Open-label Trial of IVIG in Children With PANS (NCT04609761) | Clinical Trial Compass
CompletedPhase 2
Open-label Trial of IVIG in Children With PANS
Sweden17 participantsStarted 2021-01-12
Plain-language summary
Open-label prospective trial to study efficacy, safety and tolerability of intravenous immunoglobulin (IVIG) once monthly for 6 months in children and adolescents with PANS. Number of subjects: 10. Age range: 4-17 years.
Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a recently defined research diagnosis describing an abrupt, dramatic onset of neuropsychiatric symptoms including obsessions/compulsions and/or food restriction in children. Immunologic mechanisms are suspected, but treatment trials are few.
Who can participate
Age range4 Years – 17 Years
SexALL
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Inclusion criteria
✓. The subject and parents/caregivers have given written consent or assent to participate in the study.
✓. Children and adolescents between the ages of 4 and 17 years at Baseline.
✓. Documented and confirmed pre-existing diagnosis of post-infectious PANS/PANDAS
✓. The subject has not been treated with IVIG previously or not been treated for the last 6 months
✓. If the patient is on long-term antibiotic prophylaxis, this should be unchanged one month before baseline and during the trial. Throat culture for Group A Streptococcus (GAS) should be performed before study start and standard phenoxymethyl penicillin treatment given if positive culture.
✓. Infections occurring during the trial should be treated according to standard clinical practice.
✓. Treatment with COX-inhibitors or corticosteroids should be discontinued at least one month before baseline and during the trial. Two-three days treatment with corticosteroids during and after IVIG treatment is allowed to reduce IVIG side effects such as headache and nausea.
✓. Any psychopharmacological treatment (e.g. SSRI, antipsychotics), if considered essential for the subject, should be kept at a stable and unchanged dose from one month before baseline and during the trial. If not considered essential, it should be discontinued at least one month before baseline.
Exclusion criteria
✕. Clinical evidence of any significant acute or chronic disease that, in the opinion of the Investigator, may interfere with successful completion of the trial or place the subject at undue medical risk. If encephalitis cannot be excluded by clinical history alone, spinal tap results are required before study start to rule out encephalitis (which would need to be treated according to encephalitis treatment guidelines). MRI should have been performed if clinically indicated.
What they're measuring
1
Change in Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) scale symptoms and impairment
✕. The subject has had a known serious adverse reaction to immunoglobulin or any severe anaphylactic reaction to blood or any blood-derived product
✕. Females of childbearing potential who are pregnant, have a positive pregnancy test at Baseline (human chorionic gonadotropin \[HCG\]-based assay), are breastfeeding, or unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device \[IUD\] or intrauterine system \[IUS\], condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence) throughout the study Note: True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence \[e.g., calendar, ovulation, symptothermal, post-ovulation methods\], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.)
✕. The subject has significant proteinuria (dipstick proteinuria ≥ 3+, known urinary protein loss \> 1 g/24 hours, or nephrotic syndrome), has a history of acute renal failure, has severe renal impairment (blood urea nitrogen \[BUN\] or creatinine more than 2.5 times the upper limit of normal \[ULN\]), and/or is on dialysis
✕. The subject has Screening Visit values of aspartate aminotransferase (AST) or alanine aminotransferase (ALT) levels exceeding 2.5 times the ULN for the expected normal range for the testing laboratory.
✕. The subject has hemoglobin \< 90 g/L at Screening
✕. The subject has a known previous infection with or clinical signs and symptoms consistent with current hepatitis B virus (HBV) or hepatitis C virus (HCV) infection
✕. The subject has a history of or current diagnosis of deep venous thrombosis or thromboembolism (e.g., myocardial infarction, cerebrovascular accident, or transient ischemic attack); history refers to an incident in the year prior to Baseline or 2 episodes over lifetime.