Clinical trials: Newborn Diseases
Duchenne Muscular Dystrophy
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies.
- Trial name
- NCT05689164
- Testing
- fordadistrogene movaparvovec
- Where
- 12 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pfizer
Type II Hyperlipidemia
A Safety and Efficacy Study of DRL-17822, a Cholesteryl Ester Transfer Protein (CETP) Inhibitor, in Patients With Abnormal Cholesterol Levels
The purpose of this study is to determine if a new drug, DRL-17822, is safe and effective in elevating high density lipoprotein cholesterol (HDL-C) and reducing low density lipoprotein cholesterol (LDL-C) in people with abnormal…
- Trial name
- NCT01388816
- Where
- 12 sites in Italy, Poland and 1 more
- Volunteers
- 176 people
- Sponsor
- Dr. Reddy's Laboratories Limited
Atopic Dermatitis
A Study of Upadacitinib in Adult Participants With Moderate-to-Severe Atopic Dermatitis and Inadequate Response to Dupilumab
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Therapies spread over the skin may not be enough to control the AD in trial participants who require systemic…
- Trial name
- Switch-Up (NCT06389136)
- Testing
- Upadacitinib 15mg Dose, Dupilumab 300mg Dose, Upadacitinib 30mg Dose
- Where
- 129 sites in United States, Canada and 7 more
- Volunteers
- 184 people
- Sponsor
- AbbVie
Hemophilia A
Study Evaluating ReFacto AF in Severe Hemophilia A
To characterize the safety and efficacy of ReFacto AF in treating acute bleeding episodes during prophylaxis treatment, including neoantigenicity.
- Trial name
- NCT00037544
- Testing
- ReFacto AF
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Cystic Fibrosis
MP-376 (Aeroquin™, Levofloxacin for Inhalation) 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
- NCT01180634
- Testing
- Aeroquin
- Where
- 112 sites in United States, Australia and 3 more
- Volunteers
- 330 people
- Sponsor
- Amgen
Phenylketonuria, Hyperphenylalaninaemia
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
The objective of this study is to evaluate the safety of long-term treatment with Kuvan.
- Trial name
- PKUDOS (NCT00778206)
- Testing
- Kuvan
- Where
- 45 sites in United States
- Volunteers
- 1,887 people
- Sponsor
- BioMarin Pharmaceutical
Patent Ductus Arteriosus
AMPLATZER Duct Occluder II Clinical Study
The objective of this study is to investigate the safety and effectiveness of the ADO II in patients with a PDA.
- Trial name
- ADO II (NCT00713700)
- Testing
- AMPLATZER Duct Occluder II
- Where
- 25 sites in United States
- Volunteers
- 192 people
- Sponsor
- Abbott Medical Devices
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Pediatric Growth Hormone Deficiency
Versartis Long-Term Safety Study of Somavaratan
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study.
- Trial name
- VISTA (NCT02068521)
- Testing
- somavaratan
- Volunteers
- 385 people
- Sponsor
- Versartis Inc.
Angelman Syndrome
A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.
This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion AS aged 5-17 years (inclusive) will be enrolled in the study.
- Trial name
- Aldebaran (NCT05630066)
- Testing
- Alogabat
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- Hoffmann-La Roche
Healthy, Atopic Dermatitis
A Study to Test Safety, Tolerability, and the Way the Body Absorbs, Distributes, and Gets Rid of a Study Drug Called MOR106, in Healthy Subjects and in Patients With Moderate to Severe Atopic Dermatitis
The clinical study consists of three parts: Part 1 with healthy volunteers. Part 2 and Part 3 including subjects with moderate to severe atopic dermatitis (a skin disease).
- Trial name
- NCT03689829
- Testing
- MOR106
- Where
- 15 sites in Germany, Spain and 2 more
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Lakefront Biotherapeutics NV
Hereditary Tyrosinemia, Type I
Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
The purpose of this study is to look at the steady-state serum concentrations of nitisinone when switching from twice daily and once daily dosing.
- Trial name
- HT-1 (NCT02323529)
- Testing
- Nitisinone
- Where
- 6 sites in Belgium, Denmark and 3 more
- Volunteers
- 18 people
- Sponsor
- Swedish Orphan Biovitrum
Familial Chylomicronemia Syndrome
Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT06360237
- Testing
- Olezarsen
- Where
- United States
- Sponsor
- Ionis Pharmaceuticals, Inc.
Retinitis Pigmentosa
Role of UC-MSC and CM to Inhibit Vision Loss in Retinitis Pigmentosa Phase I/II
The study will perform UC-MSCs and CM transplantation. There are two groups with different dosages. The first group will be transplanted with 1.5 million cells, meanwhile, the second group is 5 million cells.
- Trial name
- NCT05909488
- Testing
- 1.5 x 10^6 UC-MSC + CM, 5 x 10^6 UC-MSC + CM
- Where
- Indonesia
- Volunteers
- 30 people
- Sponsor
- PT. Prodia Stem Cell Indonesia
Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe steatoblepharon (excess eyelid fat).
- Trial name
- NCT02230761
- Testing
- XOPH5 Ointment
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Topokine Therapeutics, Inc.
Retinitis Pigmentosa
Promising ROd-cone DYstrophy Gene therapY
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a…
- Trial name
- PRODYGY (NCT05748873)
- Testing
- SPVN06
- Where
- 6 sites in United States, France
- Volunteers
- 33 people
- Sponsor
- SparingVision
Chronic Nasal Congestion, Lung Infection
Biomarker for Cystic Fibrosis
International, multicenter, observational, longitudinal study to identify biomarker/s for Cystic fibrosis and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Conditions
- Chronic Nasal Congestion, Lung Infection, Breathlessness, Clubbing Toes
- Trial name
- BioCyFi (NCT02710383)
- Where
- 5 sites in Albania, Georgia and 3 more
- Volunteers
- 54 people
- Sponsor
- CENTOGENE GmbH Rostock
Dermatitis, Atopic
Functional Study to Investigate the Efficacy of a New Medical Device (Modified Diprobase Formulation)
This clinical trial is intended to investigate the efficacy and safety of a medical device compared to untreated on patients with quiescent atopic dermatitis.
- Trial name
- NCT03672383
- Testing
- BAY987534
- Where
- Germany
- Volunteers
- 42 people
- Sponsor
- Bayer
Tourette Syndrome, Motor Tic Disorder
Study of ABX-1431 in Adult Patients With Tourette Syndrome or Chronic Motor Tic Disorder
Two-part study consisting of a double-blind, randomized, placebo-controlled, study at two target dose levels (Part 1) and an open-label, non-randomized study (Part 2) to determine the efficacy of ABX-1431 in treating adult…
- Trial name
- NCT03625453
- Testing
- ABX-1431
- Where
- 8 sites in Germany, Poland and 1 more
- Volunteers
- 50 people
- Sponsor
- Abide Therapeutics
Osteogenesis Imperfecta
Post-market Observational Study on JTIN Telescopic Nail in Osteogenesis Imperfecta Pediatric Patients
This study has been planned as part of the Orthofix Srl post-market active surveillance plan for the collection of data on both the clinical performance and the safety profile of the JTIN.
- Trial name
- JTIN (NCT05612139)
- Testing
- JTIN implantation surgery
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Orthofix s.r.l.
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