Clinical trials: Newborn Diseases
Cystic Fibrosis
A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation
- Trial name
- NCT02742519
- Testing
- ivacaftor
- Where
- 6 sites in Australia, Canada and 1 more
- Volunteers
- 14 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
End Stage Renal Disease, AV Fistula
Percutaneous Less Invasive AV Fistula for Vascular Access in ESRD
A prospective single-arm well-controlled study to evaluate the safety and effectiveness of a less invasive means of establishing vascular access to facilitate dialysis in patients with end stage renal disease.
- Conditions
- End Stage Renal Disease, AV Fistula, Fistula, End Stage Kidney Disease
- Trial name
- NCT02363972
- Testing
- Ellipsys Vascular Access Catheter
- Where
- 5 sites in United States
- Volunteers
- 103 people
- Sponsor
- Medtronic Endovascular
Dermatitis, Atopic, Dermatitis Eczema
A Study To Evaluate ASN002 In Subjects With Atopic Dermatitis
This is a dose escalation study to test the safety, tolerability and preliminary efficacy of ASN002 in people with moderate to severe atopic dermatitis (AD).
- Conditions
- Dermatitis, Atopic, Dermatitis Eczema, Dermatitis, Eczematous
- Trial name
- NCT03139981
- Testing
- ASN002
- Where
- 10 sites in United States, Canada
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Asana BioSciences
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
Alagille Syndrome
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and…
- Trial name
- ITCH (NCT02057692)
- Testing
- LUM001
- Where
- 13 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Trisomy 13, Trisomy 18
High Risk Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our proprietary algorithm method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278874
- Where
- 4 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Atopic Dermatitis (AD)
A Study to Explore the Safety, Tolerability, Efficacy, and Pharmacokinetics of LT-002-158 Tablets in Chinese Adult Subjects With Atopic Dermatitis
This study is an Ic/II phase clinical trial conducted in Chinese subjects with moderate to severe atopic dermatitis. The phase Ic study is a randomized, open label, parallel group design, mainly evaluating the safety and…
- Trial name
- NCT06931990
- Testing
- LT-002-158 tablets
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Leadingtac Pharmaceutical (Shaoxing) Co., Ltd.
Respiratory Distress Syndrome
The Safety and Efficacy of Lucinactant for Inhalation in Premature Neonates 26 to 32 Weeks Gestational Age
The primary objective of this study is to evaluate the safety and efficacy of lucinactant for inhalation administered as an aerosolized dose in two doses to preterm neonates 26 - 32 weeks gestational age who are receiving nasal…
- Trial name
- NCT02636868
- Testing
- Lucinactant delivered via investigational delivery device, nCPAP
- Where
- 54 sites in United States, Canada and 6 more
- Volunteers
- 221 people
- Sponsor
- Windtree Therapeutics
Rett Syndrome
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 5-17 years of age with RTT using endpoints including multiple clinical and…
- Trial name
- EXCELLENCE (NCT04304482)
- Testing
- ANAVEX2-73 oral liquid
- Where
- 12 sites in Australia, Canada and 1 more
- Volunteers
- 92 people
- Sponsor
- Anavex Life Sciences Corp.
Hemophilia A
Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe…
- Trial name
- NCT03947320
- Testing
- Recombinant Human Coagulation FVIII
- Volunteers
- 70 people
- Sponsor
- Sinocelltech Ltd.
Gastroesophageal Reflux Disease (GERD)
Phase 3/Safety & Efficacy of Esomeprazole in Infants
The purpose of this study is to look at the improvement of a once a day dose of esomeprazole for reducing the signs and symptoms of infants with gastroesophageal reflux disease (GERD).
- Trial name
- NCT00468559
- Testing
- Open Label Run In Esomeprazole, Double Blind Esomeprazole
- Where
- 25 sites in United States, France and 2 more
- Volunteers
- 98 people
- Sponsor
- AstraZeneca
Atopic Dermatitis
A Phase II Study of HY209 Gel for Atopic Dermatitis Patients (Shaperon)
A Randomized, Double-blinded, Placebo-controlled, Parallel, Multi-Center Phase II Clinical Study to Evaluate the Efficacy and Safety of HY209 gel for Patients with Atopic Dermatitis
- Trial name
- NCT04530643
- Testing
- HY209 0.3%, HY209 0.5%
- Where
- 3 sites in South Korea
- Volunteers
- 80 people
- Sponsor
- Shaperon
Gastrointestinal Stromal Tumors, Small-cell Lung Cancer
A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit
This open-label clinical trial will evaluate the safety and tolerability of NN3201 in subjects with advanced and/or metastatic solid tumors known to express c-Kit.
- Conditions
- Gastrointestinal Stromal Tumors, Small-cell Lung Cancer, Adenoid Cystic Carcinoma, Uveal Melanoma
- Trial name
- NCT06805825
- Testing
- NN3201
- Where
- 5 sites in United States
- Volunteers
- 67 people
- Sponsor
- Novelty Nobility, Inc.
Hereditary Angioedema
CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks
This is a multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic treatment of hereditary…
- Trial name
- NCT04656418
- Testing
- CSL312
- Where
- 28 sites in United States, Canada and 5 more
- Volunteers
- 64 people
- Sponsor
- CSL Behring
Atopic Dermatitis
A Clinical Trial in Subjects With Atopic Dermatitis (Part 1)
A Phase 2, multicenter, adaptive 2-part clinical trial designed to evaluate the safety and efficacy of ADX-629 alone and in combination with standard-of-care in adults with atopic dermatitis.
- Trial name
- NCT05717920
- Testing
- ADX-629 (Open-label)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Aldeyra Therapeutics, Inc.
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
A Phase 2b Study of Zagociguat in Patients With MELAS
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs.
- Trial name
- PRIZM (NCT06402123)
- Testing
- zagociguat 15mg, zagociguat 30mg
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 43 people
- Sponsor
- Tisento Therapeutics
PCOS (Polycystic Ovary Syndrome) of Bilateral Ovaries
Effect of Hyperandrogenism on IVF Outcomes in PCOS Patients
Ovulatory dysfunction affects 18 to 25% of infertile women, the most common identifiable condition is polycystic ovarian syndrome (PCOS).
- Trial name
- HIP (NCT05555680)
- Testing
- Ferriman-Gallwey Score
- Where
- Canada
- Volunteers
- 32 people
- Sponsor
- Clinique Ovo
Spinocerebellar Ataxia Type 3
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3).
- Trial name
- NCT05160558
- Testing
- BIIB132
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 8 people
- Sponsor
- Biogen
Retinopathy of Prematurity (ROP)
Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy
The purpose of this study is to demonstrate how well aflibercept works in babies with ROP, comparing it with laser therapy. The study also has the objective to demonstrate how safe aflibercept is when used in babies, and describe…
- Trial name
- FIREFLEYE (NCT04004208)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 90 sites in Argentina, Austria and 25 more
- Volunteers
- 113 people
- Sponsor
- Bayer
Colic
Human Milk Oligosaccharide (HMO) Supplementation in Colic Management
Efficacy and tolerability of a composition comprising of HMO in a supplement format on colic management: a double-blind, randomized, placebo-controlled trial
- Trial name
- NCT05554991
- Testing
- HMO, Parental reassurance and support
- Where
- 7 sites in Italy, Spain
- Volunteers
- 5 people
- Sponsor
- Société des Produits Nestlé (SPN)
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