Phase
Condition
Phenylketonuria
Distal Renal Tubular Acidosis
Treatment
mRNA-3705
Clinical Study ID
Ages > 1 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Participant has a body weight of ≥11.0 kilograms (kg) at the Screening Visit.
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. Forthose participants found to have an elevated blood vitamin B12 level, theparticipant may enter if, in the opinion of the Investigator, the cause of theelevation is secondary to B12 supplementation.
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 females of childbearing potential and sexually active males ofreproductive potential agree to use a highly-effective method of contraceptionduring the study and for 3 months after the last administration of study drug.
(Part 2 only) At least 1 documented MDE in the 12-month period before consent.
Exclusion
Key Exclusion Criteria:
Participant has a diagnosis of isolated MMA cb1A, cb1B, or cb1D enzymatic subtypesor methylmalonyl-CoA epimerase deficiency or combined MMA 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) History of hepatitis B (known positive hepatitis B surface antigen [HbsAg]), hepatitis C virus (HCV), or HIV (positive HIV1/HIV-2 antibodies).Participants with a past or resolved hepatitis virus B (HBV) infection (defined asthe presence of hepatitis B core antibody and absence of HbsAg) are eligible.Participants with history of positive results for HCV antibody are eligible only ifpolymerase chain reaction is negative for HCV RNA.
Study Design
Study Description
Connect with a study center
Children's Hospital at Westmead
Westmead, New South Wales 2145
AustraliaSite Not Available
Royal Children's Hospital Melbourne
Parkville, Victoria 3052
AustraliaSite Not Available
Stollery Children's Hospital University of Alberta
Edmonton, Alberta T6G 2R7
CanadaActive - Recruiting
Hospital For Sick Children
Toronto, Ontario M5G 1X8
CanadaActive - Recruiting
Hôpital Necker - Enfants Malades
Paris, 75015
FranceActive - Recruiting
Erasmus MC
Rotterdam, 3015 AA
NetherlandsActive - Recruiting
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX
NetherlandsActive - Recruiting
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, Barcelona 08950
SpainActive - Recruiting
Hospital Universitario Cruces
Barakaldo, 48903
SpainActive - Recruiting
Hospital Universitario 12 de Octubre
Madrid, 28041
SpainActive - Recruiting
CHUS - H. Clinico U. de Santiago
Santiago de Compostela, 15706
SpainActive - Recruiting
Hospital Universitario Virgen del Rocio - PPDS
Sevilla, 41013
SpainActive - Recruiting
Birmingham Children's Hospital NHS Foundation Trust
Birmingham,
United KingdomTerminated
Royal Manchester Childrens Hospital
Manchester, M13 9WL
United KingdomActive - Recruiting
UCLA Medical Center
Los Angeles, California 90095
United StatesActive - Recruiting
Lucile Packard Children's Hospital at Stanford
Palo Alto, California 94304
United StatesActive - Recruiting
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
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