A study conduct in several centres, further to a previous study, to evaluate the safety, tolerability and efficacy of ADV7103 at long term in the treatment of distal renal tubular acidosis

Conditions

Distal renal tubular acidosis MedDRA version: 20.1 Level: PT Classification code 10038535 Term: Renal tubular acidosis System Organ Class: 10038359 - Renal and urinary disorders

Michaël Acquadro, Alexia Marrel, Maria A. Manso-Silván, Catherine Guittet, Sophie Joukoff et al. (6 authors)

Orphanet Journal of Rare Diseases2022-03-285 citations

10.1186/s13023-022-02294-w

Safety, efficacy, and acceptability of ADV7103 during 24 months of treatment: an open-label study in pediatric and adult patients with distal renal tubular acidosis.

Bertholet-Thomas A, Guittet C, Manso-Silván MA, Joukoff S, Navas-Serrano V, Baudouin V, Cailliez M, Di Maio M, Gillion-Boyer O, Golubovic E, Harambat J, Knebelmann B, Nobili F, Novo R, Podracka L, Roussey-Kesler G, Granier LA.

2021-02-269 citations

10.1007/s00467-020-04873-0

Interventions

Product Code: ADV7103 Pharmaceutical Form: Prolonged-release granules INN or Proposed INN: potassium citrate CAS Number: 6100-05-6 Other

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Subject who has participated to and completed the previous Advicenne clinical Study B21CS. Are the trial subjects under 18? yes Number of subjects for this age range: 18 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Subject who has not participated to Advicenne clinical Study B21CS Subject for whom any safety issue could contraindicate her/his participation to the extension study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety and tolerability as measured by adverse events of ADV7103 at long term.; Secondary Objective: - To evaluate the efficacy on correcting metabolic acidosis as measured by bicarbonataemia of ADV7103 at long-term. - To evaluate the effects on hypocitraturia of ADV7103 at long term. - To evaluate the effects on hypercalciuria of ADV7103 at long term. - To evaluate the effects on crystalluria of ADV7103 at long term. - To evaluate the paraclinical and biological safety of ADV7103 at long term. - To evaluate the compliance to ADV7103 at long term. ; Primary end point(s): Safety and tolerability endpoint: Number/proportion of subjects presenting adverse events during the course of the study, including the incidence and severity of the adverse events ;Timepoint(s) of evaluation of this end point: During the all study period	—

Measure

Time frame

Main Objective: To evaluate the safety and tolerability as measured by adverse events of ADV7103 at long term.; Secondary Objective: - To evaluate the efficacy on correcting metabolic acidosis as measured by bicarbonataemia of ADV7103 at long-term. - To evaluate the effects on hypocitraturia of ADV7103 at long term. - To evaluate the effects on hypercalciuria of ADV7103 at long term. - To evaluate the effects on crystalluria of ADV7103 at long term. - To evaluate the paraclinical and biological safety of ADV7103 at long term. - To evaluate the compliance to ADV7103 at long term. ; Primary end point(s): Safety and tolerability endpoint: Number/proportion of subjects presenting adverse events during the course of the study, including the incidence and severity of the adverse events ;Timepoint(s) of evaluation of this end point: During the all study period

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Secondary

Measure	Time frame
Timepoint(s) of evaluation of this end point: At each study visit; Secondary end point(s): 1. Efficacy endpoints - Number/proportion of subjects presenting a bicarbonataemia in the normal ranges - Mean and change from baseline of values of bicarbonate blood level at each study visit (with baseline defined at the inclusion in the B21CS Study) - Number/proportion of subjects presenting a citraturia in the normal ranges, at each study visit including the incidence and severity of the hypocitraturia - Mean and change from baseline (with baseline defined at the inclusion in the B21CS Study) of values of citrate/creatinine urine ratio (UCi/Cr) at each study visit - Number/proportion of subjects presenting a calciuria in the normal ranges, at each study visit including the incidence and severity of the hypercalciuria - Mean and change from baseline of urine calcium/citrate ratio (UCa/Ci) at each study visit (with baseline defined at the inclusion in the B21CS Study) - Number/proportion of subjects presenting a crystalluria (mainly with hydroxyapaptite) at each study visit - Mean and change from baseline of crystalluria (mainly with hydroxyapatite) at each study visit (with baseline defined at the inclusion in the B21CS Study) 2. Safety and tolerability endpoints • Number/proportion of subjects presenting abnormal values after treatment, at each study visit including the incidence and severity of these abnormal values and the change from baseline (with baseline defined at the inclusion in the B21CS Study when applicable) - Vital signs (SBP, DBP, HR, RR) - ECG parameters (PR, QRS, QT interval, Bazett and Fridericia QTc intervals) - Liver function tests (transaminases ASAT, ALAT) - Urinalysis including urine pH and specific gravity, with m	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: At each study visit; Secondary end point(s): 1. Efficacy endpoints - Number/proportion of subjects presenting a bicarbonataemia in the normal ranges - Mean and change from baseline of values of bicarbonate blood level at each study visit (with baseline defined at the inclusion in the B21CS Study) - Number/proportion of subjects presenting a citraturia in the normal ranges, at each study visit including the incidence and severity of the hypocitraturia - Mean and change from baseline (with baseline defined at the inclusion in the B21CS Study) of values of citrate/creatinine urine ratio (UCi/Cr) at each study visit - Number/proportion of subjects presenting a calciuria in the normal ranges, at each study visit including the incidence and severity of the hypercalciuria - Mean and change from baseline of urine calcium/citrate ratio (UCa/Ci) at each study visit (with baseline defined at the inclusion in the B21CS Study) - Number/proportion of subjects presenting a crystalluria (mainly with hydroxyapaptite) at each study visit - Mean and change from baseline of crystalluria (mainly with hydroxyapatite) at each study visit (with baseline defined at the inclusion in the B21CS Study) 2. Safety and tolerability endpoints • Number/proportion of subjects presenting abnormal values after treatment, at each study visit including the incidence and severity of these abnormal values and the change from baseline (with baseline defined at the inclusion in the B21CS Study when applicable) - Vital signs (SBP, DBP, HR, RR) - ECG parameters (PR, QRS, QT interval, Bazett and Fridericia QTc intervals) - Liver function tests (transaminases ASAT, ALAT) - Urinalysis including urine pH and specific gravity, with m

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Countries

France, Serbia, Slovakia, Spain

Contacts

Public ContactDirector of Clinical Affairs

Advicenne Pharma

cguittet@advicenne.com33466 05 54 23

Outcome results

None listed