Trial · NCT00001596
Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
NCT00001596, Phase 2, Completed, William Gahl, M.D.; results first posted 2012-01-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome. Trials, record NCT00001596. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00001596
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
- NCT number
- NCT00001596
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 35 participants, actual
- Lead sponsor
- William Gahl, M.D.
- Conditions
- Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
- Interventions
- Pirfenidone, Placebo
- First posted
- 1999-11-04
- Last updated
- 2017-10-16
- Results first posted
- 2012-01-26
- Limitations and caveats
Study enrollment progressed more slowly than anticipated (spanning \>3 years instead of one). The Data Safety and Monitoring Board performed an interim analysis one year after 30 subjects enrolled and directed the study to stop due to futility.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Forced Vital Capacity (36 Months) | Measured at baseline and 36 months | Change from baseline in the Forced Vital Capacity (FVC) measurement at 36 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight). |
| Secondary | Change in Forced Vital Capacity (12 Months) | Measured at baseline and 12 months | Change from baseline in the Forced Vital Capacity (FVC) measurement at 12 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight). |
| Secondary | Change in Total Lung Capacity (36 Months) | Measured at baseline and 36 months | Change from baseline in Total Lung Capacity (TLC) measured at 36 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight. |
| Secondary | Change in Total Lung Capacity (12 Months) | Measured at baseline and 12 months | Change from baseline in Total Lung Capacity (TLC) measured at 12 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight. |
| Secondary | Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months) | Measured at baseline and 36 months | Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 36 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels. |
| Secondary | Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (12 Months) | Measured at baseline and 12 months | Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 12 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels. |
| Secondary | Change in 6 Minute Walk Test (36 Months) | Measured at baseline and 36 months | Change from baseline of the 6 minute walk test (6MWT) at 36 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes. |
| Secondary | Change in 6 Minute Walk Test (12 Months) | Measured at baseline and 12 months | Change from baseline of the 6 minute walk test (6MWT) at 12 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Forced Vital Capacity (36 Months) | Measured at baseline and 36 months | Participants from the Intent to Treat population for whom data was available at baseline and 36 months. | % of predicted volume | MEAN |
| SECONDARY | Change in Forced Vital Capacity (12 Months) | Measured at baseline and 12 months | Participants from the Intent to Treat population for whom data was available at baseline and 12 months. | % of predicted volume | MEAN |
| SECONDARY | Change in Total Lung Capacity (36 Months) | Measured at baseline and 36 months | Participants from the Intent to Treat population for whom data is available at baseline and 36 months. | % of predicted volume | MEAN |
| SECONDARY | Change in Total Lung Capacity (12 Months) | Measured at baseline and 12 months | Participants from the Intent to Treat population for whom data was available at baseline and 12 months. | % of predicted volume | MEAN |
| SECONDARY | Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months) | Measured at baseline and 36 months | Participants from the Intent to Treat population for whom data was available at baseline and 36 months. | % of predicted volume | MEAN |
| SECONDARY | Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (12 Months) | Measured at baseline and 12 months | Participants from the Intent to Treat population for whom data is available at baseline and 12 months. | % of predicted volume | MEAN |
| SECONDARY | Change in 6 Minute Walk Test (36 Months) | Measured at baseline and 36 months | Participants from the Intent to Treat population for whom data is available at baseline and 36 months. | meters | MEAN |
| SECONDARY | Change in 6 Minute Walk Test (12 Months) | Measured at baseline and 12 months | Participants from the Intent to Treat population for whom data is available at baseline and 12 months. | meters | MEAN |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Forced Vital Capacity (36 Months) | Pirfenidone | -23.52 | Standard Deviation 24.32 | % of predicted volume | |
| Change in Forced Vital Capacity (36 Months) | Placebo | -20.93 | Standard Deviation 25.59 | % of predicted volume | |
| Change in Forced Vital Capacity (12 Months) | Pirfenidone | -7.27 | Standard Deviation 20.48 | % of predicted volume | |
| Change in Forced Vital Capacity (12 Months) | Placebo | -3.55 | Standard Deviation 21.33 | % of predicted volume | |
| Change in Total Lung Capacity (36 Months) | Pirfenidone | -25.26 | Standard Deviation 24.42 | % of predicted volume | |
| Change in Total Lung Capacity (36 Months) | Placebo | -22.9 | Standard Deviation 23.69 | % of predicted volume | |
| Change in Total Lung Capacity (12 Months) | Pirfenidone | -8.96 | Standard Deviation 21.03 | % of predicted volume | |
| Change in Total Lung Capacity (12 Months) | Placebo | 0.53 | Standard Deviation 21.27 | % of predicted volume | |
| Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months) | Pirfenidone | -15.25 | Standard Deviation 29.89 | % of predicted volume | |
| Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months) | Placebo | -14.93 | Standard Deviation 29.74 | % of predicted volume |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pirfenidone | 23 | |
| Overall Study | STARTED | Placebo | 12 | |
| Overall Study | COMPLETED | Pirfenidone | 6 | |
| Overall Study | COMPLETED | Placebo | 2 | |
| Overall Study | NOT COMPLETED | Pirfenidone | 17 | |
| Overall Study | NOT COMPLETED | Placebo | 10 |
Source ClinicalTrials.gov
Adverse events44
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | Pirfenidone | 0 | 23 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 2 | 12 | 2 |
| other | Arthalgia | Musculoskeletal and connective tissue disorders | Pirfenidone | 1 | 23 | 1 |
| other | Arthalgia | Musculoskeletal and connective tissue disorders | Placebo | 0 | 12 | 0 |
| other | Chest pain | Cardiac disorders | Pirfenidone | 3 | 23 | 3 |
| other | Chest pain | Cardiac disorders | Placebo | 1 | 12 | 1 |
| other | Dizziness | Nervous system disorders | Pirfenidone | 4 | 23 | 4 |
| other | Dizziness | Nervous system disorders | Placebo | 0 | 12 | 0 |
| other | Drug eruption | Immune system disorders | Pirfenidone | 2 | 23 | 2 |
| other | Drug eruption | Immune system disorders | Placebo | 0 | 12 | 0 |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pirfenidone | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Pirfenidone | 18 | 23 |
| Total, other adverse events | Placebo | 9 | 12 |
| Total, serious adverse events | Pirfenidone | 6 | 23 |
| Total, serious adverse events | Placebo | 2 | 12 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Pirfenidone vs Placebo: serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00001596
GET https://conductscience.org/api/trials/v1/NCT00001596/events?page=2