Clinical trials: Genetic Kidney Disease
- Condition Genetic Kidney Disease
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Hemophilia A
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
Hyperoxaluria, Primary
An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Open label extension study of Oxabact OC5 in patients with primary hyperoxaluria
- Trial name
- ePHex-OLE (NCT03938272)
- Testing
- Oxabact OC5 - Oxalobacter formigenes Strain HC-1
- Where
- 8 sites in United States, Belgium and 4 more
- Volunteers
- 22 people
- Sponsor
- OxThera
Primary Hyperoxaluria
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Patients With Primary Hyperoxaluria
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- NCT02012985
- Testing
- Oxabact OC5 capsules
- Where
- 8 sites in France, Germany and 1 more
- Volunteers
- 28 people
- Sponsor
- OxThera
Primary Hyperoxaluria
A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
This study will evaluate the efficacy and safety of OC5 in patients with PH.
- Trial name
- NCT03116685
- Testing
- Oxabact OC5 - Oxalobacter formigenes HC-1
- Where
- 10 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- OxThera
Renal Fanconi Syndrome, Photophobia
Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Cystinosis disease from the blood
- Conditions
- Renal Fanconi Syndrome, Photophobia, Hypophosphatemia
- Trial name
- BioCystinosis (NCT02837523)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Atypical Hemolytic Uremic Syndrome
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician.
- Trial name
- EVIDENCE (NCT02614898)
- Testing
- Eculizumab
- Where
- 24 sites in United States, Australia and 2 more
- Volunteers
- 67 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Primary Hyperoxaluria
Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter.
- Trial name
- NCT03819647
- Testing
- stiripentol (Diacomit)
- Where
- 3 sites in France
- Volunteers
- 15 people
- Sponsor
- Biocodex
Solid Tumors, CNS Tumors
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain…
- Trial name
- STARTRK-NG (NCT02650401)
- Testing
- Entrectinib
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 69 people
- Sponsor
- Hoffmann-La Roche
Neuroblastoma, Rhabdomyosarcoma
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
This study is a Phase 1, open-label, dose escalation and cohort expansion trial designed to characterize the safety, tolerability, PK, PD, immunogenicity and preliminary antitumor activity of enoblituzumab administered IV on a…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT02982941
- Testing
- Enoblituzumab
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- MacroGenics
Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3.
- Conditions
- Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
- Trial name
- CRYSTAL (NCT06465472)
- Testing
- Stiripentol Oral Capsule, Urine samples collect, Blood samples collect
- Volunteers
- 42 people
- Sponsor
- Biocodex
Neuroblastoma, Ewing's Sarcoma
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy.
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma
- Trial name
- NCT01598454
- Testing
- racotumomab
- Where
- Argentina
- Volunteers
- 15 people
- Sponsor
- Laboratorio Elea Phoenix S.A.
Primary Hyperoxaluria
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),
- Trial name
- NCT04125472
- Testing
- Lumasiran
- Sponsor
- Alnylam Pharmaceuticals
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)
The primary objective of the study is to assess the long term safety of treatment with tolvaptan in children and adolescents with autosomal dominant polycystic kidney disease (ADPKD).
- Trial name
- NCT02964273
- Testing
- Tolvaptan
- Where
- 20 sites in Belgium, Germany and 2 more
- Volunteers
- 91 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Primary Hyperoxaluria
Study to Evaluate the Efficacy and Safety of OxabactTM on Reduction of Urinary Oxalate in Primary Hyperoxaluria Patients
The main purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- PHOENIX (NCT00638703)
- Testing
- Oxalobacter formigenes
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- OxThera
Cystinuria
Study Evaluating Patients With Cystinuria
This is an open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and acceptability of alkalising treatments at long-term in patients with cystinuria.
- Trial name
- NCT04137978
- Testing
- ADV7103, Standard of Care
- Where
- 13 sites in Belgium, France
- Sponsor
- Advicenne Pharma
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1
Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
The aim of this study is to evaluate nedosiran in participants 11 years of age and younger who have Primary Hyperoxaluria with relatively intact renal function.
- Conditions
- Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3
- Trial name
- PHYOX8 (NCT05001269)
- Testing
- nedosiran
- Where
- 13 sites in United States, Canada and 9 more
- Volunteers
- 27 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Enteric Hyperoxaluria, Primary Hyperoxaluria
Study of ALLN-177 in Patients Aged 12 Years or Older With Enteric or Primary Hyperoxaluria and Hyperoxalemia
Evaluate the efficacy and safety of ALLN-177 in reducing plasma and urinary oxalate levels in adult and pediatric patients with enteric hyperoxaluria and hyperoxalemia or primary hyperoxaluria
- Conditions
- Enteric Hyperoxaluria, Primary Hyperoxaluria, Hyperoxalemia
- Trial name
- NCT03391804
- Testing
- ALLN-177
- Where
- 9 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Allena Pharmaceuticals
Autosomal Recessive Polycystic Kidney Disease (ARPKD)
A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)
- Trial name
- NCT04786574
- Testing
- Tolvaptan (OPC-41061)
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.