Hereditary Autoinflammatory Diseases

qualitative study

CH André Mignot

Dr Maryam PIRAM

TENON

Quality of Life in Patients With Auto-inflammatory Diseases

Creation of a Tool to Assess Quality of Life in Patient With Auto-inflammatory Diseases

The interviews will be recorded in digital audio and then transcribed literally. The analysis will be phenomenological. Four age group will be interviewed : < 6 year-old (parents), 6 to 12 year-old (patients and parents) , 13 to 17 year-old (patients and parents) , > 18 year-old (patients)

Assistance Publique - Hôpitaux de Paris

The study is based on interviews that will be recorded in digital audio and then transcribed literally. The consultation for this research is 45 to 90 minutes long. This prolongs their presence in consultation with the doctor. For this reason, the study was classified as Interventional according to French regulations

During Epoch 2, participants received canakinumab 150mg (or 2mg/kg for participants weighing <= 40kg) q4w for 16 weeks. If participants were eligible for blinded escape, they received a single add-on dose of canakinumab (150 mg or 2mg/kg for participants weighing <= 40kg) between day 8 and day 28, and then received blinded uptitration to canakinumab 300 mg q4w from day 29 through day 112. If patients on the highest allowed canakinumab dose of 300 mg (or 4 mg/kg for patients weighing ≤ 40 kg) q4w and re-flared (PGA ≥ 2 and CRP ≥ 30 mg/L) were not eligible for further up-titration.

During epoch 2, participants received matching placebo to canakinumab 150 mg Participants who required blinded escape,they received a single add-on dose of canakinumab (150 mg or 2mg/kg for participants weighing <= 40kg) between day 8 and day 28, and then received blinded one dose of placebo and one dose of canakinumab q4w from day 29 through day 112. If flare or re-flare still occurred after receipt of canakinumab.
150mg, participants were uptitrated to open-label canakinumab 300 mg.

During epoch 2, participants received matching placebo to canakinumab 150 mg qw4. Participants who required blinded escape, they received a single add-on dose of canakinumab (150 mg or 2mg/kg for participants weighing <= 40kg) between day 8 and day 28 and then received blinded one dose of placebo and one dose of canakinumab q4w from day 29 through day 112. If flare or re-flare still occurred after receipt of canakinumab 150mg, participants were uptitrated to open-label canakinumab 300 mg.

During Epoch 2, participants received canakinumab 150mg (or 2mg/kg for participants weighing <= 40kg) q4w for 16 weeks. If participants were eligible for blinded escape, they received a single add-on dose of canakinumab (150 mg or 2mg/kg for participants weighing <= 40kg) between day 8 and day 28, and then received blinded uptitration to canakinumab 300 mg q4w from day 29 through day 112. If patients on the highest allowed canakinumab dose of 300 mg (or 4 mg/kg for patients weighing ≤ 40 kg) q4w and re-flared (PGA ≥ 2 and CRP ≥ 30 mg/L) were not eligible for further up-titration

During epoch 2, participants received matching placebo to canakinumab 150 mg qw4. Participants who required blinded escape,they received a single add-on dose of canakinumab (150 mg or 2mg/kg for participants weighing <= 40kg) between Day 8 and 28 and then received blinded one dose of placebo and one dose of canakinumab q4w from day 29 through day 112. If flare or re-flare still occurred after receipt of canakinumab 150mg, participants were uptitrated to open-label canakinumab 300 mg.

Epoch 2: crFMF: 150 mg

During epoch 2, participants received matching placebo to canakinumab 150 mg Participants who required blinded escape,they received a single add-on dose of canakinumab (150 mg or 2mg/kg for participants weighing <= 40kg) between day 8 and day 28, and then received blinded one dose of placebo and one dose of canakinumab q4w from day 29 through day 112. If flare or re-flare still occurred after receipt of canakinumab.
150mg, participants were uptitrated to open-label canakinumab 300 mg

Epoch 2: crCMF: Placebo

Epoch 2: HIDS/MKD: 150 mg

Epoch 2: HIDS/MKD: Placebo

Epoch 2: TRAPS: 150 mg

Epoch 2: TRAPS: Placebo

Canakinumab-naïve Japanese patients with non-exon 10 mutations received open-label canakinumab 150 mg (or 2 mg/kg for patients weighing
≤ 40 kg) q4w

Epoch 2: Non-randomized Open Label Treatment - crFMF

Participants in the 28 days to less than 2 years old cohort who received open-label canakinumab 150 mg (or 2mg/kg for patients weighing
≤ 40 kg) q4w.

Epoch 2: Non-randomized Open Label HIDS/MKD

Open-label treatment in Epoch 3 was initiated for TRAPS patients who rolled over from CACZ885D2203 or CACZ885D2207M.

Epoch 2 (Epoch 3) - Non-randomized Open Label TRAPS

Total

Sex: Female, Male

Race (NIH/OMB)

Resolution of the initial disease flare is defined as: Physician's Global Assessment of Disease activity (PGA) <2 and C-reactive protein (CRP) within normal range (<= 10 mg/L) or reduction by at least 70% from baseline. The PGA was evaluated by the investigator based on a 5-point scale: 0 = None (no) disease associated with clinical signs and symptoms; 1 = minimal disease associated signs and symptoms; 2 = mild disease associated signs and symptoms; 3 = moderate disease associated signs and symptoms; and 5 = severe disease associated signs and symptoms.

Percentage of Participants With Resolution of Initial Flare and Absence of New Flares up to the End of the Randomized Treatment Epoch (16 Weeks)

The PGA was evaluated by the investigator based on a 5-point scale: 0 = None (no) disease associated with clinical signs and symptoms; 1 = minimal disease associated signs and symptoms; 2 = mild disease associated signs and symptoms; 3 = moderate disease associated signs and symptoms; and 5 = severe disease associated signs and symptoms.

Percentage of Participants Who Achieve Physician's Global Assessment (PGA) < 2

Serologic remission was defined as C-reactive protein <= 10 mg/L.

Percentage of Participants With the Serologic Remission

Normalized SAA was defined as SAA <= 10 mg/L.

Percentage of Participants With Normalized Serum Amyloid A (SAA) Level

A responder was defined as a participant who had no flare between week 16 and week 40.

Percentage of Participants of Canakinumab Responders From Epoch 2 Who Maintained a Clinically Meaningful Response (Absence of New Flares) (40 Weeks)

Canakinumab-naïve Japanese patients with non-exon 10 mutations received open-label canakinumab 150 mg (or 2 mg/kg for patients weighing
≤ 40 kg) q4w .

Canakinumab responders who were initially randomized to canakinumab 150 mg q4w and did not re-flare in Epoch 2 were re-randomized at the start of Epoch 3 to canakinumab 150 mg q8w for 24 weeks.

Epoch 3: crFMF Re-randomized From Epoch 2 - 150 mg

Canakinumab responders who were initially randomized to canakinumab 150 mg q4w and did not re-flare in Epoch 2 were re-randomized at the start of Epoch 3 to placebo for 24 weeks.

Epoch 3: crFMF Re-randomized From Epoch 2 - Placebo

Epoch 3: HIDs/MKD Re-randomized From Epoch 2 - 150 mg

Epoch 3: HIDS/MKD Re-randomized From Epoch 2 - Placebo

Epoch 3: TRAPS Re-randomized From Epoch 2 - 150 mg

Epoch 3: TRAPS Re-randomized From Epoch 2 - Placebo

All Epoch 2 non-responders were switched to canakinumab q8w at the start of Epoch 3 for 24 weeks,

Epoch 3: crFMF, HIDS/MKD, TRAPS - Not Re-randomized

During epoch 4, participants received open label treatment according to the dose regimen administered in epoch 3. Cumulative dose received was less than 2700 mg.

Epoch 4: crFMF - Open Label Cumulative Dose <2700 mg

During epoch 4, participants received open label treatment according to the dose regimen administered in epoch 3. Cumulative dose received was >= 2700 mg and < 5400 mg.

Epoch 4: crFMF - Open Label Cumulative Dose 2700 mg - <5400 mg

During epoch 4, participants received open label treatment according to the dose regimen administered in epoch 3. Cumulative dose received was > 5400 mg.

Epoch 4: crFMF - Open Label Cumulative Dose >=5400 mg

Epoch 4: HIDS/MKD - Open Label Cumulative Dose <2700 mg

Epoch 4: HIDS/MKD - OL Cumulative Dose 2700 - <=5400 mg

Epoch 4: HIDS/MKD - Open Label Cumulative Dose >=5400 mg

Epoch 4: TRAPS - Open Label Cumulative Dose < 2700 mg

Epoch 4: TRAPS - Open Label Cumulative Dose 2700 - <5400 mg

Epoch 4: TRAPS - Open Label Cumulative Dose >=5400 mg

Epoch 2

Epoch 3

Epoch 4

Study Director

Canakinumab-naïve Japanese patients with non-exon 10 mutations with cr-FMF who received open-label canakinumab 150 mg (or 2 mg/kg for patients weighing ≤ 40 kg) q4w; and patients in the 28 days to less than 2 years old cohort with HIDS/MKD who received open-label canakinumab 150 mg (or 2mg/kg for patients weighing
≤ 40 kg) q4w

Non-randomized Open Label crFMF, HIDS/MKD Patients

Open-label treatment in Epoch 3 was initiated for TRAPS patients who rolled over from CACZ885D2203 or CACZ885D2207M

Non-randomized Open Label TRAPS Patients

Patients who received ACZ885 and/or placebo during epoch 2 and/or epoch 3.

Randomized ACZ and Placebo TRAPS Patients - Placebo Events

Patients who received ACZ885 and/or placebo during epoch 2 and/or epoch 3 .

Randomized ACZ and Placebo TRAPS Pts - No Medication Events

Patients who received ACZ885 and/or placebo during epoch 2 and/or epoch 3

Randomized ACZ and Placebo TRAPS Patients - ACZ Events

Randomized ACZ and Placebo HIDS/MKD Pts - Placebo Events

Randomized ACZ and Placebo HIDS/MKD Pts - No Medication Events

Randomized ACZ and Placebo HIDS/MKD Patients - ACZ Events

Randomized ACZ and Placebo crFMF Patients - Placebo Events

Randomized ACZ and Placebo crFMF Pts - No Medication Events

Randomized ACZ and Placebo crFMF Patients - ACZ Events

Patients who received ACZ885 during epoch 2 and/or epoch 3

Any ACZ TRAPS Patients - Placebo Events

Any ACZ TRAPS Patients - no Medication Events

Any ACZ TRAPS Patients - ACZ Events

Any ACZ HIDS/MKD Patients - Placebo Events

Any ACZ HIDS/MKD Patients - No Medication Events

Any ACZ HIDS/MKD Patients - ACZ Events

Any ACZ crFMF Patients - Placebo Events

Any ACZ crFMF Patients - No Medication Events

Any ACZ crFMF Patients - ACZ Events

Hereditary Periodic Fevers

Canakinumab solution for subcutaneous injection in vial which contained 150mg/mL canakinumab in 1 mL solution.

Canakinumab

Matching placebo to canakinumab solution for subcutaneous injection

Placebo

Novartis Investigative Site

Study of Efficacy and Safety of Canakinumab in Patients With Hereditary Periodic Fevers

A Randomized, Double-blind, Placebo Controlled Study of Canakinumab in Patients With Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), With Subsequent Randomized Withdrawal/Dosing Frequency Reduction and Open-label Long-term Treatment Epochs

Novartis Pharmaceuticals

blood samples, cells (fibroblasts) from skin biopsy tissue, DNA, urine and stool

mevalonate kinase deficiency/حمى

Quality of Life in Patients With Auto-inflammatory Diseases

Study of Efficacy and Safety of Canakinumab in Patients With Hereditary Periodic Fevers

Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase Deficiency

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