Trial Outcomes & Findings for Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis (NCT NCT03686969)

NCT ID: NCT03686969

Last Updated: 2021-04-23

Results Overview

MMT-8; a set of 8 designated muscles tested bilaterally \[potential score 0 - 150\]

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 1 participants
• Primary outcome timeframe: 32 weeks
• Results posted on: 2021-04-23

Participant Flow

Participant milestones

Measure	Octanorm 0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week	Placebo Placebo Placebo: Placebo
Overall Study STARTED	1	0
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	Octanorm 0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week	Placebo Placebo Placebo: Placebo
Overall Study Study terminated	1	0

Baseline Characteristics

Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis

Baseline characteristics by cohort

Measure	Octanorm n=1 Participants 0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week	Placebo Placebo Placebo: Placebo	Total n=1 Participants Total of all reporting groups
Age, Customized Age	52 years n=93 Participants	—	52 years n=27 Participants
Sex: Female, Male Female	1 Participants n=93 Participants	—	1 Participants n=27 Participants
Sex: Female, Male Male	0 Participants n=93 Participants	—	0 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	—	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	—	0 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	—	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	—	0 Participants n=27 Participants
Race (NIH/OMB) White	1 Participants n=93 Participants	—	1 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	—	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	—	0 Participants n=27 Participants

PRIMARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 - 150]

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

The CDASI is a clinician-scored single page instrument that separately measures activity and damage in the skin of DM patients for use in clinical practice or clinical/therapeutic studies.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Physician's Global Disease Activity (10 cm VAS assessing global disease activity from "No evidence of disease activity" to "Extremely active or severe disease activity"; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Extra-muscular activity (part of MDAAT; a combined tool that captures the physician's assessment of disease activity of various organ systems using a scale from 0 = "Not present in the last 4 weeks" to 4 = "New - in the last 4 weeks [compared to the previous 4 weeks]" and a VAS).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Measurement of aldolase in blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Measurement of creatine kinase in blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Measurement of alanine aminotransferase in blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Measurement of aspartate aminotransferase in blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Measurement of lactate dehydrogenase in blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

• Health Assessment Questionnaire (HAQ; a generic rather than a disease-specific instrument; comprised of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Total Improvement Score

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Time to clinically important deterioration

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Occurrence of all adverse events

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety with occurrence of all thromboembolic events (TEEs)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety with occurrence of all hemolytic transfusion reactions (HTRs)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety by assessing local injection site reactions

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through blood pressure values

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through heart rate values

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through body temperature values

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through respiratory rate values

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 Weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

The physical examination outcome will be analyzed based on changes from baseline as adverse events.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab sodium levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab potassium levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab glucose levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab ALAT levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab ASAT levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab LDH levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab total bilirubin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab blood urea nitrogen levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urea levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab creatinine levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab albumin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab hematocrit levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab hemoglobin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab red blood cell count levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab white blood cell count levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab platelet levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab serum haptoglobin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab plasma-free hemoglobin

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through Direct Coombs' test

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through D-dimers test

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab IgG levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab aldolase levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab creatine kinase levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through pregnancy test

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine protein levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine glucose levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine pH levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine nitrite levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine ketone levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine leukocyte levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine hemoglobin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine bilirubin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine urobilinogen levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through lab urine hemosiderin levels

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through HIV testing

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through hepatitis B testing

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 32 weeks

Population: The study was terminated prematurely. Due to the limited data available (only one patient enrolled and treated), efficacy and safety analyses were not performed.

Monitoring safety through hepatitis C testing

Outcome measures

Outcome data not reported

Adverse Events

Octanorm

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Octanorm n=1 participants at risk 0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week	Placebo Placebo Placebo: Placebo
Skin and subcutaneous tissue disorders Dermatomyositis	100.0% 1/1 • Number of events 2 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.	— 0/0 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.
General disorders Infusion site haematoma	100.0% 1/1 • Number of events 1 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.	— 0/0 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.
Investigations Blood lactate dehydrogenase increased	100.0% 1/1 • Number of events 1 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.	— 0/0 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.
Investigations Blood creatine phosphokinase increased	100.0% 1/1 • Number of events 1 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.	— 0/0 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.
Investigations Aldolase increased	100.0% 1/1 • Number of events 1 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.	— 0/0 • The patient was treated according to the protocol until week 14 Treatment after week 14 was discontinued because of early study termination Number of patients at risk for SAEs, All-Cause Mortality, and other adverse events in placebo group is 0 as 0 patients were enrolled into the placebo group.

Additional Information

Mikaela Raymond

CRMG

Phone: 866-337-1868

Email: ctgov@cllinicalresearchmgt.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place