Trial Outcomes & Findings for Relevance of Monitoring Blood and Salivar Levels of Drugs Used in Rheumatic Autoimmune Diseases (NCT NCT03122431)

NCT ID: NCT03122431

Last Updated: 2021-12-16

Results Overview

Serum levels of thalidomide by liquid chromatography and tandem mass spectrometry (HPLC-MS/MS)

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

93 participants

Primary outcome timeframe

12 months

Results posted on

2021-12-16

Participant Flow

Participant milestones

Participant milestones
Measure
Inactive SLE With Standard Dose of HCQ
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed full 2016-AAO dose of HCQ (4-5.5 mg/kg/day, real body weight).
Inactive SLE With Reduced Dose of HCQ
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed reduced 2016-AAO dose of HCQ (2-3.0 mg/kg/day, real body weight)
SLE/Cutaneous Lupus With Thalidomide
Adult patients with CLE (biopsy proven)/SLE (American College of Rheumatology classification criteria, 1997) treated with thalidomide. Inclusion criteria: 1. absence of pregnancy potential for females; 2. agreement of male patients to use condoms throughout the treatment period with thalidomide and up to 30 days after its ending, even though they had already undergone vasectomy; 3. active skin lesions; 4. no response to previous use of HCQ, prednisone 20 mg/day, and immunosuppressants/dapsone or indication of thalidomide according to the attending physician; 5) agreement of recruited patients to participate in the study according to signed informed consent form, and agreement to thalidomide use according to signed responsibility term at starting treatment and at each renewal of the prescription; 6) a normal NCS; 7\) normal serum vitamin B12 levels; 8) negative tests for hepatitis B/C and HIV; 9) negative antiphospholipid antibodies. Exclusion criteria: 1. other associated autoimmune diseases, such as Sjogren's syndrome; 2. previous or current alcoholism; 3. diabetes mellitus; 4. history of PN; 5. previous use of thalidomide; 6. previous or current use of leflunomide, TNF-alpha inhibitors, chemotherapy and other neurotoxic drugs; 7. history of cancer or thrombosis; 8. current renal or nervous system lupus activities, autoimmune haemolytic anaemia and/or thrombocytopenia \<50,000/mm3; 9. inability to understand the study.
Overall Study
STARTED
41
32
20
Overall Study
COMPLETED
41
32
9
Overall Study
NOT COMPLETED
0
0
11

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Baseline hydroxychloroquine levels in this arm were not measured.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Inactive SLE With Standard Dose of HCQ
n=41 Participants
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed full 2016-AAO dose of HCQ (4-5.5 mg/kg/day, real body weight).
Inactive SLE With Reduced Dose of HCQ
n=32 Participants
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed reduced 2016-AAO dose of HCQ (2-3.0 mg/kg/day, real body weight).
SLE/Cutaneous Lupus With Thalidomide
n=20 Participants
This subproject includes only one arm of lupus patients with active and refractory cutaneous disease and eligible for Thalidomide 100mg/day for 12 months.
Total
n=93 Participants
Total of all reporting groups
Age, Continuous
37 years
STANDARD_DEVIATION 10.5 • n=41 Participants
37 years
STANDARD_DEVIATION 6.9 • n=32 Participants
44.8 years
STANDARD_DEVIATION 7.6 • n=20 Participants
37 years
STANDARD_DEVIATION 8.7 • n=93 Participants
Sex: Female, Male
Female
39 Participants
n=41 Participants
29 Participants
n=32 Participants
17 Participants
n=20 Participants
85 Participants
n=93 Participants
Sex: Female, Male
Male
2 Participants
n=41 Participants
3 Participants
n=32 Participants
3 Participants
n=20 Participants
8 Participants
n=93 Participants
Race/Ethnicity, Customized
Caucasian
13 Participants
n=41 Participants
6 Participants
n=32 Participants
9 Participants
n=20 Participants
28 Participants
n=93 Participants
Race/Ethnicity, Customized
Non-Caucasian
28 Participants
n=41 Participants
26 Participants
n=32 Participants
11 Participants
n=20 Participants
65 Participants
n=93 Participants
Drug blood levels
1343.5 ng/mL
STANDARD_DEVIATION 521.5 • n=41 Participants • Baseline hydroxychloroquine levels in this arm were not measured.
1404.9 ng/mL
STANDARD_DEVIATION 492.0 • n=32 Participants • Baseline hydroxychloroquine levels in this arm were not measured.
1374.2 ng/mL
STANDARD_DEVIATION 506.8 • n=73 Participants • Baseline hydroxychloroquine levels in this arm were not measured.

PRIMARY outcome

Timeframe: 12 months

Serum levels of thalidomide by liquid chromatography and tandem mass spectrometry (HPLC-MS/MS)

Outcome measures

Outcome measures
Measure
SLE/Cutaneous Lupus With Thalidomide
n=20 Participants
Adult patients with CLE (biopsy proven)/SLE (American College of Rheumatology classification criteria, 1997) treated with thalidomide.
Inactive SLE With Reduced Dose of HCQ
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed reduced 2016-AAO dose of HCQ (2-3.0 mg/kg/day, real body weight)
Serum Levels of Thalidomide
415.1 ng/mL
Standard Deviation 326.0

PRIMARY outcome

Timeframe: 12 months

Serum levels of hydroxycloroquine by LCMS

Outcome measures

Outcome measures
Measure
SLE/Cutaneous Lupus With Thalidomide
n=41 Participants
Adult patients with CLE (biopsy proven)/SLE (American College of Rheumatology classification criteria, 1997) treated with thalidomide.
Inactive SLE With Reduced Dose of HCQ
n=32 Participants
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed reduced 2016-AAO dose of HCQ (2-3.0 mg/kg/day, real body weight)
Serum Levels of Hydroxycloroquine
991.6 ng/mL
Standard Deviation 576.3
569.0 ng/mL
Standard Deviation 533.4

Adverse Events

SLE/Cutaneous Lupus With Thalidomide

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Inactive SLE With Standard Dose of HCQ

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

Inactive SLE With Reduced Dose of HCQ

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

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
SLE/Cutaneous Lupus With Thalidomide
n=20 participants at risk
SLE/cutaneous lupus with thalidomide arm (12 months).
Inactive SLE With Standard Dose of HCQ
n=41 participants at risk
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed full 2016-AAO dose of HCQ (4-5.5 mg/kg/day, real body weight).
Inactive SLE With Reduced Dose of HCQ
n=32 participants at risk
Lupus nephritis patients on stable inactive disease for at least 6 months prescribed reduced 2016-AAO dose of HCQ (2-3.0 mg/kg/day, real body weight).
Nervous system disorders
Only sensory peripheral neuropathy
60.0%
12/20 • Number of events 12 • 12 months
0.00%
0/41 • 12 months
0.00%
0/32 • 12 months

Additional Information

Prof Eloisa Bonfa

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

Phone: 551130617490

Results disclosure agreements

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