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Extension Study in Primary Distal Renal Tubular Acidosis

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ClinicalTrials.gov Identifier: NCT03831152
Recruitment Status : Recruiting
First Posted : February 5, 2019
Last Update Posted : September 6, 2019
Sponsor:
Information provided by (Responsible Party):
Advicenne Pharma

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:

  1. subjects who participated in Study B23CS and were adherent to the protocol;
  2. subjects ≥ 6 months of age who are living in Europe and did not participate in Study B23CS; and
  3. infants younger than 6 months of age

Condition or disease Intervention/treatment Phase
Acidosis, Renal Tubular Drug: ADV7103 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : August 21, 2019
Estimated Primary Completion Date : March 30, 2021
Estimated Study Completion Date : March 30, 2021


Arm Intervention/treatment
Experimental: Experimental ADV7103
All patients receive ADV7103 at their individualized dose
Drug: ADV7103
Single group assignment




Primary Outcome Measures :
  1. The Safety of ADV7103 will be assessed by evaluating the frequency of Treatment-Emergent Adverse events as compared to placebo [ Time Frame: To 30 months ]
    Number/proportion of subjects presenting with ADV7103 treatment-related adverse events (AEs) during the study, by severity grade.



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Months to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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:

  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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03831152


Locations
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United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: Christy Kang    404-712-9998    christy.kang@emory.edu   
Principal Investigator: Laurence Greenbaum, MD         
Sponsors and Collaborators
Advicenne Pharma
Investigators
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Principal Investigator: Larry Greenbaum, M.D. Emory University

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Responsible Party: Advicenne Pharma
ClinicalTrials.gov Identifier: NCT03831152     History of Changes
Other Study ID Numbers: B24CS
First Posted: February 5, 2019    Key Record Dates
Last Update Posted: September 6, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Acidosis, Renal Tubular
Acidosis
Acid-Base Imbalance
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn