Trial · NCT03238235
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
NCT03238235, Phase 2, Completed, Italfarmaco; results first posted 2024-11-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy. Trials, record NCT03238235. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03238235
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
- NCT number
- NCT03238235
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 51 participants, actual
- Lead sponsor
- Italfarmaco
- Conditions
- Becker Muscular Dystrophy
- Interventions
- Givinostat, Placebo
- First posted
- 2017-08-03
- Last updated
- 2024-11-18
- Other registries' IDs
- eudract:2017-001629-41
- Results first posted
- 2024-11-18
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies | after 12 months of treatment (at Visit 11) | The primary efficacy assessment was the mean total fibrosis (%) on log scale assessed through histological examination of bicep muscle biopsies at two timepoints (At baseline and at Visit 11). More particularly, patients underwent two biopsies of muscle from the brachial biceps: the first before starting the study treatment (Visit 2, baseline), and the second at the end of treatment (Visit 11). For each patient, the percentage of total fibrosis was calculated as log of the least square mean of the available fields at each evaluation. |
| Secondary | Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus | after 12 months of treatment (at Visit 11) | Evaluation was performed comparing Magnetic Resonance Spectroscopy (MRS) before and after 12 months of treatment with Givinostat versus placebo. Please note that Statistics data are expressed as log least square mean (IC 95%) for the vastus lateralis and as least square mean (IC 95%) back-transformed on the original scale for the soleus. |
| Secondary | Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles | after 12 months of treatment (at Visit 11) | Evaluation was performed comparing Dixon Magnetic Resonance Imaging (MRI) before and after 12 months of treatment with Givinostat versus placebo. The lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle. Please note that statistics data are expressed as: Least Square Means (95% CI) for whole thigh, quadriceps, hamstrings, triceps surae and pelvis girdle; while as Log Least Square Means (95% CI) for medial thigh. |
| Secondary | Mean Change From Baseline to Visit 11 in Cross-sectional Area (CSA), in cm2 of Lower Limb Muscles | after 12 months of treatment (Visit 11) | Evaluation was performed comparing Dixon MRI findings before and after 12 months of treatment with Givinostat versus placebo. Dixon technique has advantages in multiple applications for evaluation of musculoskeletal system diseases. It allows more robust fat suppression than do other sequences and can be used in combination with multiple different sequences (GRE and SE) and using different weightings (T1, T2, or proton density). The lower limb muscles assessed were: whole thigh, quadriceps, medial thigh, hamstrings, triceps surae and pelvic girdle (data showed as least square mean) |
| Secondary | Mean Change From Baseline to Visit 11 in Contractile Area of Lower Limb Muscles (MRI) | after 12 months of treatment | A summary of mean change in the contractile area of lower limb muscles comparing MRI findings before and after 12 months of treatment in the ITT set is reported. The lower limb muscles considered are the same as the outcome 3. Please note that statistic data are expressed as Least Square Means (95% CI) except for Hamstrings which is expressed as Log Least Square Means. |
| Secondary | Mean Change From Baseline to Visit 11 in Biopsy Histology Parameters: CSA by Type (I or II Fibers), Total CSA | After 12 months of treatment (Visit 11) | A summary of the mean change in cross-sectional area (CSA) by type after 12 months of treatment in the ITT set is reported. Please note that descriptive statistic data are expressed as Log Least Square Means (95% CI) for CSA type I fibers, and as Least Square Means (95% CI) for CSA type II fibers and Total CSA. |
| Secondary | Mean Change From Baseline to Visit 11 in Percentage for the Following Histology Parameters: Fibers With Nuclear Centralizations (%), Total Number of Fibers (%), Regenerative Fibers (%). | after 12 months of treatment (Visit 11) | Evaluation of histologic parameters such as Fiber with nuclear centralizations (%), Total number of fibers (%), and Regenerative fibers (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean for Regenerative fibers (%), and total number of fibers (Slides I), while are expressed as least square mean for Fibers with nuclear centralizations (%) and total number of fibers (Slides II and III). |
| Secondary | Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameter MFA(%) | after 12 months of treatment (Visit 11) | Evaluation of histology parameters such as Muscle Fibers Area \[MFA\](%), were performed comparing muscle biopsies after 12 months of treatment with Givinostat. MFA fraction was determined from biopsies using the mean of available fields. Please note that descriptive statistic data are expressed as least square mean. |
| Secondary | Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameters Adipose Tissue (%) | after 12 months of treatment (Visit 11) | Evaluation of histology parameters such as Adipose tissue (%) were performed comparing muscle biopsies after 12 months of treatment with Givinostat. Please note that descriptive statistic data are expressed as log least square mean. |
| Secondary | Mean Change From Baseline to Visit 11 in Other Histological Structures | after 12 months of treatment (Visit 11) | Evaluation of other histological structures like necrotic cells, vessels and any other nonconnective tissue present in the microscopic field were performed comparing muscle biopsies after 12 months of treatment with Givinostat. The value is shown as % compared to the arithmetic mean of the two positive control (i.e., healthy subjects) bands present in the same western blot. Please note that descriptive statistic data are expressed as log least square mean. |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes25
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies | after 12 months of treatment (at Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | log[percentage of total fibrosis] | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus | after 12 months of treatment (at Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | log[percentage of fat in muscle] | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles | after 12 months of treatment (at Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | percentage of fat in muscle | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Cross-sectional Area (CSA), in cm2 of Lower Limb Muscles | after 12 months of treatment (Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | centimeters^2 | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Contractile Area of Lower Limb Muscles (MRI) | after 12 months of treatment | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | centimeters^2 | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Biopsy Histology Parameters: CSA by Type (I or II Fibers), Total CSA | After 12 months of treatment (Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | micrometers^2 | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Percentage for the Following Histology Parameters: Fibers With Nuclear Centralizations (%), Total Number of Fibers (%), Regenerative Fibers (%). | after 12 months of treatment (Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. | percentage of fibers | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameter MFA(%) | after 12 months of treatment (Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | percentage of muscle fiber area | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Percentage for the Biopsy Histology Parameters Adipose Tissue (%) | after 12 months of treatment (Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | percentage of total tissue | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline to Visit 11 in Other Histological Structures | after 12 months of treatment (Visit 11) | Intent-To-Treat Analysis (ITT) Set: all patients assigned treatment by randomization who received at least one dose of study medication. According to the intent to treat principle, patients were analyzed according to the treatment assigned. Efficacy analyses were performed on the ITT set. | percentage of total tissue | LEAST_SQUARES_MEAN |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 outcomesResults by group132
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies | Givinostat | -0.017 | 95% Confidence Interval -0.367 to 0.333 | log[percentage of total fibrosis] | |
| Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies | Placebo | -0.054 | 95% Confidence Interval -0.507 to 0.399 | log[percentage of total fibrosis] | |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus | Givinostat | Vastus Lateralis | 0.017 | 95% Confidence Interval -0.053 to 0.086 | log[percentage of fat in muscle] |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus | Placebo | Vastus Lateralis | 0.064 | 95% Confidence Interval -0.019 to 0.147 | log[percentage of fat in muscle] |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus | Givinostat | Soleus | -4.287 | 95% Confidence Interval -6.126 to -2.447 | log[percentage of fat in muscle] |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Vastus Lateralis and Soleus | Placebo | Soleus | -4.021 | 95% Confidence Interval -6.404 to -1.637 | log[percentage of fat in muscle] |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles | Givinostat | Whole Thigh | 0.642 | 95% Confidence Interval -0.328 to 1.611 | percentage of fat in muscle |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles | Placebo | Whole Thigh | 1.996 | 95% Confidence Interval 0.813 to 3.180 | percentage of fat in muscle |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles | Givinostat | Quadriceps | 0.610 | 95% Confidence Interval -0.506 to 1.726 | percentage of fat in muscle |
| Mean Change From Baseline to Visit 11 in the Percentage of Fat Fraction of Lower Limb Muscles | Placebo | Quadriceps | 2.572 | 95% Confidence Interval 1.227 to 3.918 | percentage of fat in muscle |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 results by groupParticipant flow14
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Givinostat | 34 | |
| Overall Study | STARTED | Placebo | 17 | |
| Overall Study | Per Protocol | Givinostat | 25 | |
| Overall Study | Per Protocol | Placebo | 14 | |
| Overall Study | ITT | Givinostat | 34 | |
| Overall Study | ITT | Placebo | 17 | |
| Overall Study | Safety | Givinostat | 34 | |
| Overall Study | Safety | Placebo | 17 | |
| Overall Study | PK | Givinostat | 34 | |
| Overall Study | PK | Placebo | 17 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 participant flowAdverse events148
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 | Lymphocytosis | Blood and lymphatic system disorders | Givinostat | 1 | 34 | 1 |
| other | Lymphocytosis | Blood and lymphatic system disorders | Placebo | 0 | 17 | 0 |
| other | Sinus tachycardia | Cardiac disorders | Givinostat | 0 | 34 | 0 |
| other | Sinus tachycardia | Cardiac disorders | Placebo | 1 | 17 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Givinostat | 2 | 34 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 0 | 17 | 0 |
| other | Abdominal pain upper | Gastrointestinal disorders | Givinostat | 3 | 34 | 3 |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 1 | 17 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Givinostat | 16 | 34 | 16 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 0 | 17 | 0 |
1 to 10 of 148Source ClinicalTrials.gov
See all 148 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Givinostat | 0 | 34 |
| Total, all-cause mortality | Placebo | 0 | 17 |
| Total, other adverse events | Givinostat | 30 | 34 |
| Total, other adverse events | Placebo | 9 | 17 |
| Total, serious adverse events | Givinostat | 0 | 34 |
| Total, serious adverse events | Placebo | 0 | 17 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Givinostat vs Placebo: Percentage of Patients Who Fell During the 6MWT | Trial comparisons |
| Givinostat vs Placebo: Count of Participant Who Lose Ambulation During the Study (From Baseline to Month 12) | Trial comparisons |
| Givinostat vs Placebo: deaths from any cause | Trial comparisons |
| Givinostat vs Placebo: Percentage of Patients With < 10% Worsening in 6MWT After 12 Months of Treatment. | Trial comparisons |
| Givinostat vs Placebo: serious adverse events | 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/NCT03238235
GET https://conductscience.org/api/trials/v1/NCT03238235/registered?page=2