Trial · NCT02697734
Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease
NCT02697734, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2021-10-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease. Trials, record NCT02697734. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02697734
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease
- NCT number
- NCT02697734
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 73 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Cushing's Disease
- Interventions
- osilodrostat, osilodrostat Placebo
- First posted
- 2016-03-03
- Last updated
- 2021-11-01
- Results first posted
- 2021-10-19
Source ClinicalTrials.gov
Registered outcomes36
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Randomized Participants With a Complete Response | at Week 12 | A complete responder at week 12 is defined as a participant who had a mean urine free cortisol ≤ upper limit of normal (mUFC ≤ ULN) at Week 12. Participants who had a missing mUFC assessment at Week 12 were counted as non-responders for the primary endpoint. |
| Secondary | Percentage of Participants With mUFC ≤ ULN at Week 36 | At Week 36 | The complete response rate in both arms combined at Week 36. A complete responder at Week 36 is defined as a participant who had mean urine free cortisol \<= upper limit of normal (mUFC \<= ULN) at Week 36. Participants with missing mUFC at Week 36 were counted as non-responders. |
| Secondary | Change From Baseline in mUFC | Baseline, weeks 2,5,8,12,14,17,20,23,26,29,32,36,40,48,60,72,84,96 | To assess the change in mean urinary free cortisol (mUFC) from baseline by treatment arm. |
| Secondary | Time-to-first Control of mUFC - Number (%) of Participants With mUFC <=ULN | up to 12 weeks | To assess time-to-first control of mUFC, (in days) from randomization to the first mUFC collection that was ≤ ULN before completion/discontinuation of placebo-controlled period. Participants who did not achieve post-baseline mUFC control were censored at discontinuation or completion of placebo-controlled period, whichever was earlier. |
| Secondary | Time-to-first Control of mUFC - Median Time to First Controlled mUFC Response | up to 12 weeks | To assess time-to-first control of mUFC, (in days) from randomization to the first mUFC collection that was ≤ ULN before completion/discontinuation of placebo-controlled period. Participants who did not achieve post-baseline mUFC control were censored at discontinuation or completion of placebo-controlled period, whichever was earlier. The median time-to-first control and corresponding two-sided 95% Confidence Interval were calculated using Kaplan-Meier methodology of Brookmeyer and Crowley (1982). |
| Secondary | Time-to-first Control of mUFC - % Event Probability Estimates | up to 12 weeks | To assess time-to-first control of mUFC, (in days) from randomization to the first mUFC collection that was ≤ ULN before completion/discontinuation of placebo-controlled period. Participants who did not achieve post-baseline mUFC control were censored at discontinuation or completion of placebo-controlled period, whichever was earlier. % Event probability estimate is the estimated probability that a participant will have an event prior to the specified time point. % Event probability estimates are obtained from the Kaplan-Meier survival estimates for all treatment groups; Greenwood formula is used for Confidence Interval (CI) of Kaplan-Meier (KM) estimates. |
| Secondary | Time-to-escape During Osilodrostat Treatment From Collection of Normal mUFC (≤ ULN) to the First mUFC > 1.3 x ULN - Number (%) of Participants | up to 48 weeks | To assess time-to-escape from the first collection of normal mUFC (≤ ULN) to the first mUFC \> 1.3 x ULN on two consecutive visits on the highest tolerated dose of osilodrostat and not related to a dose interruption or dose reduction due to safety reasons. Escape will not be assessed for participants during the first 26 weeks. |
| Secondary | Time-to-escape During Osilodrostat Treatment From Collection of Normal mUFC (≤ ULN) to the First mUFC > 1.3 x ULN - Median Time to Escape From Normal mUFC | from week 26 to week 48 | To assess time-to-escape from the first collection of normal mUFC (≤ ULN) to the first mUFC \> 1.3 x ULN on two consecutive visits on the highest tolerated dose of osilodrostat and not related to a dose interruption or dose reduction due to safety reasons. Escape will not be assessed for participants during the first 26 weeks. The median time-to-escape and corresponding two-sided 95% Confidence Interval were calculated using Kaplan-Meier methodology of Brookmeyer and Crowley (1982). |
| Secondary | Time-to-escape During Osilodrostat Treatment From Collection of Normal mUFC (≤ ULN) to the First mUFC > 1.3 x ULN - % Event Probability Estimates | week 26 and week 36 | Escape is defined as the first loss of control of urinary free cortisol (UFC) that meets all of the following criteria: 1. prior normalization of UFC has occurred (median urinary free cortisol (mUFC)≤ upper limit of normal (ULN)); 2. patient reached the highest tolerated dose of osilodrostat; 3. 2 consecutive mUFC (collected at scheduled visits) were above 1.3x ULN; 4. the loss of control of UFC is not related to a dose interruption or dose reduction due to safety reasons; 5. happened beyond Week 26 when the patients have a chance to be treated with doses as high as 30 mg bid. * Event probability estimate is the estimated probability that a participant will have an event prior to the specified time point. * Event probability estimates are obtained from the Kaplan-Meier survival estimates for all treatment groups; Greenwood formula is used for CI of KM estimates. |
| Secondary | Change From Baseline in Bone Mineral Density (BMD) by Dual-energy X-ray Absorptiometry (DXA) Scan at the Femoral Neck, Hip and Spinal Cord - QC Corrected | Baseline, week 48 | The change from baseline in bone mineral density at the femoral neck, hip and spinal cord at Week 48 by treatment arm - QC corrected. An increase in bone mineral density is indicative of an improvement. |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 registered outcomesOutcomes36
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Randomized Participants With a Complete Response | at Week 12 | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With mUFC ≤ ULN at Week 36 | At Week 36 | Full Analysis Set participants: comprises all randomized participants who received at least one dose of osilodrostat. Only a single arm is reported since the endpoint is 'To assess the complete response rate in both arms combined at Week 36 in patients receiving osilodrostat treatment.' | Percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in mUFC | Baseline, weeks 2,5,8,12,14,17,20,23,26,29,32,36,40,48,60,72,84,96 | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo). The number analyzed varied from one visit to another because of missed visits, missed assessments, early discontinuation from the study, and completion of the extension phase. | nmol/24hr | MEAN |
| SECONDARY | Time-to-first Control of mUFC - Number (%) of Participants With mUFC <=ULN | up to 12 weeks | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time-to-first Control of mUFC - Median Time to First Controlled mUFC Response | up to 12 weeks | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo). | Days | MEDIAN |
| SECONDARY | Time-to-first Control of mUFC - % Event Probability Estimates | up to 12 weeks | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo). | Percent (event probability estimates) | NUMBER |
| SECONDARY | Time-to-escape During Osilodrostat Treatment From Collection of Normal mUFC (≤ ULN) to the First mUFC > 1.3 x ULN - Number (%) of Participants | up to 48 weeks | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time-to-escape During Osilodrostat Treatment From Collection of Normal mUFC (≤ ULN) to the First mUFC > 1.3 x ULN - Median Time to Escape From Normal mUFC | from week 26 to week 48 | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo). | days | MEDIAN |
| SECONDARY | Time-to-escape During Osilodrostat Treatment From Collection of Normal mUFC (≤ ULN) to the First mUFC > 1.3 x ULN - % Event Probability Estimates | week 26 and week 36 | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo). | Percent (event probability estimates) | NUMBER |
| SECONDARY | Change From Baseline in Bone Mineral Density (BMD) by Dual-energy X-ray Absorptiometry (DXA) Scan at the Femoral Neck, Hip and Spinal Cord - QC Corrected | Baseline, week 48 | Full Analysis Set: comprises all randomized participants who received at least one dose of study drug (osilodrostat or placebo). The number analyzed varied from one visit to another because of missed visits, missed assessments, early discontinuation from the study, and completion of the extension phase. | g/cm2 | MEAN |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 outcomesResults by group385
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Randomized Participants With a Complete Response | Osilodrostat Group | 37 | Participants | ||
| Percentage of Randomized Participants With a Complete Response | Osilodrostat Placebo Group | 2 | Participants | ||
| Percentage of Participants With mUFC ≤ ULN at Week 36 | All Participants Combined | 80.8 | 95% Confidence Interval 69.9 to 89.1 | Percentage of participants | |
| Change From Baseline in mUFC | Osilodrostat Group | actual - baseline | 421.4 | Standard Deviation 291.25 | nmol/24hr |
| Change From Baseline in mUFC | Osilodrostat Placebo Group | actual - baseline | 451.5 | Standard Deviation 535.09 | nmol/24hr |
| Change From Baseline in mUFC | Osilodrostat Group | change from baseline at week 2 (n=47,24) | -139.3 | Standard Deviation 404.45 | nmol/24hr |
| Change From Baseline in mUFC | Osilodrostat Placebo Group | change from baseline at week 2 (n=47,24) | 164.9 | Standard Deviation 543.82 | nmol/24hr |
| Change From Baseline in mUFC | Osilodrostat Group | change from baseline at week 5 (n=46,25) | -252.8 | Standard Deviation 338.48 | nmol/24hr |
| Change From Baseline in mUFC | Osilodrostat Placebo Group | change from baseline at week 5 (n=46,25) | -37.3 | Standard Deviation 280.84 | nmol/24hr |
| Change From Baseline in mUFC | Osilodrostat Group | change from baseline at week 8 (n=44,25) | -330.2 | Standard Deviation 303.35 | nmol/24hr |
1 to 10 of 385Source ClinicalTrials.gov
See all 385 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Phase - up to Week 48 | STARTED | Osilodrostat Group | 48 | |
| Core Phase - up to Week 48 | STARTED | Osilodrostat Placebo Group | 25 | |
| Core Phase - up to Week 48 | COMPLETED | Osilodrostat Group | 42 | |
| Core Phase - up to Week 48 | COMPLETED | Osilodrostat Placebo Group | 23 | |
| Core Phase - up to Week 48 | NOT COMPLETED | Osilodrostat Group | 6 | |
| Core Phase - up to Week 48 | NOT COMPLETED | Osilodrostat Placebo Group | 2 | |
| Optional Extension Phase | STARTED | Osilodrostat Group | 38 | |
| Optional Extension Phase | STARTED | Osilodrostat Placebo Group | 22 | |
| Optional Extension Phase | COMPLETED | Osilodrostat Group | 33 | |
| Optional Extension Phase | COMPLETED | Osilodrostat Placebo Group | 20 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events425
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 | Anaemia | Blood and lymphatic system disorders | Placebo Controlled Period - Osilodrostat Arm. | 2 | 48 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Controlled Period - Placebo Arm. | 2 | 25 | |
| other | Anaemia | Blood and lymphatic system disorders | Overall Study Period - Osilodrostat Arm. | 2 | 48 | |
| other | Anaemia | Blood and lymphatic system disorders | Overall Study Period - Placebo Arm. | 1 | 25 | |
| other | Anaemia | Blood and lymphatic system disorders | Overall Study Period - All Participants. | 3 | 73 | |
| other | Palpitations | Cardiac disorders | Placebo Controlled Period - Osilodrostat Arm. | 0 | 48 | |
| other | Palpitations | Cardiac disorders | Placebo Controlled Period - Placebo Arm. | 2 | 25 | |
| other | Palpitations | Cardiac disorders | Overall Study Period - Osilodrostat Arm. | 2 | 48 | |
| other | Palpitations | Cardiac disorders | Overall Study Period - Placebo Arm. | 0 | 25 | |
| other | Palpitations | Cardiac disorders | Overall Study Period - All Participants. | 2 | 73 |
1 to 10 of 425Source ClinicalTrials.gov
See all 425 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo Controlled Period - Osilodrostat Arm. | 0 | 48 |
| Total, all-cause mortality | Placebo Controlled Period - Placebo Arm. | 0 | 25 |
| Total, all-cause mortality | Overall Study Period - Osilodrostat Arm. | 0 | 48 |
| Total, all-cause mortality | Overall Study Period - Placebo Arm. | 0 | 25 |
| Total, all-cause mortality | Overall Study Period - All Participants. | 0 | 73 |
| Total, other adverse events | Placebo Controlled Period - Osilodrostat Arm. | 45 | 48 |
| Total, other adverse events | Placebo Controlled Period - Placebo Arm. | 21 | 25 |
| Total, other adverse events | Overall Study Period - Osilodrostat Arm. | 47 | 48 |
| Total, other adverse events | Overall Study Period - Placebo Arm. | 24 | 25 |
| Total, other adverse events | Overall Study Period - All Participants. | 71 | 73 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers3
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Osilodrostat Group vs Osilodrostat Placebo Group: Time-to-first Control of mUFC - Number (%) of Participants With mUFC <=ULN | Trial comparisons |
| Osilodrostat Group vs Osilodrostat Placebo Group: Percentage of Randomized Participants With a Complete Response | 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/NCT02697734
GET https://conductscience.org/api/trials/v1/NCT02697734/registered?page=2