Clinical trials: Congenital Disorder
Gaucher Disease
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00376168
- Testing
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 32 people
- Sponsor
- Pfizer
Alagille Syndrome (ALGS)
A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time.
- Trial name
- NCT05543174
- Testing
- TAK-625
- Where
- 8 sites in Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
Fetal Growth Retardation
MAXOMAT ® in the Treatment of Severe Early Onset Intrauterine Growth Retardation on Pre-pubertal Children
1. To test for equivalence in terms of catch-up growth between the 2 therapeutic regimens 2. To specify the best period of treatment 3. To assess the efficacy of treatment based on final adult height of these children
- Trial name
- NCT00452491
- Testing
- somatropin
- Where
- France
- Volunteers
- 306 people
- Sponsor
- Sanofi
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Metastatic Castration-resistant Prostate Cancer
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castration-resistant Prostate Cancer.
- Trial name
- rechARge (NCT06764485)
- Testing
- BMS-986365, Enzalutamide, Abiraterone
- Where
- 227 sites in United States, Argentina and 24 more
- Volunteers
- 960 people
- Sponsor
- Celgene
ROSAH
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
The purpose of this study is to evaluate the safety and tolerability of DF-003 in retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome patients.
- Trial name
- NCT06395285
- Testing
- DF-003
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 12 people
- Sponsor
- Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
GSD1
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
- Trial name
- NCT03517085
- Testing
- DTX401, steroid regimen
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Atopic Dermatitis
Long-term Safety and Efficacy of Lebrikizumab in Adult and Adolescent Participant With Moderate-to-Severe Atopic Dermatitis
The main purpose of the study is to assess the long-term tolerability and effectiveness of lebrikizumab in adult and adolescent participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- ADlong (NCT05916365)
- Testing
- Lebrikizumab
- Where
- 32 sites in Germany, Poland
- Volunteers
- 174 people
- Sponsor
- Almirall, S.A.
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia
- Trial name
- NCT05353192
- Testing
- Recombinant human growth hormone
- Where
- China
- Volunteers
- 39 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Alopecia Areata, Vitiligo
A Pilot Cohort Study of Risk Factors and Novel Methods of Skin Lesion Assessments in Adults With Atopic Dermatitis, Alopecia Areata, Psoriasis or Vitiligo
The objective of the study is to explore various clinical and biochemical parameters and their potential associations with disease severity, activity, and prognosis in atopic dermatitis, psoriasis, alopecia areata, and vitiligo.
- Conditions
- Alopecia Areata, Vitiligo, Atopic Dermatitis, Psoriasis
- Trial name
- Derma-001 (NCT06319781)
- Testing
- Observational
- Where
- 3 sites in Denmark
- Volunteers
- 370 people
- Sponsor
- Blueskin AS
Friedreich's Ataxia
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
In this study, researchers will learn more about a disease called Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their…
- Trial name
- NCT07887828
- Volunteers
- 40 people
- Sponsor
- Biogen
Healthy Volunteer, Down Syndrome
A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662
This single-center, single-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down…
- Trial name
- NCT01667367
- Testing
- RG1662
- Where
- United Kingdom
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Hemophilia A
Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A
An open-label, single dose pharmacokinetic study of Xyntha (Moroctocog Alfa (AF-CC), Recombinant Factor VIII) in male Chinese subjects with hemophilia A
- Trial name
- NCT02461992
- Testing
- Intravenous infusions of Xyntha
- Where
- 2 sites in China
- Volunteers
- 13 people
- Sponsor
- Pfizer
Angelman Syndrome, Fragile X Syndrome
Single Dose Pharmacokinetic (PK) Study
The trial is a Phase 1 Single Dose PK Study in Adolescent Subjects with Fragile X syndrome (FXS) or Angelman syndrome (AS). The primary objective of the study is to evaluate the pharmacokinetics (PK) of OV101 following a single 5…
- Trial name
- NCT03109756
- Testing
- OV101
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ovid Therapeutics Inc.
Skin Laxity, Muscle Contracture
Evaluation of the Immediate Effect and Safety of DMA™ Novel Muscle Stimulation Technology
A total of at least 15 and up to 17 healthy subjects at a single site, aged 25-45 years old, who wish to receive a lifting effect and improved muscle definition, will be included in the study.
- Trial name
- NCT04296201
- Testing
- DMA treatment
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Lumenis Be Ltd.
Tumor or Lymphoma
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of JNJ-42756493 in Adult Participants With Advanced or Refractory Solid Tumors or Lymphoma
The purpose of this study is to evaluate the safety, pharmacokinetics (study of what the body does to a drug), and pharmacodynamics (study of what a drug does to the body) of JNJ-42756493, a pan-fibroblast growth factor receptor…
- Trial name
- NCT01703481
- Testing
- JNJ-42756493: Part 1, JNJ-42756493: Part 2, JNJ-42756493: Part 3
- Where
- 37 sites in United States, France and 1 more
- Volunteers
- 188 people
- Sponsor
- Janssen Research & Development, LLC
Allergy, Dermatitis
Randomized Study for Effectiveness and Safety Evaluation of Dexamethasone 0.5 mg + Fumarate Clemastine 1 mg Compared to Dexamethasone 0.5 mg in Patients With Allergic Dermatitis
The aim of this study is to prove the efficacy of the dexamethasone 0.5 mg + 1 mg clemastine fumarate tablet compared to 0.5 mg of dexamethasone in reducing the signs and symptoms of allergic dermatitis.
- Trial name
- NCT01239719
- Testing
- Dexamethasone + clemastine, Dexamethasone
- Volunteers
- 96 people
- Sponsor
- Azidus Brasil
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