Phase
Condition
Phenylketonuria
Distal Renal Tubular Acidosis
Treatment
mRNA-3705
Placebo
Clinical Study ID
Ages > 1 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
(Part 1 only) Participant has a body weight of ≥11.0 kilograms at the screeningvisit.
Participant has a diagnosis of isolated MMA due to MUT deficiency confirmed bymolecular genetic testing.
Participant has a blood vitamin B12 level equal to or above the lower limit ofnormal (based on laboratory reference range) confirmed in the screening period.
Participant or their legally authorized representative is willing and able toprovide informed consent and/or assent as mandated by local regulations and iswilling and able to comply with study-related assessments.
Sexually active participants of childbearing or reproductive potential agree to usea highly effective method of contraception, consistent with local regulations,during the study and for 3 months after the last administration of study drug.
(Part 2 only) Participants with 2 screening MMA levels ≥400 micromolar.
(Parts 2 and 3 only) Participant is ≥5 years of age at the time of informedconsent/assent.
Exclusion
Key Exclusion Criteria:
Participant has a diagnosis of isolated MMA cofactor adenosyl-cobalamin (cb1A, cb1B,or cb1D) enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combinedMMA with homocystinuria.
Participant has previously received gene therapy for the treatment of MMA.
Participant has a history of organ transplantation or planned organ transplantationduring the period of study participation.
Participant has an active, unstable, or clinically significant medical condition notrelated to MMA or history of noncompliance that, in the investigator's opinion,could potentiate the risk while participating in this study, interfere with theinterpretation of study results, or limit the participant's participation in thestudy. This may include, but is not limited to, history of relevant food or drugallergies; history of cardiovascular, central nervous, gastrointestinal, orinfectious disease; history of clinically significant pathology; and/or history ofcancer.
(Part 2 only) Participant has the partial MUT deficiency disease phenotype, asassessed by genotyping, clinical phenotype/presentation, or vitamin B12-responsiveMMA.
Note: Additional inclusion/exclusion criteria may apply, per protocol.
Study Design
Study Description
Connect with a study center
Children's Hospital at Westmead
Westmead, New South Wales 2145
AustraliaSite Not Available
Children's Hospital at Westmead
Westmead 2143973, New South Wales 2155400 2145
AustraliaSite Not Available
Royal Children's Hospital Melbourne
Parkville, Victoria 3052
AustraliaSite Not Available
Royal Children's Hospital Melbourne
Parkville 2153770, Victoria 2145234 3052
AustraliaSite Not Available
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
Hôpital Necker - Enfants Malades
Paris, 75015
FranceSite Not Available
Hôpital Necker - Enfants Malades
Paris 2988507, 75015
FranceActive - Recruiting
Erasmus MC
Rotterdam, 3015 AA
NetherlandsSite Not Available
Erasmus MC
Rotterdam 2747891, 3015 AA
NetherlandsActive - Recruiting
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
NetherlandsSite Not Available
Universitair Medisch Centrum Utrecht
Utrecht 2745912, 3584 CX
NetherlandsActive - Recruiting
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona 08950
SpainSite Not Available
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat 3122826, Barcelona 08950
SpainActive - Recruiting
Hospital Universitario Cruces
Barakaldo, 48903
SpainSite Not Available
Hospital Universitario Cruces
Barakaldo 3109453, 48903
SpainActive - Recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
SpainSite Not Available
Hospital Universitario 12 de Octubre
Madrid 3117735, 28041
SpainActive - Recruiting
CHUS - H. Clinico U. de Santiago
Santiago de Compostela, 15706
SpainSite Not Available
CHUS - H. Clinico U. de Santiago
Santiago de Compostela 3109642, 15706
SpainActive - Recruiting
Hospital Universitario Virgen del Rocio - PPDS
Sevilla, 41013
SpainSite Not Available
Hospital Universitario Virgen del Rocio - PPDS
Seville 2510911, 41013
SpainActive - Recruiting
Birmingham Children's Hospital NHS Foundation Trust
Birmingham,
United KingdomSite Not Available
Birmingham Children's Hospital NHS Foundation Trust
Birmingham 2655603,
United KingdomTerminated
Royal Manchester Childrens Hospital
Manchester, M13 9WL
United KingdomSite Not Available
Royal Manchester Children's Hospital
Manchester 2643123, M13 9WL
United KingdomActive - Recruiting
UCLA Medical Center
Los Angeles, California 90095
United StatesSite Not Available
Lucile Packard Children's Hospital at Stanford
Palo Alto, California 94304
United StatesSite Not Available
UCLA Medical Center
Los Angeles 5368361, California 5332921 90095
United StatesActive - Recruiting
Lucile Packard Children's Hospital at Stanford
Palo Alto 5380748, California 5332921 94304
United StatesActive - Recruiting
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
The Children's Hospital of Philadelphia
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesActive - Recruiting
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