Clinical trials: Tolerability
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
Infantile Colic, Infantile Functional Gastrointestinal Disorders
A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants
The purpose of the study is to evaluate the gastrointestinal absorption of nepadutant after single dose as oral solution (and the effect of age on its oral absorption) in infants.
- Trial name
- NCT00655083
- Testing
- Nepadutant
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Menarini Group
Pneumococcal Infections
A Study to Evaluate the Interchangeability of V114 and Prevnar 13™ in Healthy Infants (V114-027/PNEU-DIRECTION)
The goal of this study is to evaluate the safety, tolerability, and immunogenicity of the Pneumococcal Conjugate Vaccines (PCVs) V114 and Prevnar 13™ in healthy infants switched from Prevnar 13™ to V114 during the four-dose PCV…
- Trial name
- NCT03620162
- Testing
- Prevnar 13™, V114, RotaTeq™
- Where
- 34 sites in United States, Puerto Rico and 2 more
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Dyskinesia, Cerebral Palsy
Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
The primary objective of this study is to evaluate the efficacy of valbenazine versus placebo on improving chorea in pediatric and adult participants who have dyskinesia due to cerebral palsy (DCP) with choreiform movements.
- Trial name
- NCT05206513
- Testing
- Valbenazine
- Where
- 38 sites in United States, Argentina and 8 more
- Volunteers
- 86 people
- Sponsor
- Neurocrine Biosciences
Cervical Cancer, Vulvar Cancer
Immunogenicity and Tolerability of Broad Spectrum Human Papillomavirus (HPV) Vaccine in Adult and Young Adult Women (V503-004)
This study will assess the safety and immunogenicity of GARDASIL®9 (V503) in 16- to 45-year-old women. The primary hypothesis of the study states that anti-HPV 16, 18, 31, 33, 45, 52, and 58 geometric mean titers (GMTs) at 4…
- Conditions
- Cervical Cancer, Vulvar Cancer, Vaginal Cancer, Genital Warts
- Trial name
- NCT03158220
- Testing
- V503
- Where
- 24 sites in Austria, Belgium and 4 more
- Volunteers
- 1,212 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Thalassemia
Short-term Clinical Study of CN128 in Thalassemia Patients
1. Primary objectives: To study the tolerance and safety of multiple oral administration of CN128 in patients with thalassemia aged 16 years and above.
- Trial name
- NCT03935633
- Testing
- CN128 Tablets
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.
Pulmonary Arterial Hypertension
Effects of Two Dosing Regimens of Bosentan in Children With Pulmonary Arterial Hypertension
The primary objective of AC-052-373 was to assess the pharmacokinetic (PK) profile of two dosing regimens of the pediatric formulation of bosentan in children with pulmonary arterial hypertension (PAH) <12 years of age.
- Trial name
- FUTURE 3 (NCT01223352)
- Testing
- bosentan
- Volunteers
- 64 people
- Sponsor
- Actelion
Charcot-Marie-Tooth Disease
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period. -Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be…
- Trial name
- PREMIER (NCT04762758)
- Testing
- (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 350 people
- Sponsor
- Pharnext S.C.A.
Epilepsy
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
This is a phase 2, crossover study of Ataluren for the treatment of nonsense mutation Dravet syndrome or cyclin-dependent kinase-like 5 (CDKL5) deficiency, resulting in drug-resistant epilepsy.
- Trial name
- NCT02758626
- Testing
- ataluren
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
NAFLD, Non Alcoholic Steatohepatitis (NASH)
DHA and Vitamin D in Children With Biopsy-proven NAFLD
Non-alcoholic fatty liver disease (NAFLD) has reached epidemic proportions and is rapidly becoming the one of most common causes of chronic liver disease in children.
- Trial name
- VitD_DHA (NCT02098317)
- Testing
- DHA plus Vitamin D
- Where
- Italy
- Volunteers
- 66 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
Primary Immunodeficiency Diseases (PID)
Phase 2/3 Study of IGSC, 20% in PIDD
The purpose of this study is to develop a 20% subcutaneous (SC) immunoglobulin preparation for the treatment of patients with primary immunodeficiency diseases (PIDD).
- Trial name
- NCT01218438
- Testing
- Immune Globulin Intravenous (Human), 10% Solution, Immune Globulin Subcutaneous (Human), 20% Solution
- Where
- 18 sites in United States, Canada
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
Postmenopause
Effects of Wellnara on Climacteric Symptoms
Aim of this NIS is to obtain further information on efficacy, tolerability, and acceptance of Wellnara in a large user population under the conditions of routine medical practice.
- Trial name
- NCT00913926
- Testing
- E2/LNG oral (Wellnara, BAY86-5029)
- Where
- Germany
- Volunteers
- 749 people
- Sponsor
- Bayer
Breast Cancer
Anti-tumour Effects & Tolerability of Faslodex Alone or in Combination With Arimidex in Post Menopausal Women Prior to Surgery for Primary Breast Cancer
To compare the anti-tumour effects as measured by changes in various biomarkers, of a combination of Faslodex and Arimidex with Faslodex alone and Arimidex alone in postmenopausal women patients with primary breast cancer who are…
- Trial name
- NCT00259090
- Testing
- Fulvestrant, Anastrazole
- Where
- United Kingdom
- Volunteers
- 120 people
- Sponsor
- AstraZeneca
Limb-Girdle Muscular Dystrophy, Type 2E
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Trial name
- NCT03652259
- Testing
- SRP-9003
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Sickle Cell Disease
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell Disease
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
- Trial name
- NCT00842088
- Testing
- HQK-1001
- Where
- 9 sites in United States, Jamaica
- Volunteers
- 24 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Turner Syndrome
A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome
A 104 week dose finding open label trial followed by an optional 78 week open label extension of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus daily somatropin product in prepubertal…
- Trial name
- NCT05690386
- Testing
- Lonapegsomatropin, Somatropin
- Where
- 19 sites in United States
- Volunteers
- 48 people
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Fetal Alcohol Syndrome, Attention Deficit Hyperactivity Disorder (ADHD)
Open-Label Study of the Long Term Tolerability and Safety of Atomoxetine in Children With FASD and ADD/ADHD
Determine if atomoxetine is safe and well tolerated by children with fetal alcohol syndrome.
- Trial name
- B4Z-US-050 (NCT00418262)
- Testing
- Atomoxetine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Oklahoma
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Cystic Fibrosis
Safety, Pharmacokinetic and Pharmacodynamic Study of MP-376 in Patients With Cystic Fibrosis
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT00503490
- Testing
- MP-376 (Levofloxacin solution for Inhalation)
- Where
- 14 sites in United States
- Volunteers
- 40 people
- Sponsor
- Amgen
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