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)
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|ClinicalTrials.gov Identifier: NCT04786574|
Recruitment Status : Not yet recruiting
First Posted : March 8, 2021
Last Update Posted : March 8, 2021
|Condition or disease||Intervention/treatment||Phase|
|Autosomal Recessive Polycystic Kidney Disease (ARPKD)||Drug: Tolvaptan (OPC-41061)||Phase 3|
Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 3), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).
The trial will be the first trial of tolvaptan in a pediatric ARPKD population.
Participants in this study will be assigned to tolvaptan for 24 months and closely monitored over the course of the study.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)|
|Estimated Study Start Date :||April 1, 2021|
|Estimated Primary Completion Date :||March 31, 2025|
|Estimated Study Completion Date :||July 1, 2025|
|Experimental: Tolvaptan (OPC-41061)||
Drug: Tolvaptan (OPC-41061)
Tolvaptan suspension will be administered orally or via nasogastric tube at doses of 0.15 mg/kg once daily in the AM, 0.30 mg/kg once daily in the AM, 0.5 mg/kg once daily in the AM, 0.75 mg/kg split dose (0.5 mg/kg AM and 0.25 mg/kg 8 hours later), and 1 mg/kg split dose (0.67 mg/kg AM and 0.33 mg/kg 8 hours later) based on age. Treatment duration is 2 years.
- The percentage of subjects that will have RRT by 1 year of age. [ Time Frame: From Enrollment to 1 year of age ]
- Rate of change of eGFR by Schwartz formula from pre-treatment to after 2 years of treatment [ Time Frame: From Enrollment to 2 years of age ]
- Acceptance and palatability of the suspension formulation will be assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose [ Time Frame: From Enrollment to 2 years of age ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04786574
|Contact: Lindsay Getz||(919) firstname.lastname@example.org|
|Study Director:||Rosa Real, MD||Otsuka Pharmaceutical Development & Commercialization, Inc.|