Abstract: TH-PO1117

Safety and Efficacy of ADV7103, an Innovative Prolonged-Release Oral Alkalising Combination Product, after 6-Months of Treatment in Distal Renal Tubular Acidosis (dRTA) Patients

Session Information

Category: Fluid, Electrolytes, and Acid-Base

  • 704 Fluid, Electrolyte, Acid-Base Disorders

Authors

  • Bertholet-Thomas, Aurélia, Centre de référence des maladies rénales rares, Bron, France
  • Guittet, Catherine, Advicenne, Nîmes, France
  • Manso, Maria Asuncion, Advicenne, Nîmes, France
  • Granier, Luc andre, Advicenne, Nîmes, France

Group or Team Name

  • B22CS study investigators
Background

A new innovative age-adapted prolonged-release granule combination of potassium citrate and potassium bicarbonate, ADV7103, has been developed in order to achieve sustained physiological blood pH values in dRTA patients with a simplified dosing regimen . The current standards of care (SoC) require multiple administrations and are not always well tolerated.
The objective of this clinical study was to assess safety of ADV7103 after treatment for 6 months as well as to follow-up bicarbonate levels and evaluate patient’s satisfaction.

Methods

Adult and pediatric dRTA patients (N=30) were included in a multicentre (N=12), open-label, 24-month study. They received ADV7103 twice a day at appropriate doses.
Preliminary data after 6 months of treatment were analysed, including adverse events and bicarbonataemia. Improvement of quality of life was evaluated at by patients and/or their parents using a 100-mm visual analogue scale.

Results

A total of 17 patients presented adverse events. Among the 45 adverse events observed, 40 were unrelated, 1 (abdominal pain) was unlikely related, 3 (alopecia, dyspepsia and abdominal pain) were possibly related, and 1 (diarrhea) was probably related to the treatment. The 5 latter adverse events were all of mild intensity. There was only one serious adverse event unrelated to the product (wisdom teeth removal).
Efficacy was maintained after 6 months treatment, with blood bicarbonate levels above 21 mM in 79% of the patients. Only three patients presented bicarbonatemia levels below 20 mM. ADV7103 doses ranged from 1.3 to 7.2 mEq/kg/day.
Patients and/or their parents were extremely satisfied with ADV7103. The change of alkalising treatment from the their SoC to ADV7103 allowed an average improvement of their quality of life of 80.5%, ranging from 76 to 98% depending of the age group considered.

Conclusion

The present preliminary results confirm the excellent safety and efficacy of ADV7103, a combination product allowing treatment with only 2-daily doses. The level of satisfaction of the patients is very high and clinicians are expecting registration of the product for first-line treatment of dRTA.

Funding

  • Private Foundation Support