Clinical trials: Open Label
Mucopolysaccharidosis Type II (MPS II)
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system.
- Trial name
- NCT04571970
- Testing
- RGX-121
- Where
- 2 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- REGENXBIO Inc.
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Anemia, Chronic Kidney Disease
Study to Evaluate Darbepoetin Alfa in Pediatric Subjects With Anemia Due to Chronic Kidney Disease
The purpose of this study is to find out more about darbepoetin alfa in children less than 1 year of age with anemia (a decrease in red blood cells) due to kidney failure.
- Trial name
- NCT01428154
- Testing
- Darbepoetin alfa
- Sponsor
- Amgen
Glycogen Storage Disease Type II
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD.
- Trial name
- Baby-COMET (NCT04910776)
- Testing
- avalglucosidase alfa
- Where
- 16 sites in United States, Belgium and 7 more
- Volunteers
- 17 people
- Sponsor
- Sanofi
Peanut Allergy
Real World, Open Label, QOL Assessment of Peanut Immunotherapy AR101 in Children and Adolescents
To compare the HRQOL of AR101 characterized oral desensitization immunotherapy (CODIT™) in combination with standard of care (peanut avoidance, education) versus standard of care alone in peanut-allergic subjects aged 4 to 17…
- Trial name
- NCT03703791
- Testing
- AR101
- Where
- 11 sites in Spain
- Volunteers
- 1 people
- Sponsor
- Aimmune Therapeutics, Inc.
Treatment of Type I Diabetes Mellitus.
Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy
Phase IB will evaluate the safety of autologous, ex vivo-engineered, co-stimulation impaired dendtritic cells to maintain and improve functional residual beta cell mass in new onset Type I Diabetes Mellitus (T1DM) patients.
- Trial name
- NCT01947569
- Testing
- Biological
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- DiaVacs, Inc.
Neoplasms
Continued Treatment for Participants Enrolled in Studies of BXQ-350
This is a study to allow continued treatment for participants enrolled in studies of BXQ-350. This study is intended only for those who have completed the required study observation period or are still on treatment upon the…
- Trial name
- ETERNITI (NCT04404569)
- Testing
- BXQ-350
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Bexion Pharmaceuticals, Inc.
Atopic Dermatitis
Study of Ruxolitinib Cream in Children With Atopic Dermatitis
This is an open-label maximum use trial to evaluate ruxolitinib safety, tolerability and blood levels after its topical application twice daily to affected areas (≥ 35% BSA) in pediatric participants with atopic dermatitis (AD)…
- Trial name
- NCT05034822
- Testing
- Ruxolitinib cream
- Where
- 15 sites in United States
- Volunteers
- 29 people
- Sponsor
- Incyte Corporation
Myeloid Leukemia, Philadelphia Positive
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
The aim of this study is to support development of asciminib in the pediatric population (1 to <18 years) previously treated with one or more TKIs.
- Trial name
- NCT04925479
- Testing
- Asciminib Pediatric formulation group, Asciminib Adult formulation group
- Where
- 39 sites in United States, China and 12 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Metastatic Breast Cancer
Multicenter Follow Up Study Of Subjects Who Participated In An Original Protocol Of Exemestane Vs. Megestrol Acetate In Postmenopausal Women With Metastatic Breast Cancer
Long term efficacy of exemestane as compared to megestrol acetate in the treatment of women with natural or induced postmenopausal status with advanced breast cancer whose disease has progressed following anti-estrogens or…
- Trial name
- NCT01237327
- Testing
- Megestrol acetate, exemestane (Aromasin)
- Where
- 7 sites in China
- Volunteers
- 84 people
- Sponsor
- Pfizer
Homozygous Familial Hypercholesterolaemia (HoFH)
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein…
- Trial name
- NCT04681170
- Testing
- Lomitapide
- Where
- 12 sites in Germany, Israel and 4 more
- Volunteers
- 46 people
- Sponsor
- Amryt Pharma
HIV Infections
Safety and Effectiveness of TRIZIVIR (Abacavir/Lamivudine/Zidovudine) With Efavirenz in HIV-Infected Patients Who Have Never Taken Anti-HIV Drugs
The purpose of this study is to determine the effect of treatment with Trizivir (TZV) plus efavirenz (EFV) or TZV alone on viral load (level of HIV in the blood).
- Trial name
- NCT00011895
- Testing
- Abacavir sulfate, Lamivudine and Zidovudine, Efavirenz
- Where
- 42 sites in United States
- Volunteers
- 400 people
- Sponsor
- GlaxoSmithKline
Acquired Immune Deficiency Syndrome (AIDS), HIV Infections
Study of Cobicistat-Boosted Atazanavir (ATV/co), Cobicistat-Boosted Darunavir (DRV/co) and Emtricitabine/Tenofovir Alafenamide (F/TAF) in Children With HIV
The goal of this clinical study is to learn more about the safety and dosing of study drugs, cobicistat-boosted Atazanavir (ATV/co), cobicistat-boosted darunavir (DRV/co) and emtricitabine/tenofovir alafenamide (F/TAF), in…
- Trial name
- NCT02016924
- Testing
- ATV, DRV, Cobicistat
- Where
- 23 sites in United States, Argentina and 5 more
- Volunteers
- 133 people
- Sponsor
- Gilead Sciences
Undernutrition
Study of Blood Parameters of the Malnourished Children: Before and After Giving Nutritional Biscuit's Treatment
Present study was designed to evaluate the efficacy of the nutritional intervention, in the SAM (Severe Acute malnutrition)children by monitoring various Anthropometric,Biochemical ,Hormonal and Enzymatic parameters before and…
- Trial name
- NCT02459223
- Testing
- Nutritional biscuits and khichadi, Khichadi
- Where
- India
- Volunteers
- 105 people
- Sponsor
- Maharashtra University of Health Sciences
Autistic Disorder
Open-Label Extension Study of Kuvan for Autism
This is an open-label extension study available only to subjects who completed an earlier double-blind, placebo-controlled study of sapropterin in children with autism.
- Trial name
- NCT00943579
- Testing
- Kuvan®
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- The Children's Health Council
Asthma, Chronic Obstructive Pulmonary Disease (COPD)
Open-Label Assessment of the Albuterol Spiromax® Dry Powder Inhaler (DPI)
This is a prospective, open-label, multicenter Phase 3 study evaluating the performance of the Albuterol Spiromax dose counter in patients with a diagnosis of asthma and/or COPD.
- Trial name
- NCT01857323
- Testing
- Albuterol Spiromax®
- Where
- 30 sites in United States
- Volunteers
- 317 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL)
A Study Evaluating the Safety, Tolerability, Pharmacokinetics and Preliminary Activity of Idasanutlin in Combination With Either Chemotherapy or Venetoclax in Treatment of Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias or Solid Tumors
This is a Phase I/II, multicenter, open-label, multi-arm study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of idasanutlin, administered as a single agent or in combination with…
- Conditions
- Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Neuroblastoma, Solid Tumors
- Trial name
- NCT04029688
- Testing
- Idasanutlin, Venetoclax, Cyclophosphamide
- Where
- 14 sites in United States, Canada and 4 more
- Volunteers
- 38 people
- Sponsor
- Hoffmann-La Roche
Malaria
In Vivo Efficacy of Artemether-lumefantrine and Amodiaquine-artesunate in Mozambican Children
This is a classical in vivo clinical trial, following World Health organization's recommendations, ran as a multisite study within Mozambique trying to assess the efficacy and safety in 5 sites of the two oral ACTS…
- Trial name
- MEFI (NCT02168569)
- Testing
- Coartem™ (Artemether-lumefantrine combination), Coarsucam™ (Amodiaquine-artesunate combination)
- Where
- 5 sites in Mozambique
- Volunteers
- 700 people
- Sponsor
- Centro de Investigacao em Saude de Manhica
Glioblastoma (GBM), DIPG
Prolonged Exposure to Doxorubicin in Patients With Glioblastoma Multiforme and Diffuse Intrinsic Pontine Glioma
The standard therapy of glioblastoma (GBM) consists of gross total resection followed by focal irradiation to the tumor bed with concomitant and adjuvant temozolomide (TMZ).
- Conditions
- Glioblastoma (GBM), DIPG, Brainstem Glioma, Pediatric, Diffuse Spinal Glioma
- Trial name
- NCT02758366
- Testing
- Doxorubicin
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Meyer Children's Hospital IRCCS
Moderate to Severe Chronic Plaque-type Psoriasis
Study to Assess the Long-term Safety, Tolerability, Efficacy of Secukinumab in Pediatric Patients of Age 6 to <18 Years, With Moderate to Severe Plaque Psoriasis
This was an open-label, parallel-group, two-arm, multicenter study in pediatric subjects aged 6 years to less than 18 years, at randomization, with moderate to severe chronic plaque psoriasis.
- Trial name
- NCT03668613
- Testing
- secukinumab low dose, secukinumab high dose
- Where
- 23 sites in United States, Belgium and 7 more
- Volunteers
- 84 people
- Sponsor
- Novartis Pharmaceuticals
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