White label – Pompe disease - ClinicalTrials.eu

Study on the Effects of Pemafibrate and Tofogliflozin in Patients with Nonalcoholic Steatohepatitis (NASH) and Liver Fibrosis

Not recruiting

2 1
Investigated Drugs:
- Pemafibrate
- Tofogliflozin
Study on the Effectiveness and Safety of Mitapivat in Children with Pyruvate Kinase Deficiency Not Receiving Regular Blood Transfusions

Not recruiting

3 1
Investigated Diseases:
- Pyruvate kinase deficiency anaemia
Investigated Drugs:
- Mitapivat
Study of Daratumumab Injections for Patients with Moderate to Severe Chronic Fatigue Syndrome

Not recruiting

2 1 1 1
Investigated Diseases:
- Chronic fatigue syndrome
Investigated Drugs:
- Daratumumab
Study Comparing Bictegravir/Lenacapavir to Current Therapy in People with HIV-1 Who Are Successfully Treated

Not recruiting

3 1 1
Investigated Diseases:
- HIV infection
Investigated Drugs:
Study on Pembrolizumab for Patients with High-Risk Stage III Melanoma After Surgery

Not recruiting

3 1 1
Investigated Drugs:
- Pembrolizumab
+7
Study on the Effectiveness and Safety of Avalglucosidase Alfa for Babies with Infantile-Onset Pompe Disease

Not recruiting

3 1 1 1
Investigated Diseases:
- Glycogen storage disease type II
Investigated Drugs:
- Avalglucosidase Alfa
Study on the Effectiveness and Safety of Ianalumab for Patients with Active Sjögren’s Syndrome

Not recruiting

3 1 1
Investigated Diseases:
- Dry eye
- Sjogren’s syndrome
Investigated Drugs:
- Ianalumab
+5
Study on the Safety and Effectiveness of AMX0035 (Phenylbutyrate and Ursodoxicoltaurine) for Adults with Amyotrophic Lateral Sclerosis (ALS)

Not recruiting

3 1 1
Investigated Diseases:
- Amyotrophic lateral sclerosis
Investigated Drugs:
- Phenylbutyrate
- Ursodoxicoltaurine
+4
Study of DYNE-251 safety and effectiveness in patients with Duchenne muscular dystrophy who are candidates for exon 51 skipping treatment

Not recruiting

2 1
Investigated Diseases:
- Duchenne muscular dystrophy
Investigated Drugs:
- Fragment Antibody Targeting Human Tfr1 Conjugated To Phosphorodiamidate Morpholino Oligomer
Study on the Effect of Cannabidiol (CardiolRx) on Heart Recovery in Patients with Acute Myocarditis

Not recruiting

2 1 1
Investigated Diseases:
- Myocarditis
Investigated Drugs:
- Cannabidiol

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