Pulmonary Alveolar Proteinosis Clinical Trial
— MetPAPOfficial title:
Oral or Enteral Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.
Verified date | November 2020 |
Source | Assistance Publique - Hôpitaux de Paris |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to determine the safety and tolerance of an oral administration of methionine in the treatment of pulmonary alveolar proteinosis due to the double mutation Ala393Thr / Ser567Leu in the MARS gene. This disease is very severe and especially leads to chronic respiratory insufficiency. There is no curative treatment for this disease. The MARS gene encodes the methionine tRNA synthetase (MetRS). Mutations in this gene leads to a defect in MetRS function. In cultured mutated yeast, addition of methionine in culture medium restores MetRS function. Therefore, the investigators hypothesized that treatment of patients with methionine could have beneficial effects on the disease.
Status | Completed |
Enrollment | 3 |
Est. completion date | June 1, 2020 |
Est. primary completion date | June 1, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 18 Years |
Eligibility | Inclusion Criteria: - Minor Patient with alveolar proteinosis by double mutation Ala393Thr and SER567LEU of the MARS gene, genetically proven. - Patient in need of prolonged hospitalization in Necker for treatment of bronchial-alveolar washes in the context of care. - Patient for which methionine can be administered orally or by enteral probe (Nasogastric or gastrostomy probe) - Signed Informed consent form by parents / legal guardian Exclusion Criteria: - Patient with alveolar proteinosis by other mutations of the MARS gene - Patient with alveolar proteinosis secondary to another etiology or without identified cause - Refusal to participate in the study - High blood pressure requiring drug treatment - Heart failure - Known hypersensitivity to one of the substances used or potentially used in the study: methionine, vitamins B6, B12, B9 and C - Pre-Hypermethioninemia (Methioninemia > + 2 DS of normal for age) whatever the cause |
Country | Name | City | State |
---|---|---|---|
France | Hôpital Necker-Enfants Malades | Paris | Ile De France |
Lead Sponsor | Collaborator |
---|---|
Assistance Publique - Hôpitaux de Paris |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Tolerance Assessment | No adverse event from day 0 to day 75. | From day 0 to day 75 | |
Secondary | Respiratory rate (cycles /min) | number of cycles per minute | At day 0, day 15, day 30, day 45, day 60, day 75 | |
Secondary | Oxygen need (L/min) | Flow in L/min | At day 0, day 15, day 30, day 45, day 60, day 75 | |
Secondary | Respiratory signs of struggle | Presence or absence of signs | At day 0, day 15, day 30, day 45, day 60, day 75 | |
Secondary | Lung lesions | Lesions appearance on thoracic CT scan, scored form 0 to 4 | At Day 60 | |
Secondary | Lipo-proteinaceous material | Fluid examination | At each bronchial-alveolar washes during the 2,5 months | |
Secondary | Weight | To evaluate Nutritional status | At Day 15, Day 30, Day 45, Day 60, Day 75 | |
Secondary | mid upper arm circumference / head circumference rapport | To evaluate Nutritional status | At Day 15, Day 30, Day 45, Day 60, Day 75 | |
Secondary | Hepatomegaly | liver damage evaluate by physician during clinical examination | At Day 0, Day 15, Day 30, Day 45, Day 60, Day 75 | |
Secondary | cholestasis and hepatic cytolysis | liver damage evaluate by biological parameters : ASAT, ALAT, GGT, PAL, Bilirubin | At Day 0, Day 15, Day 30, Day 60, Day 75 | |
Secondary | Hepatomegaly | liver damage evaluate by echography | At Day 0 and Day 60 | |
Secondary | C reactive protein | Biological parameters to evaluate Systemic inflammation | At Day 0, Day 30, Day 60 | |
Secondary | sedimentation rate | Biological parameters to evaluate Systemic inflammation | At Day 0, Day 30, Day 60 | |
Secondary | Immunoglobulin G level | Biological parameters to evaluate Systemic inflammation | At Day 0, Day 30, Day 60 | |
Secondary | Haemoglobin level | Biological parameters to evaluate inflammatory anaemia | At Day 0, Day 30, Day 60 | |
Secondary | Plasma concentration of methionine | Variation of the concentration for each patient | From Day 0 to Day 75 | |
Secondary | Plasma concentration of homocysteine | Variation of the concentration for each patient | From Day 0 to Day 75 |
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