A Phase I/II Study of Bucladesine in Patients with Severe Methylmalonic Acidemia

A Phase I/II Multi-Institutional Study of Bucladesine in Patients with Severe Methylmalonic Acidemia - McAMP

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2041210065

Enrollment

Registered

2021-09-04

Start date

2022-03-10

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

methylmalonic acidemia

Interventions

Bucladesine will be administered intravenously over 60 minutes once a week. Starting dose is 0.3mg/kg. PK and safety data will be evaluated. Then, dosage will be incerased to 0.55mg/kg and administere

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (1) Diagnosis of methylmalonic acidemia (MMA) defined by biochemical diagnostic criteria on the MMA guideline 2019 (2) Developmental quotient (DQ) 15mEq/L) metabolic acidosis, and pH80ug/dL iii) Hyperlactacidemia: blood lactic acid level>22mg/dL iv) Hypoglycemia: random blood glucose level<70mg/dL (3) 3 months old or older at a time of starting the investigational drug (4) Written informed consent obtained by a patient (or a parent/legal guardian of the patient if a patient is under the age of 20)

Exclusion criteria

Exclusion criteria: (1) Vitamin B12 responsive MMA (2) History of liver or kidney transplant (3) AST or ALT elevated more than 5 times the upper limit of normal (4) Any serious diseases not related to MMA (5) Gestatoinal age <37 weeks at birth: This does not apply if over 12 months old at the time consent is obtained. (6) History of allergy to bucladesine (7) History of acute myocardial infarction or cardiomyopathy (8) History of arrhythmia (9) Having diabetes (10) Having any renal cyst, OR, Family history of polycystic kidney disease (11) History of illicit drug or alcohol abuse (12) Use of any investigational drug or device in other clinical studies within 30 days before obtaining an informed consent (13) Pregnant or breast feeding women. Not following contraception requirement during the clinical study. (14) Not eligible for this clinical study decided by an investigator or sub-investigator

Design outcomes

Primary

Measure	Time frame
Suspected adverse reaction rate	—

Secondary

Measure	Time frame
Adverse event rate, Serious suspected adverse reaction rate, PK, Clinical response	—

Contacts

Public ContactTsutomu Yamaguchi

Kyushu University Hospital

yamaguchi.tsutomu.989@m.kyushu-u.ac.jp+81-92-642-5110

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026