Trial Outcomes & Findings for Combined Treatment of Methotrexate + Glucocorticoids Versus Glucocorticoids Alone in Patients With PM and DM (NCT NCT00651040)

NCT ID: NCT00651040

Last Updated: 2016-05-12

Results Overview

The primary endpoint which has benn measured was the total dose of glucocorticoids administered between baseline and the end of treatment.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

31 participants

Primary outcome timeframe

1 year

Results posted on

2016-05-12

Participant Flow

Trial has reached 62% of planned recruitment (31 patients randomized).

Participant milestones

Participant milestones
Measure
1Prednison
Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms. ARM 1 has only Prednisone Prednisone: Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms
2Prednison MTX
MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose. Methotrexate: MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.
Overall Study
STARTED
16
15
Overall Study
COMPLETED
13
14
Overall Study
NOT COMPLETED
3
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Combined Treatment of Methotrexate + Glucocorticoids Versus Glucocorticoids Alone in Patients With PM and DM

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
1Prednison
n=16 Participants
Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms. ARM 1 has only Prednisone Prednisone: Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms
2Prednison MTX
n=15 Participants
MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose. Methotrexate: MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.
Total
n=31 Participants
Total of all reporting groups
Age, Continuous
51.6 years
STANDARD_DEVIATION 14.4 • n=93 Participants
53.5 years
STANDARD_DEVIATION 17.4 • n=4 Participants
52.6 years
STANDARD_DEVIATION 15.7 • n=27 Participants
Sex: Female, Male
Female
10 Participants
n=93 Participants
10 Participants
n=4 Participants
20 Participants
n=27 Participants
Sex: Female, Male
Male
6 Participants
n=93 Participants
5 Participants
n=4 Participants
11 Participants
n=27 Participants
Region of Enrollment
Czech Republic
13 participants
n=93 Participants
8 participants
n=4 Participants
21 participants
n=27 Participants
Region of Enrollment
Sweden
3 participants
n=93 Participants
1 participants
n=4 Participants
4 participants
n=27 Participants
Region of Enrollment
Switzerland
0 participants
n=93 Participants
4 participants
n=4 Participants
4 participants
n=27 Participants
Region of Enrollment
Hungary
0 participants
n=93 Participants
1 participants
n=4 Participants
1 participants
n=27 Participants
Region of Enrollment
Poland
0 participants
n=93 Participants
1 participants
n=4 Participants
1 participants
n=27 Participants

PRIMARY outcome

Timeframe: 1 year

The primary endpoint which has benn measured was the total dose of glucocorticoids administered between baseline and the end of treatment.

Outcome measures

Outcome measures
Measure
Prednison1
n=16 Participants
Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms. ARM 1 has only Prednisone Prednisone: Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms
Prednison Methotrexate 2
n=15 Participants
MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose. Methotrexate: MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.
The Primary Endpoint is the Total Dose of Glucocorticoids Administered Between Baseline and the End of Treatment.
124 mg/kg
Standard Deviation 65.7
135 mg/kg
Standard Deviation 45.2

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome data not reported

Adverse Events

1 Prednison

Serious events: 4 serious events
Other events: 14 other events
Deaths: 0 deaths

2 Prednison Methotrexate

Serious events: 5 serious events
Other events: 11 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
1 Prednison
n=16 participants at risk
Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms. ARM 1 has only Prednisone Prednisone: Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms
2 Prednison Methotrexate
n=15 participants at risk
MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose. Methotrexate: MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
tumor
12.5%
2/16 • 1 year
20.0%
3/15 • 1 year
Musculoskeletal and connective tissue disorders
worsening of muscles
12.5%
2/16 • 1 year
0.00%
0/15 • 1 year
Skin and subcutaneous tissue disorders
worsening of skin
0.00%
0/16 • 1 year
6.7%
1/15 • 1 year
General disorders
synkopa
0.00%
0/16 • 1 year
6.7%
1/15 • 1 year

Other adverse events

Other adverse events
Measure
1 Prednison
n=16 participants at risk
Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms. ARM 1 has only Prednisone Prednisone: Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms
2 Prednison Methotrexate
n=15 participants at risk
MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose. Methotrexate: MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.
Infections and infestations
infection
50.0%
8/16 • 1 year
40.0%
6/15 • 1 year
Musculoskeletal and connective tissue disorders
Musculoskeletal
50.0%
8/16 • 1 year
20.0%
3/15 • 1 year
Cardiac disorders
cardiovascular
12.5%
2/16 • 1 year
13.3%
2/15 • 1 year
Gastrointestinal disorders
gastrointestinal
12.5%
2/16 • 1 year
20.0%
3/15 • 1 year
General disorders
general
18.8%
3/16 • 1 year
20.0%
3/15 • 1 year
Endocrine disorders
endocrine
12.5%
2/16 • 1 year
0.00%
0/15 • 1 year
Nervous system disorders
neurological
18.8%
3/16 • 1 year
0.00%
0/15 • 1 year
Psychiatric disorders
psychiatric
6.2%
1/16 • 1 year
0.00%
0/15 • 1 year
Reproductive system and breast disorders
gynecological
0.00%
0/16 • 1 year
6.7%
1/15 • 1 year

Additional Information

Rheumatology Institute Prague

Rheumatology Institute Prague

Phone: +420234075111

Results disclosure agreements

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