Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2008-007086-23-IT
• Date of registration: 27/01/2009
• Prospective Registration: No
• Primary sponsor: OSPEDALE POLICLINICO S. MATTEO
• Public title: Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP
• Scientific title: Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP
• Date of first enrolment: 12/12/2008
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007086-23
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Farmaco associato a WLL versus solo WLL

Countries of recruitment

Italy

Key inclusion & exclusion criteria

Inclusion criteria:
autoimmune PAP diagnosis; age >= 18 years; persistant or progressive respiratory failure (PaO2 < 60 mmHg) or latent respiratory failure (SatO2 < 90% or desaturation > 5% in Bruce`s test) or persistant pulmonary TC abnormalities (intensity score >=3) (reasidual disease treated group)
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
non autoimmune PAP; contraindications to WLL; contraindication to GM-CSF; pregnancy; refusal to the use of contraceptive methods during the study period; contraindications to the use of inhaled medicines.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Autoimmune PAP
MedDRA version: 9.1 Level: LLT Classification code 10037316 Term: Pulmonary alveolar proteinosis

Intervention(s)

Trade Name: Leukine
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Sargramostim
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: .5-

Primary Outcome(s)

Main Objective: Total resolution of the disease in 100% of patients treated with WLL/inhaled GM-CSF.

Secondary Objective: 1)to avoid recurrence of the disease requiring multiple WWL, 2) to avoid or reduce the number of respiratory infections due to persistant pulmonary abnormalities PAP associated 3) evaluation of outcome determinants in the diverse treated groups, 4) ancillary study to establish whether the addition of inhaled GM-CSF may influence PAP pathophysiology.

Primary end point(s): Primary efficacy endpoint (total resolution of disease in 100% of patients trated with WLL/inhaled GM-CSF) will be documented as follows: a. functional endpoints: measuere of alveolar-arterial oxygen tension gradient (PA-a O2), measure of oxygen saturation during exercise, according to the modified Bruce`s protocol. b. Imaging endpoint: score of the pulmonary CT performed after 3 and 10 months after WLL. c. Severity score: score based on symptoms and PaO2 level at entry. The score will be associated to a quality of life questionnaire (SF36).

Secondary ID(s)

FARM7MCPK4