Phase
Condition
Phenylketonuria
Distal Renal Tubular Acidosis
Treatment
Placebo
mRNA-3705
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
Stollery Children's Hospital University of Alberta
Edmonton, Alberta T6G 2R7
CanadaSite Not Available
Hospital For Sick Children
Toronto, Ontario M5G 1X8
CanadaSite Not Available
Hôpital Necker - Enfants Malades
Paris, 75015
FranceSite Not Available
Erasmus MC
Rotterdam, 3015 AA
NetherlandsSite Not Available
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
NetherlandsSite Not Available
Hospital Universitario Cruces
Barakaldo, 48903
SpainSite Not Available
Hospital Universitario 12 de Octubre
Madrid, 28041
SpainSite Not Available
Birmingham Children's Hospital NHS Foundation Trust
Birmingham,
United KingdomSite Not Available
Royal Manchester Children's Hospital
Manchester, M13 9WL
United KingdomSite Not Available
Royal Manchester Childrens Hospital
Manchester, 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
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available

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