Phase
Condition
Distal Renal Tubular Acidosis
Arginase Deficiency
Metabolic Disorders
Treatment
Veverimer
Placebo
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Written informed consent.
≥ 18 years old (male/female)).
CKD with eGFR < 60 mL/min/1.73m²; not expected to need dialysis/ transplant duringstudy.
2 SBC values 12-21 mmol/L within 6 months pre-screening
During screening: 2 central SBC values 12-21 mmol/L
Willing to maintain stable diet .
Expect to keep oral alkali therapy dose stable.
Women of childbearing potential: negative pregnancy test and agree to abstinence orcontraception.
Exclusion
Exclusion Criteria:
Any participant deemed by the Investigator to be an inappropriate candidate forphysical performance testing (e.g., severe musculoskeletal pain, non-ambulatorystatus) or with a screening STS5 time < 10 seconds (i.e., very mobile).
Any participant deemed by the Investigator to be an inappropriate candidate for CPET (e.g., advanced chronic obstructive pulmonary disease [COPD], major cardiovascular [CV] event in last 6 months, systolic blood pressure [SBP] > 200 mmHg or diastolicblood pressure [DBP] > 120 mmHg). Only applicable to sites performing CPET and ifthe participant will take part in CPET.
History or current diagnosis of:
Clinically significant gastroparesis or a history of bariatric surgery.
Bowel obstruction, swallowing disorders, severe gastrointestinal disorders,including inflammatory bowel disease, major gastrointestinal surgery, or knownactive gastric/duodenal ulcers.
Severe recurrent diarrhea or severe recurrent constipation, in the opinion ofthe Investigator.
Pernicious anemia, atrophic or autoimmune gastritis, achlorhydria orhypochlorhydria.
Active Helicobacter pylori infection at screening.
Active, recurrent, or metastatic malignancy at the start of screening.
History of malignancy, except under the following conditions:
Carcinoma in situ (e.g., of the cervix, breast, or bladder) that has beencompletely excised and shows no evidence of residual disease.
Non-melanoma skin cancers (e.g., basal cell carcinoma, squamous cell carcinoma)that have been completely excised and show no evidence of recurrence.
Low grade prostate cancer, in the opinion of the Investigator (i.e., nometastasis, Gleason score < 6), with no significant worsening for > 6 monthsprior to the screening visit.
Any other malignancy that was treated with curative intent and has been incomplete remission for ≥ 5 years prior to the screening visit.
Evidence of acute fluid overload or history of recurrent fluid overload, in theopinion of the Investigator.
Screening hemoglobin < 10 g/dL.
Presence of primary respiratory alkalosis, as assessed by venous blood gas (VBG)analysis at time of screening.
Serum gastrin level > 500 pg/mL.
Investigational medication administration within 28 days prior to start ofscreening.
Use of GI polymer binders or sodium zirconium cyclosilicate within 28 days prior tothe start of screening or have an expectation to initiate treatment during thestudy.
Use of acid reducing drugs, including potassium competitive acid blockers,H2-blockers or PPIs within 28 days prior to the start of screening or have anexpectation to initiate treatment during the study.
Use of GLP-1 inhibitors within 6 months prior to the start of screening or have anexpectation to initiate treatment during the study.
Participants that are taking any of the following medications and have not been on astable dose for at least 28 days prior to screening or have an expectation to changedose during the study: diuretics, non-ophthalmic carbonic anhydrase inhibitors,diabetes drugs, RAAS inhibitors, calcium or magnesium supplements, non polymerphosphate binders, and SGLT-2 inhibitors. These medications also should not bestarted during the study.
Participants that are taking more than 30 units of insulin daily.
History of alcoholism or drug/chemical abuse within 1 year prior to the start ofscreening, in the opinion of the Investigator.
Current, regular use of inhaled/ingested cannabis/THC products.
Inability to take the IP or otherwise comply with the protocol.
Any medical condition, uncontrolled systemic disease or serious concurrent illnessthat would significantly decrease study compliance or jeopardize the safety of theparticipant or affect the validity of the trial results, in the opinion of theInvestigator.
Study Design
Connect with a study center
Academic Medical Research Institute
Los Angeles, California 90022
United StatesActive - Recruiting
Research Site
Los Angeles, California 90022
United StatesActive - Recruiting
Research Site
Edgewater, Florida 32132
United StatesActive - Recruiting
Velocity Clinical Research
Edgewater, Florida 32132
United StatesActive - Recruiting
Belkis Delgado MD PA
Miami Springs, Florida 33166
United StatesActive - Recruiting
Research Site
Miami Springs, Florida 33166
United StatesActive - Recruiting
Research Site
Miami Springs 4164223, Florida 4155751 33166
United StatesSite Not Available
Research Site
Chicago, Illinois 60643
United StatesActive - Recruiting
Research by Design
Chicago, Illinois 60643
United StatesActive - Recruiting
Research Site
Chicago 4887398, Illinois 4896861 60643
United StatesSite Not Available
Indiana University School of Medicine
Indianapolis, Indiana 46202
United StatesActive - Recruiting
Nephrology Associates of Kentuckiana
Louisville, Kentucky 40205
United StatesActive - Recruiting
Research Site
Louisville, Kentucky 40205
United StatesActive - Recruiting
Research Site
Louisville 4299276, Kentucky 6254925 40205
United StatesSite Not Available
New York-Presbyterian Queens
Fresh Meadows, New York 11365
United StatesActive - Recruiting
Brookview Hills Research Associates
Winston-Salem, North Carolina 27103
United StatesActive - Recruiting
Clinical Advancement Center
San Antonio, Texas 78212
United StatesActive - Recruiting
Research Site
San Antonio, Texas 78212
United StatesActive - Recruiting
Research Site
San Antonio 4726206, Texas 4736286 78212
United StatesSite Not Available

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