Phase
Condition
Distal Renal Tubular Acidosis
Phenylketonuria
Metabolic Disorders
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 in Part 1.
Confirmed diagnosis of PA based on diagnosis by molecular genetic testing viacentral laboratory (PCCA and/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.
For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestationalage at the time of birth without other conditions/comorbidities that in the opinionof the Investigator may interfere with the interpretation of study results.
Body weight ≥3 kilograms (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 (for example,vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence ofproper perfusion, abnormal movements including bicycling, abnormal tone, lowbody 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 with elevated 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 considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in theInvestigator or Sponsor's opinion that could interfere with or limit theparticipation in the study.
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
CanadaSite Not Available
Stollery Children's Hospital University of Alberta
Edmonton 5946768, Alberta 5883102 T6G 2R7
CanadaActive - Recruiting
Hospital For Sick Children
Toronto, Ontario M5G 1X8
CanadaSite Not Available
Hospital For Sick Children
Toronto 6167865, Ontario 6093943 M5G 1X8
CanadaActive - Recruiting
CHU de Marseille - Hôpital de la Timone
Marseille, 13005
FranceSite Not Available
CHU de Marseille - Hôpital de la Timone
Marseille 2995469, 13005
FranceSite Not Available
Hôpital Necker - Enfants Malades
Paris, 75019
FranceSite Not Available
Hôpital Necker - Enfants Malades
Paris 2988507, 75019
FranceActive - Recruiting
Fujita Health University Hospital
Toyoake-shi, Aichi-ken 1865694 470-1192
JapanActive - Recruiting
Tohoku University Hospital
Sendai-Shi, Miyagi 980-8574
JapanSite Not Available
Tohoku University Hospital
Sendai 2111149, Miyagi 2111888 980-8574
JapanActive - Recruiting
National Center for Child Health and Development
Tokyo, 113-8519
JapanSite Not Available
National Center for Child Health and Development
Tokyo 1850147, 113-8519
JapanSite Not Available
Erasmus MC
Rotterdam 2747891, South Holland 2743698 3015 GD
NetherlandsActive - Recruiting
Erasmus MC
Rotterdam, Zuid-Holland 3015 GD
NetherlandsSite Not Available
Universitair Medisch Centrum Utrecht - PPDS
Utrecht, 3584 CX
NetherlandsSite Not Available
Universitair Medisch Centrum Utrecht - PPDS
Utrecht 2745912, 3584 CX
NetherlandsSite Not Available
King Abdullah Children's Specialist Hospital
Riyadh, Ar Riya 14611
Saudi ArabiaSite Not Available
King Fahad Medical City
Riyadh, Ar Riya 11564
Saudi ArabiaSite Not Available
King Faisal Specialist Hospital & Research Center - Riyadh
Riyadh, Ar Riya 11211
Saudi ArabiaSite Not Available
King Abdullah Children's Specialist Hospital
Riyadh 108410, Ar Riya 14611
Saudi ArabiaSite Not Available
King Fahad Medical City
Riyadh 108410, Ar Riya 11564
Saudi ArabiaSite Not Available
King Faisal Specialist Hospital & Research Center - Riyadh
Riyadh 108410, Ar Riya 11211
Saudi ArabiaSite Not Available
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona 8950
SpainSite Not Available
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat 3122826, Barcelona 8950
SpainSite Not Available
Hospital Universitario Cruces
Barakaldo, Biscay 48903
SpainSite Not Available
Hospital Universitario Cruces
Barakaldo 3109453, Biscay 48903
SpainActive - Recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
SpainSite Not Available
Hospital Universitario 12 de Octubre
Madrid 3117735, 28041
SpainActive - Recruiting
Hospital Universitario Virgen del Rocio - PPDS
Sevilla, 41013
SpainSite Not Available
Hospital Universitario Virgen del Rocio - PPDS
Seville 2510911, 41013
SpainSite Not Available
Birmingham Children's Hospital
Birmingham, B4 6NH
United KingdomCompleted
University Hospital Birmingham NHS Foundation Trust
Birmingham, B15 2TH
United KingdomSite Not Available
Birmingham Children's Hospital
Birmingham 2655603, B4 6NH
United KingdomCompleted
University Hospital Birmingham NHS Foundation Trust
Birmingham 2655603, 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 KingdomSite Not Available
Great Ormond Street Hospital for Children NHS Foundation Trust
London 2643743, 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
Willink Biochemical Genetics Unit - PPDS
Manchester, M13 9WL
United KingdomSite Not Available
Willink Biochemical Genetics Unit - PPDS
Manchester 2643123, 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
UCSD Altman Clinical and Transalational Research Institute Building
Los Angeles, California 90027
United StatesSite Not Available
Lucile Packard Children's Hospital Stanford
Stanford, California 94304
United StatesSite Not Available
University of Stanford Medical Center
Stanford, California 94304
United StatesActive - Recruiting
Ronald Reagan UCLA Medical Center
Los Angeles 5368361, California 5332921 90095
United StatesActive - Recruiting
UCSD Altman Clinical and Transalational Research Institute Building
Los Angeles 5368361, California 5332921 90027
United StatesSite Not Available
Lucile Packard Children's Hospital Stanford
Stanford 5398563, California 5332921 94304
United StatesActive - Recruiting
Nicklaus Children's Hospital
Miami, Florida 33155
United StatesSite Not Available
University of South Florida - 12901 Bruce B Downs
Tampa, Florida 33606-3603
United StatesSite Not Available
Nicklaus Children's Hospital
Miami 4164138, Florida 4155751 33155
United StatesSite Not Available
University of South Florida - 12901 Bruce B Downs
Tampa 4174757, Florida 4155751 33606-3603
United StatesSite Not Available
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago 4887398, Illinois 4896861 60611
United StatesActive - Recruiting
Johns Hopkins Hospital
Baltimore, Maryland 21287
United StatesActive - Recruiting
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit
Baltimore, Maryland 21287
United StatesSite Not Available
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit
Baltimore 4347778, Maryland 4361885 21287
United StatesCompleted
Boston Children's Hospital
Boston, Massachusetts 02115
United StatesSite Not Available
Boston Children's Hospital
Boston 4930956, Massachusetts 6254926 02115
United StatesCompleted
University of Michigan
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
University of Michigan Hospitals
Ann Arbor, Michigan 48109
United StatesSite Not Available
University of Michigan Hospitals
Ann Arbor 4984247, Michigan 5001836 48109
United StatesActive - Recruiting
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York, New York 10029
United StatesSite Not Available
Icahn School of Medicine at Mount Sinai - Clinical Research Unit
New York 5128581, New York 5128638 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 StatesSite Not Available
Duke University Medical System (Duke Health)
Durham 4464368, North Carolina 4482348 27710
United StatesActive - Recruiting
Ann and Robert H Lurie Childrens Hospital of Chicago
Cincinnati, Ohio 45229-3026
United StatesSite Not Available
Cincinnati Children's Hospital
Cincinnati, Ohio 45229
United StatesActive - Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesCompleted
University Hospitals Cleveland Medical Center - 11100 Euclid Ave
Cleveland, Ohio 44106-2624
United StatesSite Not Available
Cincinnati Children's Hospital Medical Center
Cincinnati 4508722, Ohio 5165418 45229
United StatesCompleted
University Hospitals Cleveland Medical Center - 11100 Euclid Ave
Cleveland 5150529, Ohio 5165418 44106-2624
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
Children's Hospital of Philadelphia (CHOP)
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesActive - Recruiting
Texas Children's Hospital
Houston, Texas 77030
United StatesSite Not Available
Texas Children's Hospital
Houston 4699066, Texas 4736286 77030
United StatesActive - Recruiting
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.