Study Design
Single-arm, open-label clinical trial (interim analysis). First clinical investigation in Asia of ketogenic metabolic intervention for ADPKD. 10 patients enrolled, 7 completed 12 months. Mayo Imaging Class C or higher. Median age 44 years, median baseline eGFR 57 mL/min/1.73 m2. MRI-based volumetric measurements at baseline, 3 months, and 12 months.
Intervention
Structured ketogenic diet with multidisciplinary supervision, individualized dietitian guidance, KetoCitra supplementation (BHB + alkaline citrate), digital self-monitoring of ketones, glucose, and nutrients.
Key Results
TKV: Significantly decreased
Paired t-test p=0.026; Wilcoxon p=0.016. In a population where 5-10% annual growth is expected, kidneys actually shrank.
Liver Volume: Significantly decreased
At both 3 and 12 months (Wilcoxon p=0.049 at 12 months).
eGFR: Stabilized
No significant change vs. historical pre-trial decline of approximately -5.6 mL/min/1.73 m2/year.
Dose-Response: Strong correlation
Mean blood ketone levels at 3 months showed strong negative correlation with TKV change (Spearman rho = -0.86, p=0.012). Higher ketones = more kidney shrinkage.
Context & Comparison
Tolvaptan (TEMPO 3:4) reduced annual TKV growth from +5.51%/year (placebo) to +2.80%/year — kidneys still grow. This ketogenic intervention achieved actual TKV decrease.
Significance
First long-term clinical trial data showing ketogenic metabolic therapy can reverse kidney growth in ADPKD. The strong dose-response relationship between ketone levels and kidney volume reduction supports a causal mechanism. Enrollment completed with >50 participants plus a control group; final results expected in approximately one year.