NCT03887169View on ClinicalTrials.gov

Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

September 16, 2019

Primary Completion Date

June 1, 2020

Study Completion Date

June 1, 2020

Conditions
Pulmonary Alveolar ProteinosisMutation Ala393Thr of the MARS GenemutationSer567Leu of the MARS Gene
Interventions
DRUG

Methionine

Administration of methionine from D1 to D60

DRUG

Vitamin B12, B9, B6, C supplementation

In case of hyperhomocysteinemia

DIAGNOSTIC_TEST

Methionine/homocysteine Dosage

Plasma concentration control of methionine and homocysteine from D0 to D75

DIAGNOSTIC_TEST

Thoracic CT scan

At D60

DIAGNOSTIC_TEST

Abdominal and liver ultrasound.

At D60

DIAGNOSTIC_TEST

Brain MRI

In case of abnormal neurological examination

Trial Locations (1)

75015

Hôpital Necker-Enfants Malades, Paris

All Listed Sponsors
lead

Assistance Publique - Hôpitaux de Paris

OTHER

NCT03887169 - Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene. | Biotech Hunter | Biotech Hunter