Bartter SyndromeAZNCT03847571
Acetazolamide (AZ) for Management of Alkalosis in Bartter Syndrome
An observational study of Bartter Syndrome, run by Tehran University of Medical Sciences at 4 sites in Iran.
Trial summary
In this prospective controlled cross over clinical trial, the investigators aim to evaluate the efficacy and safety of acetazolamide for the management of metabolic alkalosis in children with Bartter syndrome. Urine and blood electrolytes will be measured before and after acetazolamide treatment. Read more about this study
- Who is it for?
- Ages 1 year to 10 years
- Conditions the study is for
- Bartter Syndrome
- What is being tested?
- Acetazolamide
- Type of treatment
- Pill or oral medicine
- How many people
- 20 participants
- Where
- 4 sitesIran
- Study dates
- 10 Jan 2019to 30 Dec 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 year to 10 years
- All sexes
- No healthy volunteers
- Child
Study population: Children between 1 and 10 years of age with failure to thrive, polyuria and unexplained hypokalemic metabolic alkalosis will be recruited for this trial.
Who may be able to join
- Hypokalemia
- metabolic alkalosis
- normal blood pressure
- random urine chloride >20 milliequivalent per liter (mEq/L)
- Elevated serum aldosterone and renin levels
Who cannot join
- Hypertension
- History of emesis
- Prior use of laxatives
- Cystic fibrosis ofpancrease
Trial locations and contacts
4 sites in 1 country.
Iran4 sites, 4 recruiting
- Recruiting
Fateme Ghane Sharbaf
Mashhad
Fateme Ghane Sharbaf, MD, 00 98 9153112139, ghanesharbaf@gmail.com
Farahnak Assadi, 3125600477, fassadi@rush.edu
- Recruiting
Semnan University of Medical Sciences
Semnan, 011000
Mojgan Mazaheri, MD, 9123069789, mojganmazaheri@yahoo.com
Farahnak Assadi, MD, 3125600477, fassadi@rush.edu
- Not yet recruiting
Banafshe Dormansh
Tehran
Banafshe Dormanesh, MD, 00 98 9123886725, dr.dormansh@yahoo.com
Farahnak Assadi, MD, 3125600477, Farahnak_Assadi@rush.edu
- Recruiting
Simin Sadeghi
Zahedan
Simin Sadeghi, MD, 00 98 54533295573, sisadegh@yahoo.com
Farahnak Assadi, MD, 3125600477, fassadi@rush.edu
About this study
Official title: Acetazolamide (AZ) for Management of Refractory Hypokalemia Metabolic Alkalosis in Bartter Syndrome
In this prospective controlled cross over clinical trial, the investigators aim to evaluate the efficacy and safety of acetazolamide for the management of metabolic alkalosis in children with Bartter syndrome. Urine and blood electrolytes will be measured before and after acetazolamide treatment. The primary end point is a change in polyuria, hypokalemia, and metabolic alkalosis.
Read the detailed description
Bartter syndrome is a hereditary salt-loosing tubulopathy caused by several gene mutations encoding the sodium reabsorption in the thick ascending limb of loop of Henle, with poor response to treatment. The effects of inhibition of proximal tubular reabsorption of bicarbonate by acetazolamide have not been previously studied in Batter patients.
The present study is designed to assess he efficacy and safety of acetazolamide for the management of children with Bartter syndrome. The primary end point is change in polyuria, hypokalemia, and metabolic alkalosis.
In this prospective observational crossover clinical trial, patients between ages 1 and 10 years with clinical diagnosis of Bartter syndrome (hypokalemia, metabolic alkalosis, normal blood pressure, elevated urine chloride >20 milliequivalent per liter, high serum aldosterone and plasma renin levels) will be enrolled in a 4- week clinical trial. After initial clinical and laboratory evaluations, patients will receive acetazolamide 5.0 mg/kg orally as a single daily dose and each patient will act as his/her own control. Renal electrolyte and 24-hour urine output will be measured at baseline and after the 4 weeks acetazolamide treatment.
- Conditions
- Bartter Syndrome
- Being tested
- Acetazolamide
- Keywords
- Acetazolamide, Hypokalemic metabolic alkalosis, Bartter syndrome
- Medical subject headings
- Bartter Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AcetazolamideDrug
Correction of metabolic alkalosis by inhibition of the filtered bicarbonate load reabsorption in the proximal tubules using acetazolamide (AZ)
| Study group | Role | What they receive |
|---|---|---|
| Acetazolamide | Oral administration of acetazolamide 5 mg/kg/day for 4 weeks Receives: Drug: Acetazolamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Metabolic alkalosis
Change in serum bicarbonate level
Measured: 4 weeks
Urine output
Change in 24-hr urine volume
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2019
- Study start10 Jan 2019
- Primary completion30 Oct 2019 (estimated)
- Study completion30 Dec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bartter SyndromeStudies 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
- Observational
- Observational model
- Case crossover
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tehran University of Medical Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Farahnak Assadi, MD, Study director, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03847571
- Sponsor's protocol ID
- Acetazolamide (AZ)
- Acronym
- AZ
- Status last verified
- Feb 2019
- First submitted
- 16 Feb 2019
- Last updated
- 20 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Acetazolamide (AZ) for Management of Refractory Hypokalemia Metabolic Alkalosis in Bartter Syndrome
- ClinicalTrials.gov ID
- NCT03847571
- Acronym / sponsor ID
- AZ / Acetazolamide (AZ)
- Conditions
- Bartter Syndrome
- Keywords
- Acetazolamide, Hypokalemic metabolic alkalosis, Bartter syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Tehran University of Medical SciencesUniversity, hospital or other
- Study start
- 10 Jan 2019 (actual)
- Primary completion
- 30 Oct 2019 (estimated)
- Study completion
- 30 Dec 2019 (estimated)
- First posted
- 20 Feb 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this prospective controlled cross over clinical trial, the investigators aim to evaluate the efficacy and safety of acetazolamide for the management of metabolic alkalosis in children with Bartter syndrome. Urine and blood electrolytes will be measured before and after acetazolamide treatment. The primary end point is a change in polyuria, hypokalemia, and metabolic alkalosis.
Detailed description
Bartter syndrome is a hereditary salt-loosing tubulopathy caused by several gene mutations encoding the sodium reabsorption in the thick ascending limb of loop of Henle, with poor response to treatment. The effects of inhibition of proximal tubular reabsorption of bicarbonate by acetazolamide have not been previously studied in Batter patients.
The present study is designed to assess he efficacy and safety of acetazolamide for the management of children with Bartter syndrome. The primary end point is change in polyuria, hypokalemia, and metabolic alkalosis.
In this prospective observational crossover clinical trial, patients between ages 1 and 10 years with clinical diagnosis of Bartter syndrome (hypokalemia, metabolic alkalosis, normal blood pressure, elevated urine chloride >20 milliequivalent per liter, high serum aldosterone and plasma renin levels) will be enrolled in a 4- week clinical trial. After initial clinical and laboratory evaluations, patients will receive acetazolamide 5.0 mg/kg orally as a single daily dose and each patient will act as his/her own control. Renal electrolyte and 24-hour urine output will be measured at baseline and after the 4 weeks acetazolamide treatment.
Study design
- Observational model
- Case crossover
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Acetazolamide | – | Drug: AcetazolamideOral administration of acetazolamide 5 mg/kg/day for 4 weeks |
Who may qualify
- Ages
- 1 year to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children between 1 and 10 years of age with failure to thrive, polyuria and unexplained hypokalemic metabolic alkalosis will be recruited for this trial.
Key inclusion criteria (5 listed)
- Hypokalemia
- metabolic alkalosis
- normal blood pressure
- random urine chloride >20 milliequivalent per liter (mEq/L)
- Elevated serum aldosterone and renin levels
Key exclusion criteria (4 listed)
- Hypertension
- History of emesis
- Prior use of laxatives
- Cystic fibrosis ofpancrease
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 |
|---|---|---|
| AcetazolamideCorrection of metabolic alkalosis by inhibition of the filtered bicarbonate load reabsorption in the proximal tubules using acetazolamide (AZ) | Drug | Acetazolamide |
Primary outcome
Metabolic alkalosis
Time frame: 4 weeks
Change in serum bicarbonate level
Urine output
Time frame: 4 weeks
Change in 24-hr urine volume
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2019.
Related trials for Bartter Syndrome
RecruitingObservational study
National Registry of Rare Kidney Diseases
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
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.
Farahnak Assadi, MD study director
Rush University Medical Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Bartter Syndrome, run by Tehran University of Medical Sciences at 4 sites in Iran.
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 5 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 20 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Iran. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Farahnak Assadi, MD (3125600477, fassadi@rush.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Metabolic alkalosis (time frame: 4 weeks). The study lists 2 primary outcomes in all. 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 20 Feb 2019.