Trial · NCT00425607
Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
NCT00425607, Phase 2, Completed, Monica E. Kleinman; results first posted 2017-05-24
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria. Trials, record NCT00425607. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00425607
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
- NCT number
- NCT00425607
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Monica E. Kleinman
- Conditions
- Progeria, Hutchinson-Gilford Syndrome
- Interventions
- Lonafarnib
- First posted
- 2007-01-23
- Last updated
- 2019-06-25
- Results first posted
- 2017-05-24
- Limitations and caveats
This was an open label clinical trial on a very rare disease population. Patient numbers are small, though 26 participants represents a significant portion of the world's population of children with HGPS
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Participants With Successful Rate of Weight Gain | Assessed at weeks 16, 32, 52, 68, 84 and 104 | Activity was assessed by determining the change in rate of weight gain over two years from baseline (determined pre-therapy for each patient). Primary outcome success was predefined as a 50% increase over pre-therapy in estimated annual rate of weight gain, or change from pre-therapy weight loss to statistically significant on-study weight gain. |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion of Participants With Successful Rate of Weight Gain | Assessed at weeks 16, 32, 52, 68, 84 and 104 | Results are reported for the 25 patients with classic HGPS who completed at least 2y of therapy. One additional patient with a prior history of strokes died of a stroke after 5 mo on study and is not included in the analysis. Two additional patients had nonclassic mutations and are not included in this analysis. | proportion of participants | NUMBER |
Source ClinicalTrials.gov
Results by group1
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion of Participants With Successful Rate of Weight Gain | Lonafarnib | 0.36 | 95% Confidence Interval 0.18 to 0.58 | proportion of participants |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Lonafarnib | 29 | |
| Overall Study | Received Treatment | Lonafarnib | 28 | |
| Overall Study | COMPLETED | Lonafarnib | 27 | |
| Overall Study | NOT COMPLETED | Lonafarnib | 2 |
Source ClinicalTrials.gov
Adverse events7
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 |
|---|---|---|---|---|---|---|
| serious | Vomiting | Gastrointestinal disorders | Lonafarnib | 2 | 28 | 2 |
| serious | Stroke | Nervous system disorders | Lonafarnib | 3 | 28 | 3 |
| serious | Bacterial infection | Infections and infestations | Lonafarnib | 1 | 28 | 1 |
| serious | Subdural hematoma | Nervous system disorders | Lonafarnib | 1 | 28 | 1 |
| serious | Extremity-lower (gait/walking) | Musculoskeletal and connective tissue disorders | Lonafarnib | 1 | 28 | 1 |
| serious | Neuropathy: Sensory | Nervous system disorders | Lonafarnib | 1 | 28 | 1 |
| serious | Hypokalemia | Blood and lymphatic system disorders | Lonafarnib | 1 | 28 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lonafarnib | ||
| Total, other adverse events | Lonafarnib | 0 | 28 |
| Total, serious adverse events | Lonafarnib | 10 | 28 |
Source ClinicalTrials.gov
Papers8
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00425607
GET https://conductscience.org/api/trials/v1/NCT00425607/registered