Clinical trials: Neonatal Renal Disorder (Disorder)
Mitochondrial Disease, Chronic Kidney Disease
Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
The purpose of this expanded access program is to enable access to R007 (probucol) tablets for the compassionate treatment of adults with mitochondrial disease who also have chronic kidney disease.
- Trial name
- NCT06931834
- Testing
- Probucol
- Where
- United States
- Sponsor
- RiboNova Inc.
Polycystic Kidney, Autosomal Recessive
A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
This study evaluates safety and tolerability of a single ascending dose of a tesevatinib liquid formulation administered to pediatric subjects with ARPKD.
- Trial name
- NCT03096080
- Testing
- Tesevatinib
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Kadmon Corporation, LLC
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
Sarcoma, Neuroblastoma
To Evaluate the Safety, Activity and Pharmacokinetics of Marqibo in Children and Adolescents With Refractory Cancer
Background: Marqibo(Registered Trademark) is a new anticancer drug. It combines Vincristine sulfate, which is a widely used anticancer drug, and packages it into a tiny fat bubble known as a liposome.
- Conditions
- Sarcoma, Neuroblastoma, Wilms Tumor, Leukemia
- Trial name
- NCT01222780
- Testing
- Marqibo
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Acrotech Biopharma Inc.
Cystinuria
Bucillamine Phase 2 Trial in Patients With Cystinuria
Subjects on a standard regimen of tiopronin (cystine binding thiol drug; CBTD) plus prescribed first-line therapy (i.e. on a hydration, alkali therapy and dietary restriction) who are failing therapy will be selected for this…
- Trial name
- NCT02942420
- Testing
- Bucillamine
- Where
- 5 sites in United States
- Volunteers
- 30 people
- Sponsor
- Revive Therapeutics, Ltd.
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Fabry Disease
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)
- Trial name
- NCT04020055
- Testing
- migalastat HCl 150 mg
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 14 people
- Sponsor
- Amicus Therapeutics
Chronic Kidney Diseases, Hemodialysis
Must Cannulation Technique of Vascular Access in Patients Undergoing Haemodialysis: Contributions for a Safe Nursing Intervention
The aims of this study are to: Determine the AVF (arteriovenous fistula) survival of patients submitted to MuST compared to those submitted to RL (rope-ladder).
- Conditions
- Chronic Kidney Diseases, Hemodialysis, Vascular Access Complication, Hemodialysis Access Failure
- Trial name
- MuST (NCT05081648)
- Testing
- CT MuST, Rope-ladder cannulation technique
- Where
- 3 sites in Portugal
- Volunteers
- 101 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH
Polycystic Liver Disease
A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD…
- Trial name
- POSITANO (NCT05281328)
- Testing
- CAM2029
- Where
- 11 sites in United States, Belgium and 2 more
- Volunteers
- 71 people
- Sponsor
- Camurus AB
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Dwarfism
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation
This study aims to evaluate the efficacy and safety of recombinant human growth hormone injection in short stature children due to chronic kidney disease before transplantation.
- Trial name
- NCT03535415
- Testing
- Recombinant Human Growth Hormone Injection (rhGH)
- Where
- 12 sites in China
- Volunteers
- 68 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Renal Impairment
Effect of Febuxostat on Renal Function in Patients With Gout and Moderate to Severe Renal Impairment
The purpose of this study is to determine the effect of febuxostat, once daily (QD) or twice daily (BID), on renal function in gout patients with elevated serum urate levels and who have moderate to severe renal impairment.
- Trial name
- NCT01082640
- Testing
- Febuxostat
- Where
- 58 sites in United States
- Volunteers
- 96 people
- Sponsor
- Takeda
Nephritis, Hereditary, Hematuria-Nephropathy-Deafness Syndrome
Biomarker for Alport Syndrome (BioAlport)
International, multicenter, observational, longitudinal monitoring study to identify biomarker/s for Alport syndrome and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Trial name
- BioAlport (NCT02718027)
- Where
- 7 sites in Albania, Georgia and 5 more
- Volunteers
- 12 people
- Sponsor
- CENTOGENE GmbH Rostock
Chronic Kidney Disease
A Study of Vonapanitase (PRT-201) Administered Immediately After Radiocephalic Arteriovenous Fistula(AVF) Creation in Patients With Chronic Kidney Disease (CKD) (PATENCY-2)
This research study is designed to assess the safety and effectiveness of an experimental drug called vonapanitase (PRT-201) in patients both receiving or expecting to receive hemodialysis who have chronic kidney disease and who…
- Trial name
- NCT02414841
- Testing
- Vonapanitase
- Where
- 41 sites in United States, Canada
- Volunteers
- 696 people
- Sponsor
- Proteon Therapeutics
X-linked Hypophosphatemia
A Study of KRN23 in X-linked Hypophosphatemia
The primary objective of this study is to assess the safety and tolerability of KRN23 after a single intravenous (IV) and subcutaneous (SC) administration in XLH patients.
- Trial name
- NCT00830674
- Testing
- KRN23
- Where
- 4 sites in United States
- Volunteers
- 42 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Acidosis, Renal Tubular
Extension Study in Primary Distal Renal Tubular Acidosis
Open-label study involving longitudinal assessment of the continued safety, tolerability, and efficacy of ADV7103 in maintaining targeted serum bicarbonate levels, preventing metabolic acidosis, and preventing hypokalemia in the…
- Trial name
- NCT03831152
- Testing
- ADV7103
- Where
- United States
- Sponsor
- Advicenne Pharma
Autosomal Dominant Polycystic Kidney Disease
A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
This is an exploratory, randomized, double-blind, placebo-controlled, parallel group, multicenter, proof of concept study (Phase 2a), evaluating orally administered GLPG2737 for a double-blind (DB) treatment period of 52 weeks…
- Trial name
- NCT04578548
- Testing
- GLPG2737
- Where
- 20 sites in Belgium, Czechia and 5 more
- Volunteers
- 66 people
- Sponsor
- Lakefront Biotherapeutics NV
Polycystic Kidney, Autosomal Dominant
Tolvaptan Open-label Pilot Efficacy, Tolerability, and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
This study's purpose is to evaluate the long-term safety of open-label tolvaptan regimens to determine the maximally-tolerated dose and acquire pilot efficacy data in patients with autosomal dominant polycystic kidney disease…
- Trial name
- TEMPO 2:4 (NCT00413777)
- Testing
- Tolvaptan
- Where
- 11 sites in United States
- Volunteers
- 46 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
This is a randomized, double-Blind, placebo-controlled study to evaluate the efficacy and safety of exaluren in Alport Syndrome patients with nonsense mutations in COL4A3/4/5 genes.
- Trial name
- NCT07523581
- Testing
- Exaluren
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 24 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.