Clinical trials: Genetic Diseases
Hypercholesterolemia, Familial
Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 (mipomersen) as add-on therapy in subjects with Heterozygous Familial Hypercholesterolemia
- Trial name
- NCT00281008
- Where
- 8 sites in United States, Netherlands
- Volunteers
- 44 people
- Sponsor
- Kastle Therapeutics, LLC
Arthritis, Gouty
Celebrex In Acute Gouty Arthritis Study
This is a multicenter, double-blind, double-dummy, randomized, active-controlled study that will include an 8-day treatment period followed by a 1-week follow-up period in patients experiencing symptoms of an acute exacerbation…
- Trial name
- NCT00549549
- Testing
- Indomethacin, Celecoxib
- Where
- 81 sites in United States, Canada and 10 more
- Volunteers
- 402 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Fabry Disease
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration.
- Trial name
- NCT04519749
- Testing
- 4D-310
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- 4D Molecular Therapeutics
Thalassemia
A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)
This study is a multicenter study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of treatment with SNH-119014 in adult participants with non-transfusion-dependent thalassemia.
- Trial name
- NCT07579949
- Testing
- SNH-119014 dose 1, SNH-119014 dose 2
- Where
- 8 sites in China
- Volunteers
- 30 people
- Sponsor
- ScinnoHub Pharmaceutical Co., Ltd.
Gaucher Disease
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00478647
- Testing
- GA-GCB (velaglucerase alfa)
- Where
- 15 sites in United States, Israel and 3 more
- Volunteers
- 40 people
- Sponsor
- Shire
Type 1 Diabetes Mellitus
A Phase III Study to Investigate if the Study Drug Diamyd Can Preserve Insulin Production and Improve Glycemic Control in Patients Newly Diagnosed With Type 1 Diabetes
The objective of DIAGNODE-3 is to evaluate the efficacy and safety of three intranodal injections of 4 μg of Diamyd also known as retogatein compared to placebo, along with oral Vitamin D supplementation, to preserve endogenous…
- Trial name
- DIAGNODE-3 (NCT05018585)
- Testing
- Recombinant human glutamic acid decarboxylase (rhGAD65) formulated in Alhydrogel®, Colecalciferol 2000 IU
- Where
- 60 sites in United States, Czechia and 7 more
- Volunteers
- 321 people
- Sponsor
- Diamyd Medical AB
Infertility, Recurrent Pregnancy Loss
Preimplantation Genetic Diagnosis Using Blastocyst Biopsy and Array CGH
The investigators propose to perform a clinical randomized trial to evaluate the effect of single embryo (blastocyst) transfer (SET) with array CGH for the evaluation of the complete chromosome complement of the blastocyst in…
- Trial name
- NCT01546350
- Testing
- PGD
- Where
- 4 sites in United States
- Volunteers
- 200 people
- Sponsor
- Reprogenetics
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Dermatitis, Atopic
Evaluation of 611 in Chinese Adolescents With Moderate to Severe Atopic Dermatitis
The primary objective of the study was to evaluate the efficacy of 611 in Chinese Adolescents with moderate to severe atopic dermatitis.
- Trial name
- NCT07042126
- Testing
- 611
- Where
- 3 sites in China
- Volunteers
- 180 people
- Sponsor
- Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd.
Sickle Cell Disease
A 3-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients
A long term observational study in sickle cell disease will enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease.
- Trial name
- FISCO (NCT01220115)
- Where
- 42 sites in United States
- Volunteers
- 498 people
- Sponsor
- Novartis
DCild Development Disorders, Pervasive, Autistic Disorder
A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD)
The purpose of the study is to evaluate the effectiveness of an oral solution of risperidone (an antipsychotic medication) versus placebo in the treatment of behavioral symptoms in children with Pervasive Developmental Disorders…
- Conditions
- DCild Development Disorders, Pervasive, Autistic Disorder, Developmental Disabilities, Asperger Syndrome
- Trial name
- NCT00261508
- Testing
- risperidone
- Volunteers
- 80 people
- Sponsor
- Janssen-Ortho Inc., Canada
Cervical Cancer
Bintrafusp Alfa Combination Therapy in Participants With Cervical Cancer (INTR@PID 046)
This study was to evaluate the safety and tolerability of bintrafusp alfa in combination with other anti-cancer therapies in participants with locally advanced or advanced cervical cancer.
- Trial name
- NCT04551950
- Testing
- M7824, Carboplatin, Paclitaxel
- Where
- 13 sites in United States, Japan and 1 more
- Volunteers
- 25 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Atopic Dermatitis
A Study of QY201 Tablet in Subjects With Moderate to Severe Atopic Dermatitis
This is a phase Ib/II, randomized, double-blind, placebo-controlled, parallel, multicenter study of a certain phase to evaluate the efficacy, safety, and pharmacokinetic characteristics of QY201 tablet in subjects in moderate to…
- Trial name
- NCT05525715
- Volunteers
- 260 people
- Sponsor
- E-nitiate Biopharmaceuticals (Hangzhou) Co., Ltd.
Osteochondrodysplasia
Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia
This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.
- Trial name
- upreACH-2 (NCT06067425)
- Testing
- SAR442501
- Where
- 9 sites in Australia, China and 3 more
- Volunteers
- 16 people
- Sponsor
- Sanofi
Atopic Dermatitis
A Phase II Clinical Study Evaluating of ZL-82 Tablets in Atopic Dermatitis
This clinical trial aims to explore whether the drug ZL-82 tablets can be used to treat moderate to severe atopic dermatitis in adults, and to understand the safety and tolerability of the drug.
- Trial name
- NCT07193589
- Testing
- The high-dose group of ZL-82 tablets, The low-dose group of ZL-82 tablets
- Where
- China
- Volunteers
- 111 people
- Sponsor
- Chengdu Zenitar Biomedical Technology Co., Ltd
Irritable Bowel Syndrome
Multicenter Observational Prospective Study to Describe Irritable Bowel Syndrome in the Russian Federation
Observational Prospective Study to Describe Irritable Bowel Syndrome in the Russian Federation
- Trial name
- ROMERUS (NCT03549143)
- Testing
- IBS
- Where
- 37 sites in Russia
- Volunteers
- 1,000 people
- Sponsor
- Abbott
Healthy Volunteers
Study to Access the Relative Bioavailability of Subcutaneous Dose of Nemolizumab When Administered Via Auto-Injector Versus Dual-Chamber Syringe
This study was to compare the rate and extent of absorption of a single dose of nemolizumab administered with auto-injectors [AI] (test) versus dual-chamber syringes [DCS] (reference) under controlled conditions in healthy adult…
- Trial name
- NCT05405985
- Testing
- Nemolizumab
- Where
- 2 sites in United States
- Volunteers
- 192 people, healthy volunteers welcome
- Sponsor
- Galderma R&D
Huntington Disease
Dose-response Evaluation of the Cellavita HD Product in Patients With Huntington's Disease
Cellavita HD is a stem-cell therapy for Huntington's Disease. This is a prospective, phase II, single-center, randomized (2:2:1), triple-blind, placebo controlled study, with two test doses of Cellavita HD product.
- Trial name
- ADORE-DH (NCT03252535)
- Testing
- Cellavita HD lower dose, Cellavita HD higher dose
- Where
- Brazil
- Volunteers
- 49 people
- Sponsor
- Azidus Brasil
Phenylketonurias
Evaluation of PKU Sphere
A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.
- Trial name
- NCT03419819
- Testing
- PKU Sphere
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Vitaflo International, Ltd
Hypophosphatasia
A Prospective Sub-Study of the Global Hypophosphatasia Registry
In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and…
- Trial name
- NCT05234567
- Testing
- Asfotase Alfa
- Where
- 12 sites in United States
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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