Clinical trials: Newborn Diseases
MPS IV A
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
- Trial name
- NCT01275066
- Testing
- BMN 110 Weekly, BMN 110 Every Other Week
- Where
- 28 sites in United States, Argentina and 15 more
- Volunteers
- 177 people
- Sponsor
- BioMarin Pharmaceutical
Ankyloglossia
Case Series of Infants With Anterior Ankyloglossia Being Treated by the Sublingual Neonatal Incision Platform Device
A study assessing the feasibility of a novel device designed specifically to treat infant tongue-tie.
- Trial name
- SNIP (NCT07655895)
- Testing
- Frenectomy of anterior ankylogossia using the novel device
- Where
- United Kingdom
- Volunteers
- 6 people
- Sponsor
- Europa Life Sciences Limited
Gout
A Study of Dotinurad Versus Allopurinol in Participants With Gout
The primary objective of this study is to evaluate the efficacy of dotinurad in lowering serum uric acid (sUA) at Week 24 compared with allopurinol in adult participants with hyperuricemia associated with gout.
- Trial name
- NCT07089875
- Testing
- Allopurinol, Dotinurad
- Where
- 119 sites in United States, Poland and 1 more
- Volunteers
- 500 people
- Sponsor
- Crystalys Therapeutics
Psoriasis, Atopic Dermatitis
A Study of EDP1066 in Healthy Participants and Participants With Mild to Moderate Psoriasis and Atopic Dermatitis
Evelo will investigate the safety and tolerability of EDP1066 and its potential to be a medicinal product in healthy volunteers and individuals with mild to moderate psoriasis and atopic dermatitis.
- Trial name
- NCT03542994
- Testing
- EDP1066
- Where
- 7 sites in United Kingdom
- Volunteers
- 114 people, healthy volunteers welcome
- Sponsor
- Evelo Biosciences, Inc.
Acute Intermittent Porphyria
Porphozym in the Treatment of Acute Attacks in AIP
A multi-centre, double-blind, randomized, placebo controlled, parallel group trial, investigating the efficacy and safety of Porphozym (recombinant human porphobilinogen deaminase)in the treatment of acute attacks in AIP.
- Trial name
- NCT00418795
- Testing
- recombinant human porphobilinogen deaminase (Porphozym)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Zymenex A/S
Angioedema
Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema
The purpose of this study is to assess the efficacy and safety of Icatibant, a bradykinin antagonist in the treatment of acute cutaneous and/or abdominal attacks in patients with hereditary angioedema (HAE).
- Trial name
- NCT00097695
- Testing
- Icatibant
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- Shire
Thyroid Eye Disease
A Safety, Tolerability, and Efficacy Study of Veligrotug (VRDN 001) in Participants With Thyroid Eye Disease (TED)
The investigational drug, veligrotug (VRDN-001), is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R).
- Trial name
- THRIVE (NCT05176639)
- Testing
- Veligrotug
- Where
- 53 sites in United States, Australia and 5 more
- Volunteers
- 113 people
- Sponsor
- Viridian Therapeutics, Inc.
Leber Hereditary Optic Neuropathy
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
- Trial name
- NCT02064569
- Testing
- GS010
- Where
- France
- Volunteers
- 19 people
- Sponsor
- GenSight Biologics
Thrombocytopenia, Neonatal Alloimmune
A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
The purpose of this study is to evaluate the effectiveness of nipocalimab compared with placebo in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).
- Trial name
- FREESIA-1 (NCT06449651)
- Testing
- Nipocalimab
- Where
- 21 sites in Belgium, Brazil and 10 more
- Volunteers
- 39 people
- Sponsor
- Janssen Research & Development, LLC
Atopic Dermatitis
A Phase 3 Study of LNK01001 Capsule in Subjects With Moderate to Severe Atopic Dermatitis.
This is a randomized, double-blind study comparing LNK01001 to placebo in participants with moderately to severely atopic dermatitis who were candidates for systemic therapy (ie, patients with a history of inadequate response to…
- Trial name
- NCT06277245
- Testing
- LNK01001
- Where
- China
- Volunteers
- 356 people
- Sponsor
- Lynk Pharmaceuticals Co., Ltd
Burns
Fish Skin Compared to Cadaver Skin as Temporary Cover for Full Thickness Burns
Objectives and rationale: Optimal burn management involves removing all the dead or burned tissue as early as feasible and cover with an autograft called split thickness skin graft (STSG) taken from the patient.
- Trial name
- Kereburn (NCT03984331)
- Testing
- Fish skin, Cadaver skin
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Kerecis Ltd.
Atopic Dermatitis
A Study in Male and Female Adult Patients With Atopic Dermatitis Treated With Dupilumab in Taiwan
Primary objective: To characterize the use patterns of phototherapy and immunosuppressants prior to dupilumab treatment for adult AD patients, who are eligible for dupilumab reimbursement in Taiwan (e.g., used regimens, reason…
- Trial name
- NCT05442645
- Where
- China
- Volunteers
- 80 people
- Sponsor
- Sanofi
Solid Tumor
A Study of LY3200882 in Participants With Solid Tumors
The main purpose of this study is to evaluate the safety of the study drug known as LY3200882 in participants with solid tumors.
- Trial name
- NCT02937272
- Testing
- LY3200882, LY3300054, Gemcitabine
- Where
- 18 sites in United States, Australia and 6 more
- Volunteers
- 223 people
- Sponsor
- Eli Lilly and Company
Oculopharyngeal Muscular Dystrophy
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months of follow up in Study BNTC-OPMD-NH-001 may be eligible to participate…
- Trial name
- NCT06185673
- Testing
- BB-301: Dose escalation phase 1b cohort 1, BB-301: Dose escalation phase 1b cohort 2, BB-301: Dose escalation phase 1b cohort 3
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Benitec Biopharma, Inc.
Pompe Disease (Late-onset)
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric…
- Trial name
- NCT03911505
- Testing
- Cipaglucosidase Alfa, Miglustat
- Where
- 17 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- Amicus Therapeutics
Acute Hepatic Porphyria
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and…
- Trial name
- ELEVATE (NCT04883905)
- Where
- 30 sites in United States, Belgium and 8 more
- Volunteers
- 150 people
- Sponsor
- Alnylam Pharmaceuticals
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
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
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Hereditary Angioedema
A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).
- Trial name
- NCT04654351
- Testing
- TAK-667
- Where
- 3 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Takeda
Dermatitis, Atopic
Crisaborole Ointment 2% Skin Biomarker Biopsy Study in Atopic Dermatitis
This study is being conducted to characterize the mechanism of action of crisaborole ointment 2%, by evaluation of efficacy and changes in key skin biomarkers in atopic dermatitis (AD) lesions treated with crisaborole ointment 2%…
- Trial name
- NCT03233529
- Testing
- Crisaborole ointment 2% BID
- Where
- Canada
- Volunteers
- 40 people
- Sponsor
- Pfizer
Eyelid Diseases, Eyelid Movement Disorders
Feasibility of Dynamic Muscle Stimulation + Radiofrequency for Improving Blink Quality in Subjects with Dry Eye Disease
To demonstrate that DMSt + RF improves eye blink quality in subjects with dry eye disease
- Conditions
- Eyelid Diseases, Eyelid Movement Disorders, Dry Eye, Dry Eye Syndromes
- Trial name
- NCT05945069
- Testing
- TriLift device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Lumenis Be Ltd.
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