A Multi-center, Longitudinal, Observational Study of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) to Establish the Rate, Characteristics, and Determinants of Disease Progressio

Phase 4

Completed

Conditions: ADPKD
autosomal polycystic kidney disease
10029149

Registration Number: NL-OMON36219

Lead Sponsor: Otsuka America Pharmaceutical

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 100

Inclusion Criteria

1. Ages 12 to 70 years, inclusive (18 to 70 inclusive in the Netherlands)
2. Existing diagnosis of ADPKD by modified-Ravine criteria:
• With family history: several cysts per kidney (3 if by sonography, 5 if by computerized
tomography or MRI)
• Without family history: 10 cysts (by any radiologic method) per kidney and exclusion of other
cystic kidney diseases
3. Ability to provide written, informed consent/assent prior to initiation of any study-related
procedures, and ability, in the opinion of the investigator, to comply with all requirements of the
study.
4. Total kidney volume >= 300 cc/m height by ultrasound (within 1 year prior to baseline) or >= 250
cc/m height by MRI (within 1 year prior to baseline).

Exclusion Criteria

1. Any medical condition, in the opinion of the Investigator, that could interfere with evaluation of the study objectives (eg, inability to comply with MRI)
2. Current or expected (within the next six months) interventions for the treatment of ADPKD
affecting kidney volume without the prior approval of the sponsor

Study & Design

Study Type: Observational invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary:<br /><br><br /><br>• Investigate a correlation between the rate of kidney enlargement and the<br /><br>decline of renal function in ADPKD patients<br /><br>• Establish the utility of renal volume measurements as a prognostic marker of<br /><br>disease progression</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary:<br /><br><br /><br>• Evaluate and establish the level of, and associations between, the impact of<br /><br>disease on patient reported outcomes (PROs) for pain, health related quality of<br /><br>life (HRQoL), and ADPKD-associated health burden<br /><br>• Evaluate the impact of disease on medical resource utilization (MRU) and<br /><br>productivity</p><br>