Distal Renal Tubular AcidosisPhase 3NCT03644706
Study Evaluating Subjects With Distal Renal Tubular Acidosis
A Phase 3 study testing ADV7103 in people with Distal Renal Tubular Acidosis, run by Advicenne Pharma at 8 sites in 2 countries.
Trial summary
This is a phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled withdrawal study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis in pediatric and adult subjects with primary Distal Renal Tubular Acidosis (dRTA). Read more about this study
- Who is it for?
- Ages 6 months to 65 years
- Conditions the study is for
- Distal Renal Tubular Acidosis
- What is being tested?
- ADV7103
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 3 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 20 Sep 2021to 20 Dec 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Female or male subjects ≥ 6 months of age and ≤ 65 years of age at time of consent;
- Subject presents with a previous diagnosis of primary dRTA of at least 4 months duration for subjects < 12 years of age, and at least one year for those ≥ 12 years of age, based on documented history of non-anion gap, hyperchloremic, hypokalemic metabolic acidosis;
- Subject requires ≥ 0.9 mEq/kg/day of alkali therapy to maintain serum bicarbonate levels above the LLN for the laboratory providing results;
- 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
- Heterosexually active female subjects of childbearing potential and non-sterilized males must use at least one of the following acceptable birth control methods from informed consent through 7 days after the last dose of study product:
- Double-barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository)
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- Established use of oral, injectable, or implanted hormonal methods of contraception
- Placement of an intrauterine device or intrauterine system
- Abstinence Females of childbearing potential are those who have reached the onset of menarche (or 8 years of age, whichever comes first) and are not postmenopausal (≥ 1 year without menses prior to Visit 1), surgically sterile, or status post hysterectomy (≥ 1 month prior to Visit 1). From informed consent through 7 days after the last dose of study product, female subjects must agree to refrain from egg donation and male subjects must agree to refrain from sperm donation.
Who cannot join
- Female subject who is pregnant or lactating or has plans for pregnancy during the study;
- Subject has evidence of proximal tubule dysfunction (eg, hypophosphatemia, low serum uric acid, glycosuria, or amino aciduria);
- Subject presents with another diagnosed condition as a potential etiology for her/his dRTA (eg, systemic lupus erythematosus, Sjogren's syndrome), in the opinion of the Investigator;
- 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 associated with Visit 1 expected to require intervention during the course of the study, in the opinion of the Investigator;
- Subject has any of the following laboratory abnormalities associated with Visit 1:
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- AST and/or ALT > 1.5x upper limit of normal (ULN)
- Serum potassium > 5.0 mEq/L or <3.0 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 Modified Schwartz formula for children [<12years of age] and Chronic Kidney Disease - Epidemiology Collaboration [CKD-EPI] equation for adolescents and adults [≥12 years of age])
- Total bilirubin > ULN, except with known Gilbert's disease or in patients with dRTA and known hemolytic anemia due to a defect in SLC4A1.
- 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;
- In the opinion of the Investigator, the subject has a major medical or psychiatric condition (eg, significant cardiac disease, schizophrenia) or an unstable condition (eg, uncontrolled hypertension, asthma, diabetes, hypercholesterolemia, or cardiac disease) that would potentially interfere with the subject safely completing the study;
- In the opinion of the Investigator, the subject has a history of difficulty taking oral medication and/or conditions that may hinder absorption of the study drug (eg, any difficulty of swallowing, malabsorption, delayed gastric emptying, esophageal compression, intestinal obstruction, or other chronic gastrointestinal disease);
- Self-reported or parent/guardian reported alcohol abuse or drug abuse within the past 12 months;
- Subject is a solid organ or bone marrow transplant recipient;
- Subject has a history of malignancy within 5 years prior to Visit 1, except for localized skin or cervical carcinoma; or
- Subject is known to have allergy or intolerance to any ADV7103 or placebo constituents.
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States6 sites
Emory Children's Center
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
J.W. Riley Hospital for Children/Indiana University
Indianapolis, Indiana, 46202
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of South Florida Pediatric Infectious Disease
Tampa, Florida, 33606
Montefiore Medical Center
The Bronx, New York, 10467
Canada2 sites
University of Alberta
Edmonton, Alberta, T6G 2R3
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
About this study
Official title: A Phase 3 Multicenter, Randomized, Double-Blinded, Placebo-Controlled Withdrawal Study Evaluating ADV7103 In Pediatric and Adult Subjects With Distal Renal Tubular Acidosis (dRTA)
This is a phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled withdrawal study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis in pediatric and adult subjects with primary Distal Renal Tubular Acidosis (dRTA).
Read the detailed description
The study will target enrolling at least 4 subjects in each of the following age groups: 6 months - 23 months; 2-11 years, and ≥ 12 years. Subjects will be in the study for up to 21 weeks. After screening and enrollment, subjects will participate in an 8-12 week open label period where there dose of ADV7103 will be titrated to effect, then continued for the remainder of the open-label period. Periodic measurements of bicarbonate and potassium levels will be collected during this period. Following the open-label period, subjects will enter a 6-day randomized withdrawal period. A follow-up period up to four weeks on re-established therapy completes the trial. Subjects can elect to return to their previous standard of care regimen after completing the withdrawal period or will have the opportunity to subsequently enter an open-label extension study.
- Conditions
- Distal Renal Tubular Acidosis
- Being tested
- ADV7103, Placebo
- Medical subject headings
- Acidosis, Renal Tubular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADV7103Drug
Also called Potassium Citrate and Potassium Bicarbonate
Each dose of ADV7103 contains a fixed ratio of 1/3 of ADV7103-CK (potassium citrate) and 2/3 of ADV7103-BK (potassium bicarbonate) based on the mass of active substances. The strength is 6.44 (± 10 %) mEq/g of ADV7103 (alkalinizing power).
PlaceboDrug
Placebo is a combination of 2 mm green coated lactose granules and 2 mm white coated lactose granules to be taken by mouth.
Each dose of placebo contains a fixed ratio of 1/3 of green granules and 2/3 of white granules.
| Study group | Role | What they receive |
|---|---|---|
| ADV7103 | Active comparator | Patients continue to receive ADV7103 twice a day at their open label dose over 6 days Receives: Drug: ADV7103 |
| Placebo Comparator | Placebo comparator | Patients receive matched placebo twice a day until they reach a bicarbonate level of 18mEq/L Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Blood Bicarbonate Levels During Withdrawal Period
Blood bicarbonate levels were measured at the begininning (day 1) and end (day 6) of the withdrawal period, and change in blood bicarbonate levels was calculated as value at day 6 minus value at day 1
Measured: day 1, day 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2018
- Study start20 Sep 2021
- Primary completion20 Dec 2023
- Study completion20 Dec 2023
Why it stopped: COVID-19 pandemic negatively impacted the ability to enroll patients.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Distal Renal Tubular AcidosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Advicenne Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Laurence Greenbaum, MD, Ph.D., Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03644706
- Sponsor's protocol ID
- B23CS
- Status last verified
- Jun 2026
- First submitted
- 22 Aug 2018
- Last updated
- 3 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Multicenter, Randomized, Double-Blinded, Placebo-Controlled Withdrawal Study Evaluating ADV7103 In Pediatric and Adult Subjects With Distal Renal Tubular Acidosis (dRTA)
- ClinicalTrials.gov ID
- NCT03644706
- Acronym / sponsor ID
- B23CS
- Conditions
- Distal Renal Tubular Acidosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: COVID-19 pandemic negatively impacted the ability to enroll patients.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Advicenne PharmaIndustry (pharma, biotech, devices)
- Study start
- 20 Sep 2021 (actual)
- Primary completion
- 20 Dec 2023 (actual)
- Study completion
- 20 Dec 2023 (actual)
- First posted
- 23 Aug 2018 (actual)
- Results
- Posted 3 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled withdrawal study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis in pediatric and adult subjects with primary Distal Renal Tubular Acidosis (dRTA).
Detailed description
The study will target enrolling at least 4 subjects in each of the following age groups: 6 months - 23 months; 2-11 years, and ≥ 12 years. Subjects will be in the study for up to 21 weeks. After screening and enrollment, subjects will participate in an 8-12 week open label period where there dose of ADV7103 will be titrated to effect, then continued for the remainder of the open-label period. Periodic measurements of bicarbonate and potassium levels will be collected during this period. Following the open-label period, subjects will enter a 6-day randomized withdrawal period. A follow-up period up to four weeks on re-established therapy completes the trial. Subjects can elect to return to their previous standard of care regimen after completing the withdrawal period or will have the opportunity to subsequently enter an open-label extension study.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ADV7103 | Active comparator | Drug: ADV7103Patients continue to receive ADV7103 twice a day at their open label dose over 6 days |
| Placebo Comparator | Placebo comparator | Drug: PlaceboPatients receive matched placebo twice a day until they reach a bicarbonate level of 18mEq/L |
Who may qualify
- Ages
- 6 months to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Female or male subjects ≥ 6 months of age and ≤ 65 years of age at time of consent;
- Subject presents with a previous diagnosis of primary dRTA of at least 4 months duration for subjects < 12 years of age, and at least one year for those ≥ 12 years of age, based on documented history of non-anion gap, hyperchloremic, hypokalemic metabolic acidosis;
- Subject requires ≥ 0.9 mEq/kg/day of alkali therapy to maintain serum bicarbonate levels above the LLN for the laboratory providing results;
- 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
- Heterosexually active female subjects of childbearing potential and non-sterilized males must use at least one of the following acceptable birth control methods from informed consent through 7 days after the last dose of study product:
Key exclusion criteria (17 listed)
- Female subject who is pregnant or lactating or has plans for pregnancy during the study;
- Subject has evidence of proximal tubule dysfunction (eg, hypophosphatemia, low serum uric acid, glycosuria, or amino aciduria);
- Subject presents with another diagnosed condition as a potential etiology for her/his dRTA (eg, systemic lupus erythematosus, Sjogren's syndrome), in the opinion of the Investigator;
- 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 associated with Visit 1 expected to require intervention during the course of the study, in the opinion of the Investigator;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ADV7103Also: Potassium Citrate and Potassium BicarbonateEach dose of ADV7103 contains a fixed ratio of 1/3 of ADV7103-CK (potassium citrate) and 2/3 of ADV7103-BK (potassium bicarbonate) based on the mass of active substances. The strength is 6.44 (± 10 %) mEq/g of ADV7103 (alkalinizing power). | Drug | ADV7103 |
| PlaceboPlacebo is a combination of 2 mm green coated lactose granules and 2 mm white coated lactose granules to be taken by mouth. Each dose of placebo contains a fixed ratio of 1/3 of green granules and 2/3 of white granules. | Drug | Placebo Comparator |
Primary outcome
Change in Blood Bicarbonate Levels During Withdrawal Period
Time frame: day 1, day 6
Blood bicarbonate levels were measured at the begininning (day 1) and end (day 6) of the withdrawal period, and change in blood bicarbonate levels was calculated as value at day 6 minus value at day 1
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Patient educationLink provided by the sponsor.
- Company websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laurence Greenbaum, MD, Ph.D. principal investigator
Emory University
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ADV7103 in people with Distal Renal Tubular Acidosis, run by Advicenne Pharma at 8 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Blood Bicarbonate Levels During Withdrawal Period (time frame: day 1, day 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2026.