Trial · NCT01603407
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
NCT01603407, Phase 3, Completed, University of Rochester; results first posted 2022-08-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Finding the Optimum Regimen for Duchenne Muscular Dystrophy. Trials, record NCT01603407. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01603407
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Finding the Optimum Regimen for Duchenne Muscular Dystrophy
- NCT number
- NCT01603407
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 196 participants, actual
- Lead sponsor
- University of Rochester
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Prednisone, Prednisone, Deflazacort
- First posted
- 2012-05-23
- Last updated
- 2022-08-12
- Other registries' IDs
- eudract:2010-023744-33, isrctn:ISRCTN46102316
- Results first posted
- 2022-08-12
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Forced Vital Capacity | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test. |
| Primary | Rise From the Floor Velocity | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Reciprocal of time to rise from the floor |
| Primary | Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes. |
| Secondary | North Star Ambulatory Assessment (NSAA) Score | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects. The activities are graded as follows: 2 - "Normal" - no obvious modification of activity 1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function. |
| Secondary | 6 Minute Walk Test | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. |
| Secondary | Range of Motion (Goniometry) of Left Ankle | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. |
| Secondary | Range of Motion (Goniometry) of Right Ankle | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. |
| Secondary | Number of Participants Who Tolerated the Regimen | 3 years | The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication. |
| Secondary | Heart Rate | 36 months | Measured by trans-thoracic echocardiogram and 12-lead ECG. |
| Secondary | Quality of Life - Parent | Average of Months 12, 24, and 36 visits | Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Forced Vital Capacity | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 4 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm. | liters | LEAST_SQUARES_MEAN |
| PRIMARY | Rise From the Floor Velocity | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 3 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm. | rise/sec | LEAST_SQUARES_MEAN |
| PRIMARY | Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | North Star Ambulatory Assessment (NSAA) Score | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 3 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | 6 Minute Walk Test | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 5 participants in the daily prednisone arm, 2 participants in the deflazacort arm and 2 participant in the intermittent prednisone arm. | meters | LEAST_SQUARES_MEAN |
| SECONDARY | Range of Motion (Goniometry) of Left Ankle | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm. | degrees | MEAN |
| SECONDARY | Range of Motion (Goniometry) of Right Ankle | Average of Months 3, 6, 12, 18, 24, 30 and 36 visits | Data was not collected on 3 participants in the daily prednisone arm, 3 participants in the deflazacort arm and 1 participant in the intermittent prednisone arm. | degrees | MEAN |
| SECONDARY | Number of Participants Who Tolerated the Regimen | 3 years | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Heart Rate | 36 months | Data was not collected on 1 participant in the daily prednisone arm, and 2 participants in the intermittent prednisone arm. | bpm | LEAST_SQUARES_MEAN |
| SECONDARY | Quality of Life - Parent | Average of Months 12, 24, and 36 visits | Data was not collected on 3 participants in the daily prednisone arm, 1 participants in the deflazacort arm and 2 participant in the intermittent prednisone arm. | score on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group51
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Forced Vital Capacity | Daily Prednisone | 1.4 | Standard Error 0.03 | liters | |
| Forced Vital Capacity | Daily Deflazacort | 1.4 | Standard Error 0.03 | liters | |
| Forced Vital Capacity | Intermittent Prednisone | 1.5 | Standard Error 0.03 | liters | |
| Rise From the Floor Velocity | Daily Prednisone | 0.24 | Standard Error 0.01 | rise/sec | |
| Rise From the Floor Velocity | Daily Deflazacort | 0.24 | Standard Error 0.01 | rise/sec | |
| Rise From the Floor Velocity | Intermittent Prednisone | 0.18 | Standard Error 0.01 | rise/sec | |
| Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | Daily Prednisone | 71.2 | Standard Error 2.3 | score on a scale | |
| Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | Daily Deflazacort | 67.8 | Standard Error 2.3 | score on a scale | |
| Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score | Intermittent Prednisone | 65.1 | Standard Error 2.3 | score on a scale | |
| North Star Ambulatory Assessment (NSAA) Score | Daily Prednisone | 23.7 | Standard Error 0.6 | score on a scale |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Daily Prednisone | 65 | |
| Overall Study | STARTED | Daily Deflazacort | 65 | |
| Overall Study | STARTED | Intermittent Prednisone | 66 | |
| Overall Study | COMPLETED | Daily Prednisone | 54 | |
| Overall Study | COMPLETED | Daily Deflazacort | 54 | |
| Overall Study | COMPLETED | Intermittent Prednisone | 56 | |
| Overall Study | NOT COMPLETED | Daily Prednisone | 11 | |
| Overall Study | NOT COMPLETED | Daily Deflazacort | 11 | |
| Overall Study | NOT COMPLETED | Intermittent Prednisone | 10 |
Source ClinicalTrials.gov
Adverse events210
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 | Cushingoid | Metabolism and nutrition disorders | Daily Prednisone | 6 | 65 | |
| other | Cushingoid | Metabolism and nutrition disorders | Daily Deflazacort | 6 | 65 | |
| other | Cushingoid | Metabolism and nutrition disorders | Intermittent Prednisone | 3 | 66 | |
| other | Cataracts | Eye disorders | Daily Prednisone | 1 | 65 | |
| other | Cataracts | Eye disorders | Daily Deflazacort | 5 | 65 | |
| other | Cataracts | Eye disorders | Intermittent Prednisone | 0 | 66 | |
| other | Vomiting | Gastrointestinal disorders | Daily Prednisone | 19 | 65 | |
| other | Vomiting | Gastrointestinal disorders | Daily Deflazacort | 16 | 65 | |
| other | Vomiting | Gastrointestinal disorders | Intermittent Prednisone | 15 | 66 | |
| other | Diarrhea | Gastrointestinal disorders | Daily Prednisone | 9 | 65 |
1 to 10 of 210Source ClinicalTrials.gov
See all 210 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Daily Prednisone | 0 | 65 |
| Total, all-cause mortality | Daily Deflazacort | 0 | 65 |
| Total, all-cause mortality | Intermittent Prednisone | 0 | 66 |
| Total, other adverse events | Daily Prednisone | 61 | 65 |
| Total, other adverse events | Daily Deflazacort | 58 | 65 |
| Total, other adverse events | Intermittent Prednisone | 55 | 66 |
| Total, serious adverse events | Daily Prednisone | 5 | 65 |
| Total, serious adverse events | Daily Deflazacort | 10 | 65 |
| Total, serious adverse events | Intermittent Prednisone | 5 | 66 |
Source ClinicalTrials.gov
Papers62
The papers the trial lists on ClinicalTrials.gov.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U01NS061799 | FOR-DMD: Double-blind randomized trial to optimize steroid regimen in Duchenne MD | GRIGGS, ROBERT C; GUGLIERI, MICHELA | UNIVERSITY OF ROCHESTER | NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE | 2010 to 2019 |
Source NIH RePORTER
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.
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