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Extension Study in Primary Distal Renal Tubular Acidosis

Primary Purpose

Acidosis, Renal Tubular

Status
Not yet recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
ADV7103
Sponsored by
Advicenne Pharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acidosis, Renal Tubular

Eligibility Criteria

4 Months - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Subjects who participated in Study B23CS must meet all of the following criteria to be registered into this study:

1. Female or male subjects who participated in Study B23CS and were adherent to the protocol;

Subjects who did not participate in Study B23CS must meet all of the following criteria to be registered into this study:

  1. Female or male subjects ≤ 65 years of age at time of consent with the following stipulations:

    1. Subjects ≥ 6 months of age must reside in Europe
    2. Subjects < 6 months of age must reside in the United States, Canada, or Europe;
  2. Subjects < 6 months of age must be able to swallow (not suck) solid food without difficulty;
  3. Subject presents with a previous diagnosis of primary dRTA;
  4. Subject ≥ 6 months of age requires ≥ 0.9 mEq/kg/day of alkali therapy to maintain serum bicarbonate levels above the LLN;
  5. Urine pH > 5.5 and serum bicarbonate > 18 mEq/L for subjects ≥ 4 years old or > 17 mEq/L for subjects < 4 years old on alkali therapy and potassium supplementation (if indicated) on at least one occasion for each within 6 months;
  6. European subjects must be included in a social health insurance system and/or in compliance with the recommendations of the national law in force relating to biomedical research;
  7. Subject or parent/guardian is willing and able to understand and sign informed consent and willing to comply with protocol instructions; child assent when appropriate; and

Exclusion Criteria:

Subjects who participated in Study B23CS and meet any of the following criteria will be considered ineligible for registration into this study:

  1. Female subject who is pregnant or lactating or has plans for pregnancy during the study; or
  2. Subject requires therapy with potassium sparing diuretics, angiotensin converting enzyme inhibitors, angiotensin II receptor blockers, trimethoprim, drospirenone and other progestins, nephrotoxic antibiotics, penicillins, tacrolimus, or medications known to delay gastric emptying or otherwise interfere with absorption of study product.

Subjects who did not participate in Study B23CS and meet any of the following criteria will be considered ineligible for registration into this study:

  1. Female subject who is pregnant or lactating or has plans for pregnancy during the study;
  2. Subject has evidence of proximal tubule dysfunction unless the subject is < 6 months of age;
  3. Subject presents with another diagnosed condition as a potential etiology for her/his dRTA ;
  4. Subject requires therapy with potassium sparing diuretics, angiotensin converting enzyme inhibitors, angiotensin II receptor blockers, trimethoprim, drospirenone and other progestins, nephrotoxic antibiotics, penicillins, tacrolimus, or medications known to delay gastric emptying or otherwise interfere with absorption of study product;
  5. Subject has evidence of obstructive uropathy or other findings on renal ultrasound expected to require intervention during the course of the study;
  6. Subject has any of the following laboratory abnormalities:

    1. AST and/or ALT > 1.5x upper limit of normal (ULN)
    2. Serum potassium > 5.0 mEq/L or hypokalemia accompanied by clinical symptoms (eg, muscle cramps) or significant ECG changes (eg, T wave depression, U wave elevation)
    3. Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 (according to the updated Schwartz formula for children and Chronic Kidney Disease - Epidemiology Collaboration [CKD-EPI] formula for adults)
    4. Total bilirubin > ULN, except with known Gilbert's disease.
  7. Subject has been hospitalized or had outpatient surgery (other than minor skin and dRTA disease-related procedures or ear tube placement) in the past 6 months or is planning surgery in the next 6 months.

Sites / Locations

  • University of South Florida Pediatric Infectious Diseases

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Experimental ADV7103

Arm Description

All patients receive ADV7103 at their individualized dose

Outcomes

Primary Outcome Measures

The Safety of ADV7103 will be assessed by evaluating the frequency of Treatment-Emergent Adverse events as compared to placebo
Number/proportion of subjects presenting with ADV7103 treatment-related adverse events (AEs) during the study, by severity grade.

Secondary Outcome Measures

Full Information

First Posted
January 23, 2019
Last Updated
May 8, 2023
Sponsor
Advicenne Pharma
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1. Study Identification

Unique Protocol Identification Number
NCT03831152
Brief Title
Extension Study in Primary Distal Renal Tubular Acidosis
Official Title
A Phase 3B Open-Label Extension Of Study B23CS (ARENA 2) Evaluating The Continued Safety And Efficacy Of ADV7103 In Subjects With Primary Distal Renal Tubular Acidosis
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
December 2023 (Anticipated)
Primary Completion Date
January 2025 (Anticipated)
Study Completion Date
March 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Advicenne Pharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Open-label study involving longitudinal assessment of the continued safety, tolerability, and efficacy of ADV7103 in maintaining targeted serum bicarbonate levels, preventing metabolic acidosis, and preventing hypokalemia in the following groups of subjects with primary dRTA: subjects who participated in Study B23CS and were adherent to the protocol; subjects ≥ 6 months of age who are living in Europe and did not participate in Study B23CS; and infants younger than 6 months of age

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acidosis, Renal Tubular

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Experimental ADV7103
Arm Type
Experimental
Arm Description
All patients receive ADV7103 at their individualized dose
Intervention Type
Drug
Intervention Name(s)
ADV7103
Intervention Description
Single group assignment
Primary Outcome Measure Information:
Title
The Safety of ADV7103 will be assessed by evaluating the frequency of Treatment-Emergent Adverse events as compared to placebo
Description
Number/proportion of subjects presenting with ADV7103 treatment-related adverse events (AEs) during the study, by severity grade.
Time Frame
To 30 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Months
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subjects who participated in Study B23CS must meet all of the following criteria to be registered into this study: 1. Female or male subjects who participated in Study B23CS and were adherent to the protocol; Subjects who did not participate in Study B23CS must meet all of the following criteria to be registered into this study: Female or male subjects ≤ 65 years of age at time of consent with the following stipulations: Subjects ≥ 6 months of age must reside in Europe Subjects < 6 months of age must reside in the United States, Canada, or Europe; Subjects < 6 months of age must be able to swallow (not suck) solid food without difficulty; Subject presents with a previous diagnosis of primary dRTA; Subject ≥ 6 months of age requires ≥ 0.9 mEq/kg/day of alkali therapy to maintain serum bicarbonate levels above the LLN; Urine pH > 5.5 and serum bicarbonate > 18 mEq/L for subjects ≥ 4 years old or > 17 mEq/L for subjects < 4 years old on alkali therapy and potassium supplementation (if indicated) on at least one occasion for each within 6 months; European subjects must be included in a social health insurance system and/or in compliance with the recommendations of the national law in force relating to biomedical research; Subject or parent/guardian is willing and able to understand and sign informed consent and willing to comply with protocol instructions; child assent when appropriate; and Exclusion Criteria: Subjects who participated in Study B23CS and meet any of the following criteria will be considered ineligible for registration into this study: Female subject who is pregnant or lactating or has plans for pregnancy during the study; or Subject requires therapy with potassium sparing diuretics, angiotensin converting enzyme inhibitors, angiotensin II receptor blockers, trimethoprim, drospirenone and other progestins, nephrotoxic antibiotics, penicillins, tacrolimus, or medications known to delay gastric emptying or otherwise interfere with absorption of study product. Subjects who did not participate in Study B23CS and meet any of the following criteria will be considered ineligible for registration into this study: Female subject who is pregnant or lactating or has plans for pregnancy during the study; Subject has evidence of proximal tubule dysfunction unless the subject is < 6 months of age; Subject presents with another diagnosed condition as a potential etiology for her/his dRTA ; Subject requires therapy with potassium sparing diuretics, angiotensin converting enzyme inhibitors, angiotensin II receptor blockers, trimethoprim, drospirenone and other progestins, nephrotoxic antibiotics, penicillins, tacrolimus, or medications known to delay gastric emptying or otherwise interfere with absorption of study product; Subject has evidence of obstructive uropathy or other findings on renal ultrasound expected to require intervention during the course of the study; Subject has any of the following laboratory abnormalities: AST and/or ALT > 1.5x upper limit of normal (ULN) Serum potassium > 5.0 mEq/L or hypokalemia accompanied by clinical symptoms (eg, muscle cramps) or significant ECG changes (eg, T wave depression, U wave elevation) Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 (according to the updated Schwartz formula for children and Chronic Kidney Disease - Epidemiology Collaboration [CKD-EPI] formula for adults) Total bilirubin > ULN, except with known Gilbert's disease. Subject has been hospitalized or had outpatient surgery (other than minor skin and dRTA disease-related procedures or ear tube placement) in the past 6 months or is planning surgery in the next 6 months.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Andre Ulmann, M.D., Ph.D.
Organizational Affiliation
Advicenne Pharma
Official's Role
Study Chair
Facility Information:
Facility Name
University of South Florida Pediatric Infectious Diseases
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Extension Study in Primary Distal Renal Tubular Acidosis

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