, the majority of the studies recommend the recourse to the viscosupplement because of its efficiency in…
- Trial name
- REPAR (NCT03622723)
- Where
- France
- Volunteers
- 65 people
- Sponsor
- Labrha
Prostate Cancer
A Non-Interventional Study to Evaluate Time to Event and Healthcare Resource Utilisation for Prostate Cancer Participants Throughout the Disease Trajectories Until Progression or Death
Primary objective of this study is to describe the time in each prostate cancer stage from non-metastatic Hormone Sensitive Prostate Cancer (m0HSPC), metastatic Hormone Sensitive Prostate Cancer (m1HSPC), non-metastatic…
- Trial name
- NCT03619980
- Testing
- No intervention
- Where
- Sweden
- Volunteers
- 6,908 people
- Sponsor
- Astellas Pharma Europe Ltd.
Periventricular Leukomalacia, White Matter Disease
Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain.
- Conditions
- Periventricular Leukomalacia, White Matter Disease, Hypoxic-Ischemic Encephalopathy, Demyelinating Diseases
- Trial name
- NCT06985303
- Testing
- Investigational Combination Product
- Sponsor
- MGAM LLC
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Prevention of Venous Thromboembolism, Congenital Heart Disease
An Observational Study to Learn More About How Safe Rivaroxaban is And How Well it Works in Children With Congenital Heart Disease Who Had a Heart Surgery Called the Fontan Procedure
This is an observational study in which the data from children with congenital heart disease will be collected and studied. These children will include those who are prescribed rivaroxaban by their doctors after a heart surgery…
- Conditions
- Prevention of Venous Thromboembolism, Congenital Heart Disease, Fontan Procedure, Children
- Trial name
- NCT06193863
- Testing
- Rivaroxaban (Xarelto, BAY59-7939)
- Where
- Japan
- Volunteers
- 61 people
- Sponsor
- Bayer
Drug Addiction
Survey of Buprenorphine Use Among "Out of Care" Population in France (P04933)(COMPLETED)
This study was a survey of drug users who obtain buprenorphine from the black market and have used buprenorphine during the last month.
- Trial name
- NCT00756353
- Volunteers
- 600 people
- Sponsor
- Indivior Inc.
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