Tolvaptan (OPC-41061) for Autosomal Recessive Polycystic Kidney Disease

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Autosomal Recessive Polycystic Kidney DiseaseTolvaptan (OPC-41061) - Drug
Eligibility
28 - 12
All Sexes
What conditions do you have?
Select

Study Summary

This trial will test if tolvaptan can help prevent or delay kidney failure in children with ARPKD.

Eligible Conditions
  • Autosomal Recessive Polycystic Kidney Disease

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

2 Primary · 4 Secondary · Reporting Duration: From Enrollment to 2 years of age

Year 1
The percentage of subjects that will have RRT by 1 year of age.
The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.
Year 2
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
Acceptance of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose
Oral cavity
Rate of change of eGFR by Schwartz formula from pre-treatment to after 2 years of treatment

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

Tolvaptan (Single-blind Treatment Period)
32%Polyuria
29%Thirst
21%Nocturia
10%Polydipsia
9%Dry mouth
This histogram enumerates side effects from a completed 2017 Phase 3 trial (NCT02160145) in the Tolvaptan (Single-blind Treatment Period) ARM group. Side effects include: Polyuria with 32%, Thirst with 29%, Nocturia with 21%, Polydipsia with 10%, Dry mouth with 9%.

Trial Design

1 Treatment Group

Tolvaptan (OPC-41061)
1 of 1

Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Tolvaptan (OPC-41061) · No Placebo Group · Phase 3

Tolvaptan (OPC-41061)
Drug
Experimental Group · 1 Intervention: Tolvaptan (OPC-41061) · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tolvaptan
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from enrollment to 2 years of age

Who is running the clinical trial?

Otsuka Pharmaceutical Development & Commercialization, Inc.Lead Sponsor
243 Previous Clinical Trials
164,602 Total Patients Enrolled
Rosa Real, MDStudy DirectorOtsuka Pharmaceutical Development & Commercialization, Inc.
3 Previous Clinical Trials
107 Total Patients Enrolled
Nataliya Agafonova, MDStudy DirectorOtsuka Pharmaceutical Development & Commercialization, Inc.
1 Previous Clinical Trials
289 Total Patients Enrolled
Olga Sergeyeva, MDStudy DirectorOtsuka Pharmaceutical Development & Commercialization, Inc.
1 Previous Clinical Trials
10 Total Patients Enrolled

Eligibility Criteria

Age 28 - 12 · All Participants · 5 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are male or female subjects between 28 days and < 12 weeks of age, inclusive at the time of enrollment.
You have had oligohydramnios or anhydramnios.
You are able to provide informed consent and to comply with all the requirements of the trial.