A Phase 2 interventional study of MNK-1411 and Placebo in Muscular Dystrophy, Duchenne, sponsored by Mallinckrodt ARD LLC. Terminated at 16 sites in 8 countries. Open to male participants aged 4 Years to 8 Years. Per ClinicalTrials.gov, last updated 2021-03-16.
Sponsored by Mallinckrodt ARD LLC (part of Keenova Therapeutics plc) · Phase 2, Interventional, and Treatment
Among 232 studies in Muscular Dystrophy, Duchenne with a site in United States — 38 open to participants now, 62 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · United States.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, this one is the 64th largest by enrollment of the 171 interventional ones, is one of 21 that started in 2018, is one of 57 in phase 2, and was the 123rd registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's enrollment of 44 is above the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →38 of the 232 studies in Muscular Dystrophy, Duchenne with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 60 take children under 18; 12 take healthy volunteers.
This study: 4 Years to 8 Years · male · no healthy volunteers.
The full table on the hub →232 studies; the count open now beside each. This study's row is marked.
First in 2000; this study’s year is marked. 21 started in 2018.
Median enrollment 25 across the 171 interventional studies, 75 across the 55 observational. Of the 160 that completed or were terminated, 77 (48%) have results posted.
130 of 232 have an industry lead sponsor. The 10 most frequent:
Mallinckrodt ARD LLC is the lead sponsor of 3 studies on the registry; none are open to participants now.
Mallinckrodt ARD LLC has 1 study filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
19 of the studies in Muscular Dystrophy, Duchenne with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT06290713University of FloridaVasodilator and Exercise Study for DMD (VASO-REx) | 2026-11 |
| NCT06450639Hoffmann-La RocheA Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) | 2026-11-18 |
| NCT03368742Solid Biosciences Inc.Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | 2026-11-30 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, from their registry updates:
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
The main purpose of this study is to determine the effect of MNK-1411 on motor function in participants with Duchenne Muscular Dystrophy (DMD). Information is collected only from caretakers who are fluent in English, using the Pediatric Outcomes Data Collection Instrument (PODCI).
The PODCI is a validated 86-question instrument completed by the parent or legal guardian of children 2 to 10 years of age to assess a variety of health outcome measures (Uzark et al, 2012). This study will only collect information for the PODCI domains of sports and physical functioning and transfer/basic mobility.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1
Drug: MNK-1411
Participants receive placebo at a volume appropriate to body weight during Period 1
Other: Placebo
All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2
Drug: MNK-1411
MNK-1411 (1 mg/mL suspension) for subcutaneous injection
Also known as: Cosyntropin acetate, Tetracosactide Hexaacetate
Placebo suspension for subcutaneous injection
Also known as: Matching Placebo
Time to Complete 10 Meter Walk/Run[
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
Time frame: Baseline, Week 24
North Star Ambulatory Assessment (NSAA) Score
The NSAA is comprised of 17 items, each of which is graded using the standard scorecard. Each assessment is 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. The subscale scores are summed for a total score ranging from 0 to 34. The higher the total score, the better the outcome.
Time frame: Baseline, Week 24
Time to Climb 4 Standardized Stairs
Time to Climb 4 Standardized Stairs is a motor performance test
Time frame: Baseline, Week 24
Time to Stand From a Supine Position
Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
Time frame: Baseline, Week 24
Quantitative Muscle Testing Scores at Baseline
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Time frame: Baseline
Quantitative Muscle Testing Scores at Week 24
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
Time frame: Week 24
Summary of Adverse Events in the Blinded Treatment Period
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Time frame: within 28 weeks
Summary of Adverse Events in the Open Label Period
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
Time frame: within 28 weeks
Twenty-four participants with Duchenne Muscular Dystrophy (DMD) who chose to discontinue the double-blind period prior to Week 24 entered the open label extension (OLE, Period 2). Participants who did not enter OLE Period were followed up to Week 28.
| Milestone | Period 1: MNK-1411 | Period 1: Placebo | Period 2: MNK-1411 |
|---|---|---|---|
| Started | 29 | 15 | 0 |
| Completed | 20 | 9 | 0 |
| Not completed | 9 | 6 | 0 |
| Withdrew: Physician decision | 0 | 1 | 0 |
| Withdrew: Adverse event | 1 | 1 | 0 |
| Withdrew: Study terminated by sponsor | 8 | 4 | 0 |
| Milestone | Period 1: MNK-1411 | Period 1: Placebo | Period 2: MNK-1411 |
|---|---|---|---|
| Started | 0 | 0 | 24 |
| Completed | 0 | 0 | 2 |
| Not completed | 0 | 0 | 22 |
| Withdrew: Physician decision | 0 | 0 | 1 |
| Withdrew: Study terminated by sponsor | 0 | 0 | 21 |
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
| seconds | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| at Baseline | 5.9 (4.7 to 22.3) | 7.8 (3.9 to 13.0) |
| at Week 24 | 5.4 (4.1 to 8.9) | 8.7 (3.3 to 18.3) |
The NSAA is comprised of 17 items, each of which is graded using the standard scorecard. Each assessment is 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. The subscale scores are summed for a total score ranging from 0 to 34. The higher the total score, the better the outcome.
| score on a scale | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| at Baseline | 17.9 ± 6.80 | 17.1 ± 6.40 |
| at Week 24 | 20.5 ± 7.94 | 16.6 ± 8.82 |
Time to Climb 4 Standardized Stairs is a motor performance test
| seconds | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| at Baseline | 8.52 ± 8.88 | 8.47 ± 4.34 |
| at Week 24 | 4.71 ± 2.45 | 15.09 ± 13.84 |
Time to stand from a supine position is a motor function test to measure the functional capability in subjects with DMD.
| seconds | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| at Baseline | 11.14 ± 9.08 | 15.03 ± 12.45 |
| at Week 24 | 7.65 ± 4.99 | 24.89 ± 26.48 |
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
| Newtons | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| Knee flexion | 26.61 ± 14.17 | 29.87 ± 14.17 |
| Knee extension | 28.99 ± 16.24 | 26.64 ± 15.27 |
Quantitative muscle testing measured strength-knee flexion and extension measured in Newtons, using a dynamometer
| Newtons | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| Knee flexion | 33.64 ± 1578 | 25.27 ± 13.35 |
| Knee extension | 26.61 ± 14.17 | 29.87 ± 15.13 |
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
| Participants | Period 1: MNK-1411 | Period 1: Placebo |
|---|---|---|
| Exposed | 29 | 15 |
| Affected by serious adverse events | 0 | 1 |
| Affected by non-serious adverse events | 22 | 15 |
| Died from any cause | 0 | 0 |
Clinically significant changes in vital signs, height, weight, immunogenicity and laboratory assessments were reported as adverse events (AEs)
| Participants | Period 2: MNK-1411 |
|---|---|
| Exposed | 24 |
| Affected by serious adverse events | 2 |
| Affected by non-serious adverse events | 11 |
| Died from any cause | 0 |
Collected over within 28 weeks. Non-serious events are listed at a 3% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Period 1: MNK-1411 | 0/29 (0%) | 0/29 (0%) | 22/29 (75.9%) |
| Period 1: Placebo | 0/15 (0%) | 1/15 (6.7%) | 15/15 (100%) |
| Period 2: MNK-1411 | 0/24 (0%) | 2/24 (8.3%) | 11/24 (45.8%) |
| Event | Period 1: MNK-1411 | Period 1: Placebo | Period 2: MNK-1411 |
|---|---|---|---|
| Muscle disorderMusculoskeletal and connective tissue disorders | 0/29 | 1/15 | 0/24 |
| RhabdomyolysisMusculoskeletal and connective tissue disorders | 0/29 | 0/15 | 1/24 |
| nary tract infectionInfections and infestations | 0/29 | 0/15 | 1/24 |
| Event | Period 1: MNK-1411 | Period 1: Placebo | Period 2: MNK-1411 |
|---|---|---|---|
| Weight increasedInvestigations | 7/29 | 0/15 | 3/24 |
| Injection site massGeneral disorders | 1/29 | 3/15 | 1/24 |
| GastroenteritisInfections and infestations | 0/29 | 3/15 | 1/24 |
| Injection site erythemaGeneral disorders | 4/29 | 1/15 | 1/24 |
| Injection site indurationGeneral disorders | 4/29 | 1/15 | 2/24 |
| Face oedemaGeneral disorders | 1/29 | 2/15 | 0/24 |
| PyrexiaGeneral disorders | 1/29 | 2/15 | 1/24 |
| CoughRespiratory, thoracic and mediastinal disorders | 2/29 | 2/15 | 0/24 |
| PharyngitisInfections and infestations | 0/29 | 2/15 | 0/24 |
| Upper respiratory tract infectionInfections and infestations | 1/29 | 2/15 | 1/24 |
All participants enrolled into the study.
| Age, Customized(Participants) | Period 1: MNK-1411 | Period 1: Placebo | Total |
|---|---|---|---|
| Children (2-11 Years) | 29 | 15 | 44 |
| Sex: Female, Male(Participants) | Period 1: MNK-1411 | Period 1: Placebo | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 29 | 15 | 44 |
| Race and Ethnicity Not Collected(Participants) | Period 1: MNK-1411 | Period 1: Placebo | Total |
|---|---|---|---|
| Count of participants | — | — | 0 |
| Region of Enrollment(participants) | Period 1: MNK-1411 | Period 1: Placebo | Total |
|---|---|---|---|
| Turkey | 0 | 0 | 3 |
| United States | 0 | 0 | 10 |
| Italy | 0 | 0 | 2 |
| Mexico | 0 | 0 | 18 |
| Israel | 0 | 0 | 1 |
| Bulgaria | 0 | 0 | 2 |
| Serbia | 0 | 0 | 2 |
| Spain | 0 | 0 | 6 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — Discussion of statistical endpoints and analysis are included in manuscripts. Summary aggregate (basic) results (including adverse events information) and the study protocol are made available on clinicaltrials.gov (NCT03400852) when required by regulation. Individual de-identified patient data will not be disclosed. Requests for additional information should be directed to the company at medinfo@mnk.com.
Ran from Jul 2018 to primary completion in Feb 2020, 18 months.
Results were first posted in Feb 2021, 11 months after primary completion.
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Muscular Dystrophy, Duchenne →