Phase
Condition
Distal Renal Tubular Acidosis
Treatment
mRNA-3927
Clinical Study ID
All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2participants in Part 1.
≥1 year of age at the time of consent/assent if enrolled after the first 2participants.
Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (PCCAand/or PCCB mutations).
Part 2 only: At least one documented MDE in the 12-month period before consent.
Participants <1 Year of Age :
Identification by newborn screening shortly after birth or having suspected PA bypresenting with a spectrum of metabolic symptoms, and having a sibling diagnosedwith PA. Participant may enter the Screening Period while awaiting genetic testingresults, provided that all other eligibility criteria are met but would not beenrolled until diagnosis of PA is confirmed.
≥37 weeks gestational age at the time of birth without otherconditions/comorbidities that in the opinion of the Investigator may interfere withthe interpretation of study results.
Body weight ≥3 kg at Screening.
At least 1 documented PA-related event prior to Screening defined as the followingcriteria:
Clinical signs of metabolic deterioration consistent with PA (eg, vomiting, notfeeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion,abnormal movements including bicycling, abnormal tone, low body temperature,seizure[s]), OR
Meeting the criteria of MDE definition, OR
Evidence of laboratory abnormalities as evidenced by at least one of thefollowing:
Metabolic acidosis (decreased pH) with high anion gap, or compensated metabolicacidosis (reduced bicarbonate, or base deficit, or reduced PaCO2 or increasedlactate) with high anion gap.
Acute hyperammonemia.
Neutropenia or thrombocytopenia.
Exclusion
Exclusion Criteria:
Participants of all ages are excluded from the study if during Screening any of the following criteria apply:
Any individual with laboratory abnormalities achieving theresholds defined in theprotocol
Estimated glomerular filtration rate (eGFR) <30 milliliters (mL)/minute/1.73 squaremeter (m^2) for participants of all ages receiving chronic dialysis.
History of organ transplantation or planned organ transplantation during the periodof study participation.
Corrected QT interval (QTc) >480 milliseconds (ms) using Bazett's correction.
Grade 3 or 4 heart failure according to the Modified Ross Heart FailureClassification for Children or the New York Heart Association Classification.
Pregnant or breastfeeding.
Other clinically significant conditions that in the Investigator's opinion couldinterfere with the safety of the participant, the interpretation of study results,or limit the participation in the study.
Study Design
Study Description
Connect with a study center
Stollery Children's Hospital University of Alberta
Edmonton, Alberta T6G 2R7
CanadaActive - Recruiting
Hospital For Sick Children
Toronto, Ontario M5G 1X8
CanadaActive - Recruiting
Fujita Health University Hospital
Toyoake-shi, Aichi 470-1192
JapanActive - Recruiting
Tohoku University Hospital
Sendai-Shi, Miyagi 980-8574
JapanActive - Recruiting
Birmingham Children's Hospital
Birmingham, B4 6NH
United KingdomSite Not Available
University Hospital Birmingham NHS Foundation Trust
Birmingham, B15 2TH
United KingdomActive - Recruiting
Great Ormond Street Hospital (GOSH)
London, WC1N 3JH
United KingdomActive - Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
United KingdomActive - Recruiting
Manchester University NHS Foundation Trust
Manchester, M13 9WL
United KingdomActive - Recruiting
Willink Biochemical Genetics Unit
Manchester, M13 9WL
United KingdomActive - Recruiting
David Geffen School of Medicine UCLA
Los Angeles, California 90095
United StatesActive - Recruiting
Ronald Reagan UCLA Medical Center
Los Angeles, California 90095
United StatesActive - Recruiting
Lucile Packard Children's Hospital Stanford
Stanford, California 94304
United StatesActive - Recruiting
University of Stanford Medical Center
Stanford, California 94304
United StatesActive - Recruiting
Johns Hopkins Hospital
Baltimore, Maryland 21287
United StatesActive - Recruiting
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit
Baltimore, Maryland 21287
United StatesCompleted
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesCompleted
University of Michigan
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
University of Michigan Hospitals
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York, New York 10029
United StatesActive - Recruiting
Duke University Medical Center
Durham, North Carolina 27710
United StatesActive - Recruiting
Duke University Medical System (Duke Health)
Durham, North Carolina 27710
United StatesActive - Recruiting
Cincinnati Children's Hospital
Cincinnati, Ohio 45229
United StatesActive - Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesCompleted
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
Texas Children's Hospital
Houston, Texas 77030
United StatesActive - Recruiting


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