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Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03068754
Recruitment Status : Terminated (As recommended by the study's independent Data and Safety Monitoring Board (DSMB))
First Posted : March 3, 2017
Results First Posted : September 1, 2020
Last Update Posted : September 3, 2020
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Amyotrophic Lateral Sclerosis
Interventions Drug: Acthar
Drug: Placebo
Enrollment 143
Recruitment Details  
Pre-assignment Details Of 207 potential participants screened, 143 were randomized to begin treatment
Arm/Group Title Arm A: Randomized Treatment Period (RTP) Acthar Gel Arm B: RTP Placebo Arm C: Open Label Extension Period (OLE) Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Hide Arm/Group Description

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period had 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period had 3 weeks of simulated tapering, ending their participation by Week 39.

Participants who received Acthar Gel during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. Participants who received Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Period Title: Randomized Treatment Period (RTP)
Started 95 48 0 [1] 0
Intention to Treat (ITT) Population 95 48 0 0
Safety Population 95 47 0 0
Modified ITT (mITT) 93 46 0 0
Completed Taper [2] 58 28 0 0
Completed 36 Weeks of Treatment 28 9 0 0
Completed RTP [3] 27 9 0 0
Enrolled in OLE Period 25 8 0 0
Completed 28 9 0 0
Not Completed 67 39 0 0
Reason Not Completed
Adverse Event             1             3             0             0
Death             2             2             0             0
Disease progression             1             2             0             0
Lost to Follow-up             1             0             0             0
Withdrawal by Subject             6             2             0             0
Discontinued by Physician             1             0             0             0
Subject Decision based on Compensation             0             1             0             0
Trial Terminated by Sponsor             55             29             0             0
[1]
OLE Arms were not included during the RTP
[2]
Defined as completing the End of RTP Taper Visit at about Week 39 or an early termination visit
[3]
Defined as completing 36 weeks of treatment, followed by Week 39 visit or entering the OLE period
Period Title: Open Label Extension Period (OLE)
Started [1] 0 [2] 0 25 8
Completed Taper [3] 0 0 19 7
Completed OLE Period [4] 0 0 3 2
Completed Treatment in OLE [5] 0 0 4 2
Completed [6] 0 0 4 2
Not Completed 0 0 21 6
Reason Not Completed
Adverse Event             0             0             0             1
Death             0             0             0             1
Disease progression             0             0             3             1
Withdrawal by Subject             0             0             5             0
Subject Discontinued for Non-efficacy             0             0             1             0
Trial Terminated by Sponsor             0             0             12             3
[1]
Safety Population in the OLE - 4 participants who completed RTP were not treated in the OLE
[2]
RTP arms were not included during the OLE period
[3]
Completed End of Taper visits after Week 84 visit or being terminated early from the OLE period
[4]
Completed Week 84 visit, End of Taper visit and follow up visit
[5]
Completed Week 84 visit
[6]
Completed treatment in OLE period
Arm/Group Title Arm A: RTP Acthar Gel Arm B: RTP Placebo Total
Hide Arm/Group Description

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Total of all reporting groups
Overall Number of Baseline Participants 95 47 142
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Safety population
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 95 participants 47 participants 142 participants
55.9  (10.80) 56.3  (10.68) 56.0  (10.72)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 47 participants 142 participants
Female
35
  36.8%
21
  44.7%
56
  39.4%
Male
60
  63.2%
26
  55.3%
86
  60.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 47 participants 142 participants
Hispanic or Latino
38
  40.0%
19
  40.4%
57
  40.1%
Not Hispanic or Latino
57
  60.0%
28
  59.6%
85
  59.9%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 47 participants 142 participants
American Indian or Alaska Native
5
   5.3%
2
   4.3%
7
   4.9%
Asian
1
   1.1%
1
   2.1%
2
   1.4%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   2.1%
0
   0.0%
2
   1.4%
White
72
  75.8%
33
  70.2%
105
  73.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
15
  15.8%
11
  23.4%
26
  18.3%
1.Primary Outcome
Title Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Hide Description

The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival.

ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory)

A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.

Time Frame Baseline, Week 36
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Safety Population
Arm/Group Title Arm A: RTP Acthar Gel Arm B: RTP Placebo
Hide Arm/Group Description:

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Overall Number of Participants Analyzed 95 47
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline Number Analyzed 95 participants 47 participants
34.7  (6.01) 34.3  (5.84)
Week 36 Number Analyzed 27 participants 9 participants
26.4  (9.34) 30.8  (7.55)
2.Primary Outcome
Title Number of Participants Experiencing an Adverse Event During the Treatment Period
Hide Description Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events.
Time Frame by the end of the treatment period (within 36 Weeks)
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Safety Population
Arm/Group Title Arm A: RTP Acthar Gel Arm B: RTP Placebo
Hide Arm/Group Description:

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Overall Number of Participants Analyzed 95 47
Measure Type: Count of Participants
Unit of Measure: Participants
Serious Treatment Emergent Adverse Events
13
  13.7%
6
  12.8%
Non-serious Treatment-Emergent Adverse Events
74
  77.9%
40
  85.1%
Death
2
   2.1%
3
   6.4%
3.Primary Outcome
Title Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period
Hide Description Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events.
Time Frame by the time of database lock (within 84 weeks)
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Safety Population
Arm/Group Title Arm C: OLE Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Hide Arm/Group Description:
Participants who receive Acthar Gel during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Overall Number of Participants Analyzed 25 8
Measure Type: Count of Participants
Unit of Measure: Participants
Serious Treatment Emergent Adverse Events
4
  16.0%
2
  25.0%
Non-serious Treatment-Emergent Adverse Events
20
  80.0%
7
  87.5%
4.Secondary Outcome
Title Treatment Period: Spirometry (%)
Hide Description Spirometry (meaning the measuring of breath) is the most common of the lung function tests. It measures how much air can be inhaled [Forced Vital Capacity (FVC)] and exhaled [(Forced Expiratory Volume in one second (FEV1)].
Time Frame Baseline, Week 36
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Hide Analysis Population Description
Safety population
Arm/Group Title Arm A: RTP Acthar Gel Arm B: RTP Placebo
Hide Arm/Group Description:

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Overall Number of Participants Analyzed 95 47
Mean (Standard Deviation)
Unit of Measure: liters
FVC at Baseline Number Analyzed 95 participants 47 participants
85.0  (18.31) 82.1  (17.60)
FVC at Week 36 Number Analyzed 27 participants 8 participants
61.4  (27.37) 63.8  (26.05)
FEV1 at Baseline Number Analyzed 95 participants 47 participants
79.3  (18.78) 77.8  (18.52)
FEV1 at Week 36 Number Analyzed 27 participants 8 participants
59.1  (27.37) 54.3  (26.79)
5.Secondary Outcome
Title Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R
Hide Description

The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory).

The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.

Time Frame Baseline, Week 36
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Hide Analysis Population Description
Safety Population, with an actual score at the given time point
Arm/Group Title Arm A: RTP Acthar Gel Arm B: RTP Placebo
Hide Arm/Group Description:

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Overall Number of Participants Analyzed 95 47
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline Number Analyzed 93 participants 45 participants
35.8  (6.05) 35.4  (6.23)
Week 36 Number Analyzed 29 participants 9 participants
28.0  (9.50) 30.9  (7.18)
6.Secondary Outcome
Title Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R
Hide Description

The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory).

The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.

Time Frame Baseline, Week 84
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Hide Analysis Population Description
OLE population with scores at the given week; baseline is defined as the value at randomization (week 0)
Arm/Group Title Arm C: OLE Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Hide Arm/Group Description:
Participants who receive Acthar Gel during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Overall Number of Participants Analyzed 25 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 25 participants 7 participants
36.8  (5.33) 39.6  (1.81)
Week 84 Number Analyzed 5 participants 2 participants
27.4  (5.41) 21.5  (4.95)
Time Frame From the start to the end of the period; RTP is 36 weeks (plus 3 weeks if ending there), OLE is up to 21 months (up to Week 84)
Adverse Event Reporting Description Treatment-emergent adverse events (TEAEs) were collected. A TEAE is defined as an adverse event that starts or worsens on or after first dose of study drug and within 28 days from the last dose date of the study drug (For subjects entered into OLE, any TEAE started prior to first OLE dose is counted in RTP). For each system organ class and preferred term, subjects are counted only once, even if they experienced multiple events in that system organ class or preferred term.
 
Arm/Group Title Arm A: RTP Acthar Gel Arm B: RTP Placebo Arm C: OLE Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Hide Arm/Group Description

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Participants who receive Acthar Gel during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
All-Cause Mortality
Arm A: RTP Acthar Gel Arm B: RTP Placebo Arm C: OLE Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/95 (2.11%)      3/47 (6.38%)      1/25 (4.00%)      2/8 (25.00%)    
Hide Serious Adverse Events
Arm A: RTP Acthar Gel Arm B: RTP Placebo Arm C: OLE Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/95 (13.68%)      6/47 (12.77%)      4/25 (16.00%)      2/8 (25.00%)    
Gastrointestinal disorders         
Constipation  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
General disorders         
Gait disturbance  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Infections and infestations         
Pneumonia  1  6/95 (6.32%)  6 0/47 (0.00%)  0 1/25 (4.00%)  1 1/8 (12.50%)  1
Cellulitis staphylococcal  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Empyema  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Gastroenteritis  1  0/95 (0.00%)  0 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Pyelonephritis acute  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Respiratory tract infection  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Urosepsis  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Injury, poisoning and procedural complications         
Procedural pain  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Rib fracture  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Fall  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 0/8 (0.00%)  0
Femur fracture  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 0/8 (0.00%)  0
Metabolism and nutrition disorders         
Dehydration  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Failure to thrive  1  0/95 (0.00%)  0 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Nervous system disorders         
Amyotrophic lateral sclerosis  1  0/95 (0.00%)  0 1/47 (2.13%)  1 1/25 (4.00%)  1 1/8 (12.50%)  1
Cerebellar infarction  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Psychiatric disorders         
Anxiety  1  0/95 (0.00%)  0 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Depression  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Mental status changes  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Suicidal ideation  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 0/8 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Acute respiratory failure  1  2/95 (2.11%)  2 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Respiratory failure  1  2/95 (2.11%)  2 1/47 (2.13%)  1 1/25 (4.00%)  1 0/8 (0.00%)  0
Pneumonia aspiration  1  1/95 (1.05%)  1 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Pulmonary embolism  1  2/95 (2.11%)  2 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Respiratory arrest  1  1/95 (1.05%)  1 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Dyspnoea  1  0/95 (0.00%)  0 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Haemothorax  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Pleural effusion  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Vascular disorders         
Deep vein thrombosis  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Hypertensive emergency  1  1/95 (1.05%)  1 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
1
Term from vocabulary, MedDRA (19.1)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Arm A: RTP Acthar Gel Arm B: RTP Placebo Arm C: OLE Acthar Gel-Acthar Gel Arm D: OLE Placebo-Acthar Gel
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   71/95 (74.74%)      34/47 (72.34%)      18/25 (72.00%)      7/8 (87.50%)    
Cardiac disorders         
Tachycardia  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
Ear and labyrinth disorders         
Excessive cerumen production  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Gastrointestinal disorders         
Constipation  1  13/95 (13.68%)  15 4/47 (8.51%)  5 2/25 (8.00%)  3 2/8 (25.00%)  2
Dry Mouth  1  5/95 (5.26%)  5 0/47 (0.00%)  0 1/25 (4.00%)  1 1/8 (12.50%)  1
Dysphagia  1  5/95 (5.26%)  5 5/47 (10.64%)  5 0/25 (0.00%)  0 1/8 (12.50%)  1
Nausea  1  5/95 (5.26%)  6 2/47 (4.26%)  2 2/25 (8.00%)  2 1/8 (12.50%)  1
Salivary hypersecretion  1  5/95 (5.26%)  6 1/47 (2.13%)  1 0/25 (0.00%)  0 1/8 (12.50%)  1
Diarrhoea  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 1/8 (12.50%)  1
Gastrooesophageal reflux disease  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 2/8 (25.00%)  2
Inflammatory bowel disease  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Vomiting  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
General disorders         
Asthenia  1  6/95 (6.32%)  7 4/47 (8.51%)  5 1/25 (4.00%)  1 1/8 (12.50%)  1
Fatigue  1  13/95 (13.68%)  14 6/47 (12.77%)  8 3/25 (12.00%)  3 1/8 (12.50%)  1
Injection site bruising  1  13/95 (13.68%)  16 4/47 (8.51%)  6 0/25 (0.00%)  0 0/8 (0.00%)  0
Oedema  1  5/95 (5.26%)  5 1/47 (2.13%)  1 0/25 (0.00%)  0 0/8 (0.00%)  0
Oedema peripheral  1  17/95 (17.89%)  24 2/47 (4.26%)  2 2/25 (8.00%)  2 2/8 (25.00%)  2
Chest pain  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Pain  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 1/8 (12.50%)  1
Infections and infestations         
Gastroenteritis viral  1  2/95 (2.11%)  2 3/47 (6.38%)  3 0/25 (0.00%)  0 0/8 (0.00%)  0
Urinary tract infection  1  8/95 (8.42%)  12 2/47 (4.26%)  2 4/25 (16.00%)  6 0/8 (0.00%)  0
Bronchitis  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Nasopharyngitis  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
Oral candidiasis  1  0/95 (0.00%)  0 0/47 (0.00%)  0 3/25 (12.00%)  4 0/8 (0.00%)  0
Sinusitis  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 1/8 (12.50%)  1
Injury, poisoning and procedural complications         
Contusion  1  9/95 (9.47%)  14 2/47 (4.26%)  4 2/25 (8.00%)  2 0/8 (0.00%)  0
Fall  1  18/95 (18.95%)  28 14/47 (29.79%)  33 6/25 (24.00%)  11 1/8 (12.50%)  1
Laceration  1  1/95 (1.05%)  1 3/47 (6.38%)  3 0/25 (0.00%)  0 0/8 (0.00%)  0
Limb injury  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Investigations         
Blood pressure increased  1  5/95 (5.26%)  7 1/47 (2.13%)  1 0/25 (0.00%)  0 1/8 (12.50%)  1
Weight decreased  1  3/95 (3.16%)  3 5/47 (10.64%)  6 1/25 (4.00%)  1 1/8 (12.50%)  1
Metabolism and nutrition disorders         
Increased appetite  1  5/95 (5.26%)  6 0/47 (0.00%)  0 0/25 (0.00%)  0 0/8 (0.00%)  0
Abnormal loss of weight  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Decreased appetite  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
Diabetes mellitus  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Musculoskeletal and connective tissue disorders         
Arthralgia  1  8/95 (8.42%)  16 4/47 (8.51%)  8 0/25 (0.00%)  0 1/8 (12.50%)  1
Muscle atrophy  1  2/95 (2.11%)  3 3/47 (6.38%)  5 3/25 (12.00%)  3 0/8 (0.00%)  0
Muscle spasms  1  15/95 (15.79%)  16 3/47 (6.38%)  3 2/25 (8.00%)  3 0/8 (0.00%)  0
Muscular weakness  1  22/95 (23.16%)  35 9/47 (19.15%)  14 3/25 (12.00%)  3 2/8 (25.00%)  2
Musculoskeletal pain  1  4/95 (4.21%)  6 7/47 (14.89%)  7 0/25 (0.00%)  0 0/8 (0.00%)  0
Neck Pain  1  5/95 (5.26%)  5 1/47 (2.13%)  1 0/25 (0.00%)  0 1/8 (12.50%)  1
Pain in extremity  1  3/95 (3.16%)  3 3/47 (6.38%)  4 1/25 (4.00%)  1 2/8 (25.00%)  2
Back pain  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 2/8 (25.00%)  2
Bursitis  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Mobility decreased  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Nervous system disorders         
Dizziness  1  7/95 (7.37%)  7 3/47 (6.38%)  3 0/25 (0.00%)  0 1/8 (12.50%)  1
Dysarthria  1  9/95 (9.47%)  12 4/47 (8.51%)  5 0/25 (0.00%)  0 0/8 (0.00%)  0
Headache  1  11/95 (11.58%)  14 1/47 (2.13%)  6 1/25 (4.00%)  1 2/8 (25.00%)  2
Muscle contractions involuntary  1  3/95 (3.16%)  3 3/47 (6.38%)  3 2/25 (8.00%)  2 0/8 (0.00%)  0
Tremor  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
Psychiatric disorders         
Anxiety  1  6/95 (6.32%)  6 2/47 (4.26%)  3 1/25 (4.00%)  1 1/8 (12.50%)  1
Depression  1  3/95 (3.16%)  4 3/47 (6.38%)  3 0/25 (0.00%)  0 0/8 (0.00%)  0
Insomnia  1  7/95 (7.37%)  7 1/47 (2.13%)  1 2/25 (8.00%)  3 2/8 (25.00%)  2
Renal and urinary disorders         
Dysuria  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Respiratory, thoracic and mediastinal disorders         
Dyspnoea  1  9/95 (9.47%)  10 6/47 (12.77%)  8 1/25 (4.00%)  1 2/8 (25.00%)  2
Chronic respiratory failure  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Cough  1  0/95 (0.00%)  0 0/47 (0.00%)  0 3/25 (12.00%)  3 2/8 (25.00%)  2
Dyspnoea exertional  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Increased viscosity of upper respiratory secretion  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
Respiratory disorder  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Skin and subcutaneous tissue disorders         
Ecchymosis  1  5/95 (5.26%)  5 2/47 (4.26%)  3 0/25 (0.00%)  0 0/8 (0.00%)  0
Blister  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Rash  1  0/95 (0.00%)  0 0/47 (0.00%)  0 1/25 (4.00%)  1 1/8 (12.50%)  1
Skin discolouration  1  0/95 (0.00%)  0 0/47 (0.00%)  0 0/25 (0.00%)  0 1/8 (12.50%)  1
Vascular disorders         
Hypertension  1  8/95 (8.42%)  10 1/47 (2.13%)  1 1/25 (4.00%)  1 1/8 (12.50%)  1
Hot flush  1  0/95 (0.00%)  0 0/47 (0.00%)  0 2/25 (8.00%)  2 0/8 (0.00%)  0
1
Term from vocabulary, MedDRA (19.1)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Information Call Center
Organization: Mallinckrodt
Phone: 800-556-3314 ext 5
EMail: clinicaltrials@mnk.com
Layout table for additonal information
Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT03068754    
Other Study ID Numbers: MNK14042068
First Submitted: February 23, 2017
First Posted: March 3, 2017
Results First Submitted: August 14, 2020
Results First Posted: September 1, 2020
Last Update Posted: September 3, 2020