Clinical trials: Newborn Diseases
Choroideremia
Natural Disease Progression in Participants With Choroideremia
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants.
- Trial name
- NCT04795206
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 1,178 people
- Sponsor
- Biogen
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 people
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-A (NCT03681184)
- Testing
- Lumasiran
- Where
- 17 sites in United States, France and 6 more
- Volunteers
- 39 people
- Sponsor
- Alnylam Pharmaceuticals
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B
A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how…
- Conditions
- Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B
- Trial name
- NCT02493998
- Where
- 8 sites in United States, Australia and 6 more
- Volunteers
- 22 people
- Sponsor
- Allievex Corporation
Primary Insulin-like Growth Factor-1 Deficiency
IGFD Registry: A Patient Registry for Monitoring Long-term Safety and Efficacy of Increlex
This project is a retrospective (beginning January 2006) and prospective patient registry program sponsored by Ipsen (formerly Tercica, Inc.).
- Trial name
- NCT00747604
- Testing
- mecasermin [rDNA origin] injection
- Where
- United States
- Volunteers
- 1,378 people
- Sponsor
- Ipsen
Skin Laxity, Wrinkle
Safety and Efficacy of SofWave Treatment to Lift Lax Tissue in the Submental and Neck Zones and to Lift the Eyebrow
This is a prospective, open label, multi-center, self-controlled clinical study to demonstrate the safety and efficacy of SofWave treatment to lift lax tissue in the submental and neck zones and to lift the eyebrow.
- Conditions
- Skin Laxity, Wrinkle, Brow Lifting
- Trial name
- NCT04146584
- Testing
- Sofwave Treatments
- Where
- 5 sites in United States
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Atopic Dermatitis
Emollients in the Management of Atopic Dermatitis
The purpose of this study is to confirm that emollients play a major role in the maintenance therapy after clearing of inflammatory lesions and can reduce occurrence of flares in children with atopic dermatitis.
- Trial name
- NCT01779258
- Testing
- glycerol, paraffin (liquid and white soft), Atopiclair®, Locatop@
- Where
- 15 sites in Estonia, France and 3 more
- Volunteers
- 347 people
- Sponsor
- Pierre Fabre Medicament
Lax Skin, Improve Facial Wrinkles
In Vivo Study to Evaluate Tissue Remodeling Via Fibroblast Stimulation Following a Single SofWave Treatment
Open-label, non-randomized single-arm prospective, single-center, self-controlled clinical study.
- Trial name
- NCT07685821
- Testing
- Sofwave
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Cystic Fibrosis
Interferon Gamma-1b by Inhalation for the Treatment of Patients With Cystic Fibrosis
The purpose of this research study is to evaluate the safety and effectiveness of Interferon gamma-1b (IFN-g 1b) on lung function when given to patients with cystic fibrosis by inhalation (breathed into the lungs) three times a…
- Trial name
- NCT00043316
- Testing
- interferon gamma-1b
- Where
- 17 sites in United States
- Volunteers
- 66 people
- Sponsor
- InterMune
Cerebral Arteriovenous Malformation
Treatment of Cerebral Arteriovenous Malformations With SQUID Liquid Embolic Agent (CHOICE)
The objective of this study is to further establish that SQUID, an alternative liquid embolic agent with specific properties, is a safe and effective alternative in bAVM endovascular treatment strategy available to date.
- Trial name
- CHOICE (NCT03413852)
- Testing
- SQUID non-adhesive liquid embolic agent
- Where
- 19 sites in Belgium, Denmark and 3 more
- Volunteers
- 111 people
- Sponsor
- Embo-Flüssigkeiten A.G.
Renal Cell Carcinoma (RCC), Urothelial Carcinoma
A Study of DSP-7888 Dosing Emulsion in Combination With Immune Checkpoint Inhibitors in Adult Patients With Advanced Solid Tumors
This is a Phase 1b/2, open-label, multicenter study of DSP-7888 Dosing Emulsion in combination with checkpoint inhibitors (nivolumab or pembrolizumab) in adult patients with solid tumors, that consists of 2 parts: dose search…
- Conditions
- Renal Cell Carcinoma (RCC), Urothelial Carcinoma, Primary Peritoneal Cancer, Platinum-resistant Ovarian Cancer (PROC)
- Trial name
- NCT03311334
- Testing
- DSP-7888 Dosing Emulsion, Nivolumab, Pembrolizumab
- Where
- 19 sites in United States, Canada
- Volunteers
- 47 people
- Sponsor
- Sumitomo Pharma America, Inc.
Retinitis Pigmentosa
Prospective Natural History Study of Retinitis Pigmentosa
This is natural history study of rods and cones degenerations in patients with Retinitis Pigmentosa (RP) caused by pathogenic mutations in RHO, PDE6a or PDE6b gene mutations.
- Trial name
- PHENOROD2 (NCT04285398)
- Testing
- Ophthalmic examinations, Mobility Test
- Where
- 2 sites in United States, France
- Volunteers
- 82 people
- Sponsor
- SparingVision
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
- Trial name
- NCT03746587
- Testing
- Arimoclomol
- Where
- 8 sites in India
- Volunteers
- 39 people
- Sponsor
- ZevraDenmark
Fragile X Syndrome
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have…
- Trial name
- NCT01348087
- Testing
- AFQ056
- Where
- 28 sites in United States, Australia and 8 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
A Study to Evaluate the Safety of Lebrikizumab Compared to Topical Corticosteroids in Adult Patients With Atopic Dermatitis
The primary objective for this study is to evaluate the safety of lebrikizumab compared with Topical Corticosteroids (TCS) alone in patients with persistent moderate to severe Atopic Dermatitis (AD) that is inadequately…
- Trial name
- NCT02465606
- Testing
- Lebrikizumab, Topical Corticosteroid Creams (Triamcinolone acetonide 0.1% and Hydrocortisone 2.5%)
- Where
- 19 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency
The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects with A1PI deficiency and emphysema/ chronic…
- Trial name
- NCT02722304
- Testing
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 7 people
- Sponsor
- Baxalta now part of Shire
Fabry Disease
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks.
- Trial name
- NCT04974749
- Testing
- REPLAGAL
- Where
- 6 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Preterm Labor, Preterm Birth
MIRACLE of LIFE Study
The goal of this observational study is to develop and validate cell-free RNA-based biomarkers for predicting a variety of adverse pregnancy outcomes in a pregnant person population. The main question it aims to answer are: 1.
- Conditions
- Preterm Labor, Preterm Birth, Preterm Birth Complication, Preterm Premature Rupture of Membrane
- Trial name
- MoL (NCT06074601)
- Testing
- Mirvie Predictive Test for Adverse Pregnancy Outcomes
- Where
- 10 sites in United States
- Volunteers
- 10,000 people
- Sponsor
- Mirvie
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
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