NCT00030056

Brief Summary

This is a study to determine the efficacy and safety of granulocyte-macrophage colony-stimulating factor (GM-CSF, sargramostim) administered subcutaneously to patients with pulmonary alveolar proteinosis (PAP).

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2001

Typical duration for phase_2

Geographic Reach
1 country

3 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2001

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

January 30, 2002

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 31, 2002

Completed
3.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2005

Completed
Last Updated

March 25, 2015

Status Verified

January 1, 2002

First QC Date

January 30, 2002

Last Update Submit

March 24, 2015

Conditions

Pulmonary Alveolar Proteinosis

Keywords

SargramostimInjections, Subcutaneous

Interventions

GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)DRUG

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of primary or idiopathic PAP
  • Creatinine no greater than 2 mg/dL
  • Bilirubin no greater than 2 mg/dL
  • Liver enzymes no greater than 3 times normal
  • Women must use an effective method of contraception, be post menopausal, or be surgically sterilized

You may not qualify if:

  • Active respiratory infection
  • Active cardiovascular disease (e.g., cardiogenic pulmonary edema)
  • Underlying myeloproliferative disorder or leukemia
  • Other secondary cause of PAP (e.g., occupational exposure to silica or HIV with PCP)
  • At increased risk of side effects from GM-CSF therapy (i.e., rheumatoid arthritis, immune thrombocytopenia, or autoimmune thyroiditis)
  • Previous therapy with GM-CSF
  • Pregnant or nursing
  • Significant renal or liver disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

National Jewish Medical Center

Denver, Colorado, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

The University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, United States

Location

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis

Interventions

Granulocyte-Macrophage Colony-Stimulating Factorsargramostim

Condition Hierarchy (Ancestors)

Lung DiseasesRespiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

January 30, 2002

First Posted

January 31, 2002

Study Start

September 1, 2001

Study Completion

December 1, 2005

Last Updated

March 25, 2015

Record last verified: 2002-01

Locations

US(3)