Trial · NCT02851797

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

NCT02851797, Phase 3, Completed, Italfarmaco; results first posted 2023-02-02

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy. Trials, record NCT02851797. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02851797

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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 Duchenne Muscular Dystrophy
NCT number: NCT02851797
Status: Completed
Phases: Phase 3
Enrollment: 179 participants, actual
Lead sponsor: Italfarmaco
Conditions: Duchenne Muscular Dystrophy
Interventions: givinostat, placebo
First posted: 2016-08-02
Last updated: 2023-02-02
Other registries' IDs: eudract:2016-000401-36
Results first posted: 2023-02-02

Source ClinicalTrials.gov

Registered outcomes9

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Mean Change From Baseline in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment	Baseline and 18 months	The time (in seconds) to climb 4 standard-sized stairs is a TFT that represents stair-climbing ability. The test was evaluated by qualified functional evaluators (ie, physiotherapists) who were different from the site personnel who reviewed subjects' safety results. The test was performed in a standardised manner described in a specific site manual. Baseline 4SC was the measurement taken at the randomization assessment, unless this was missing, in which case baseline was taken as the last non missing value recorded prior to or on the date of first study treatment. The shorter the time, the better the outcome.
Secondary	Mean Change From Baseline in Time to Rise From Floor After 18 Months of Treatment	Baseline and 18 months	An analysis of time (in seconds) to rise from the floor by change from baseline at 18 months is presented for the Target Population in the ITT analysis set. The shorter the time, the better the outcome.
Secondary	Mean Change From Baseline in the Six-minute Walking Test (6MWT) After 18 Months of Treatment	Baseline and 18 months	This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes. The 6-Minute Walk Test is a useful measure of functional capacity targeted at people with at least moderately severe impairment. A modified version of the 6MWT recommended by American Thoracic Society (2002) for use in adults was performed. The longer the walked distance the better the outcome.
Secondary	Mean Change From Baseline in Total North Star Ambulatory Assessment (NSAA) Score After 18 Months of Treatment	Baseline and 18 months	The total 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 17 items of the NSAA, ranging from standing to running 10 meters, were graded using the standard score card with each assessment rated as 0 - unable to achieve independently, 1 - modified method but achieves goal independent of physical assistance from another, or 2 - normal with no obvious modification of activity. This scale is ordinal with 0 as the minimum score (indicating full disfunctionality, i.e. the worst outcome) and with 34 as the maximum score indicating fully-independent function (the best outcome).
Secondary	Cumulative Loss of Function on the NSAA	over 18 months	Subject cumulative number of failures across all postbaseline visits was the endpoint of interest for analysis. For each subject at each postbaseline visit, failure to perform each of the 17 items of the NSAA was assessed, where "failure" was defined as a score transition from 2 or 1 at baseline to 0 at the respective visit. The total number of failed items for the visit was calculated (maximum of 17 failed items per visit per subject). The subject's cumulative number of failures across all visits was the sum of the total failures at each postbaseline visit.
Secondary	Mean Change From Baseline of Muscle Strength Normalized Overtime	Baseline and 18 months	The mean change of muscle strength normalized was evaluated by knee extension and elbow flexion normalized by subject weight, both measured by hand-held myometry (HHM).
Secondary	Mean Change From Baseline in Vastus Lateralis Muscle Fat Fraction (VL MFF) at 18 Months	Baseline and 18 months	Vastus lateralis muscle fat fraction (VL MFF) was expressed as fat infiltration in this muscle. Fat infiltration was assessed by Magnetic Resonance (MRS).
Secondary	Number of Subjects Experiencing Treatment-emergent AEs (TEAEs), Serious AEs (SAEs), Mild TEAE Moderate TEAE, Severe TEAE	Baseline through end of study, that is the end of 18° month	Adverse Events are unfavorable changes in health, including abnormal laboratory findings, that occur in trial participants during the clinical trial or within a specified period following the trial. Serious Adverse Events include adverse events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.
Secondary	Evaluation of Acceptability/Palatability of the Oral Suspension	Week 4, EOS, early withdrawal	Acceptability and palatability of the oral suspension over time are presented. More in details, child perception of the medicine at the three timepoints hereunder specified; parent perception of the medicine based on the child's reaction at the same three timepoints; and parent problems administering the medication at the same timepoints are reported.

Source ClinicalTrials.gov

Outcomes9

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 in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment	Baseline and 18 months	ITT analysis set: the intent-to-treat (ITT) analysis set included all subjects who were randomised, received at least one dose of study drug, and had at least 1 non-missing post-baseline 4SC measure or missing post-baseline 4SC measure due to being either non-ambulatory or otherwise physically unable to perform the assessment, irrespective of any deviation from the protocol or premature discontinuation.	seconds	LEAST_SQUARES_MEAN
SECONDARY	Mean Change From Baseline in Time to Rise From Floor After 18 Months of Treatment	Baseline and 18 months	ITT analysis set: the intent-to-treat (ITT) analysis set included all subjects who were randomised, received at least one dose of study drug, and had at least 1 non-missing post-baseline 4SC measure or missing post-baseline 4SC measure due to being either non-ambulatory or otherwise physically unable to perform the assessment, irrespective of any deviation from the protocol or premature discontinuation.	seconds	LEAST_SQUARES_MEAN
SECONDARY	Mean Change From Baseline in the Six-minute Walking Test (6MWT) After 18 Months of Treatment	Baseline and 18 months	ITT analysis set: the intent-to-treat (ITT) analysis set included all subjects who were randomised, received at least one dose of study drug, and had at least 1 non-missing post-baseline 4SC measure or missing post-baseline 4SC measure due to being either non-ambulatory or otherwise physically unable to perform the assessment, irrespective of any deviation from the protocol or premature discontinuation.	meters	LEAST_SQUARES_MEAN
SECONDARY	Mean Change From Baseline in Total North Star Ambulatory Assessment (NSAA) Score After 18 Months of Treatment	Baseline and 18 months	ITT analysis set: the intent-to-treat (ITT) analysis set included all subjects who were randomised, received at least one dose of study drug, and had at least 1 non-missing post-baseline 4SC measure or missing post-baseline 4SC measure due to being either non-ambulatory or otherwise physically unable to perform the assessment, irrespective of any deviation from the protocol or premature discontinuation.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Cumulative Loss of Function on the NSAA	over 18 months	ITT analysis set: the intent-to-treat (ITT) analysis set included all subjects who were randomised, received at least one dose of study drug, and had at least 1 non-missing post-baseline 4SC measure or missing post-baseline 4SC measure due to being either non-ambulatory or otherwise physically unable to perform the assessment, irrespective of any deviation from the protocol or premature discontinuation.	cumulative number of failures	NUMBER
SECONDARY	Mean Change From Baseline of Muscle Strength Normalized Overtime	Baseline and 18 months	ITT analysis set: the intent-to-treat (ITT) analysis set included all subjects who were randomised, received at least one dose of study drug, and had at least 1 non-missing post-baseline 4SC measure or missing post-baseline 4SC measure due to being either non-ambulatory or otherwise physically unable to perform the assessment, irrespective of any deviation from the protocol or premature discontinuation.	Newtons/kg	LEAST_SQUARES_MEAN
SECONDARY	Mean Change From Baseline in Vastus Lateralis Muscle Fat Fraction (VL MFF) at 18 Months	Baseline and 18 months	MR cohort: the MR cohort included all subjects in the Target Population who were randomised to study treatment and completed at least one post-baseline MRI/MRS assessment.	percentage of fat	LEAST_SQUARES_MEAN
SECONDARY	Number of Subjects Experiencing Treatment-emergent AEs (TEAEs), Serious AEs (SAEs), Mild TEAE Moderate TEAE, Severe TEAE	Baseline through end of study, that is the end of 18° month	SAF analysis set: the safety analysis set included all subjects who were randomized and received at least 1 dose of study drug.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Evaluation of Acceptability/Palatability of the Oral Suspension	Week 4, EOS, early withdrawal	SAF analysis set: the safety analysis set included all subjects who were randomised and received at least 1 dose of study drug.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group86

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Mean Change From Baseline in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment	Givinostat	1.27	Standard Error 0.040	seconds
Mean Change From Baseline in 4 Standard Stairs (4SC) Climb After 18 Months of Treatment	Placebo	1.48	Standard Error 0.058	seconds
Mean Change From Baseline in Time to Rise From Floor After 18 Months of Treatment	Givinostat	9.33	95% Confidence Interval 5.821 to 12.838	seconds
Mean Change From Baseline in Time to Rise From Floor After 18 Months of Treatment	Placebo	12.61	95% Confidence Interval 7.491 to 17.724	seconds
Mean Change From Baseline in the Six-minute Walking Test (6MWT) After 18 Months of Treatment	Givinostat	-38.43	95% Confidence Interval -50.704 to -26.153	meters
Mean Change From Baseline in the Six-minute Walking Test (6MWT) After 18 Months of Treatment	Placebo	-48.38	95% Confidence Interval -66.288 to -30.482	meters
Mean Change From Baseline in Total North Star Ambulatory Assessment (NSAA) Score After 18 Months of Treatment	Givinostat	-2.66	95% Confidence Interval -3.563 to -1.759	score on a scale
Mean Change From Baseline in Total North Star Ambulatory Assessment (NSAA) Score After 18 Months of Treatment	Placebo	-4.58	95% Confidence Interval -5.891 to -3.260	score on a scale
Cumulative Loss of Function on the NSAA	Givinostat	3.42	95% Confidence Interval 2.692 to 4.334	cumulative number of failures
Cumulative Loss of Function on the NSAA	Placebo	5.56	95% Confidence Interval 4.002 to 7.715	cumulative number of failures

1 to 10 of 86Source ClinicalTrials.gov

See all 86 results by group

Participant flow18

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Givinostat	118
Overall Study	STARTED	Placebo	61
Overall Study	All Enrolled Subjects	Givinostat	118
Overall Study	All Enrolled Subjects	Placebo	61
Overall Study	ITT Population	Givinostat	118
Overall Study	ITT Population	Placebo	61
Overall Study	Safety Set	Givinostat	118
Overall Study	Safety Set	Placebo	61
Overall Study	PK Analysis Set	Givinostat	117
Overall Study	PK Analysis Set	Placebo	0

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events92

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	Diarrhoea	Gastrointestinal disorders	Givinostat	43	118	117
other	Diarrhoea	Gastrointestinal disorders	Placebo	11	61	16
other	Vomiting	Gastrointestinal disorders	Givinostat	34	118	64
other	Vomiting	Gastrointestinal disorders	Placebo	8	61	12
other	Abdominal pain	Gastrointestinal disorders	Givinostat	25	118	52
other	Abdominal pain	Gastrointestinal disorders	Placebo	9	61	16
other	Abdominal pain upper	Gastrointestinal disorders	Givinostat	17	118	21
other	Abdominal pain upper	Gastrointestinal disorders	Placebo	7	61	9
other	Nausea	Gastrointestinal disorders	Givinostat	8	118	9
other	Nausea	Gastrointestinal disorders	Placebo	4	61	6

1 to 10 of 92Source ClinicalTrials.gov

See all 92 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Givinostat	0	118
Total, all-cause mortality	Placebo	0	61
Total, other adverse events	Givinostat	112	118
Total, other adverse events	Placebo	57	61
Total, serious adverse events	Givinostat	8	118
Total, serious adverse events	Placebo	2	61

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
38508835	DERIVED

Source PubMed

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
Givinostat vs Placebo: deaths from any cause	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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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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Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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