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Study: (b)(6)-year-old male with mucopolysaccharidosis type iiib enrolled in: a (b)(6) study to evaluate the long-term safety and efficacy of intracerebroventricular ax 250 in patients with mucopolysaccharidosis type iiib (mps iiib, sanfilippo syndrome type b).Study treatment with ax 250 was initiated on (b)(6) 2018.The subject's most recent dose of ax 250, received prior to the sae and eosi onset, was on (b)(6) 2021.The subject's relevant medical and surgical history included recurrent bronchitis, recurrent ear, nose and throat (ent) infections, hearing impairment, speech delay, hepatomegaly, abnormal facial features, developmental delay, short neck, recurrent glue ear, splenomegaly and sleep disturbances.Relevant concomitant medications taken within 14 days of sae and eosi onset included cetirizine, paracetamol, nystatin, risperidone (risperdal) and cannabidiol.Previously reported sae/eosis for this subject included increased resistance during study drug infusion + slight swelling around rickham reservoir on (b)(6) 2018 ((b)(4)), device dysfunction on (b)(6) 2018 ((b)(4)), staphylococcus hominis in csf on (b)(6) 2018 ((b)(4)), extravasation of ip/csf on (b)(6) 2018 ((b)(4)), intracerebroventricular infection on (b)(6) 2019 ((b)(4)) and intracerebroventricular infection on (b)(6) 2019 (19-de-ax202-00037).On (b)(6) 2021, during the day, the subject felt weak and slept a lot.That evening, the subject began vomiting several times and mentioned headaches.The nausea and vomiting persisted and no diarrhea or fever was present.Due to worsening headache the subject attended the emergency room (er) at around 8 pm.At presentation the subject was clinically stable, awake and responsive.He presented with a headache, nausea and fever with a body temperature of 38.6 degrees celsius.His vital signs were stable.An intracerebroventricular (icv) puncture was performed and the puncture site was slightly reddish.The cerebrospinal fluid (csf) status showed an increased cell count of 1074/3 cells (normal range [nr] <12/3 cells), increased lactate 3.2 mmol/l (nr 0.5-2.2 mmol/l) and increased protein 356 mg/l (nr 80-320 mg/l).Laboratory tests also revealed mild leukocytosis, 15.9 mrd/l (nr 4.5-13.5 mrd/l).A csf culture was drawn which revealed staphylococcus epidermidis.The subject was hospitalized due to intracerebroventricular infection.The event was considered medically significant and was an adverse event of special interest.Treatment for the event included vancomycin, cefotaxime and paracetamol.Due to the subject¿s agitation during blood draws the subject received treatment with lorazepam.On (b)(6) 2021, the rickman catheter explantation occurred without complications.The subject received sufentanil, propofol and cisatracurium for anesthesia and ibuprofen, midazolam and sterofundin perioperatively.The subject was in good condition, was afebrile and the wound was dry, free from irritation and no liquor pillow was palpable.The event was ongoing and the subject remained hospitalized.On an unspecified date, study drug was interrupted.De-challenge information was not provided.The investigator assessed the intracerebroventricular infection as nci ctcae grade 3 in severity and not related to study drug.The investigator assessed the intracerebroventricular infection as related to the non-study device.The sponsor assessed the intracerebroventricular infection as nci ctcae grade 3 as not related to study drug.The sponsor assessed the intracerebroventricular infection as related to non-study device.Suspect products: ax 250 (ax 250) solution for infusion - 300 milligram, weekly, icv infusion.Diagnosis for use: :mucopolysaccharidosis iii (mucopolysaccharidosis iii).Lot: 251017, 251048.Non-study device (non-study device) n/a.Diagnosis for use: :mucopolysaccharidosis iii (mucopolysaccharidosis iii).Lot 131335 (exp 03/31/2022).Concomitant medical products and therapy dates: cetirizin (cetirizine hydrochloride), paracetamol (paracetamol) , nystatin (nystatin), risperdal (risperidone), cannabidiol (cannabidiol).
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Complaint sample was not returned for evaluation (disposed) and lot number information has not been provided; therefore, an evaluation of the device could not be performed, and manufacturing records could not be reviewed.The device history records as well as sterilization certificate could not be reviewed since lot number was not provided by customer.Furthermore, as specified by the physician in additional information: " the intracerebroventricular infection occurred due to the process of the puncture and the drug administration and is therefore not directly related with the device".The cause(s) of the difficulty reported by the customer could not be clearly determined.Furthermore, as specified in the ifu, in 'precautions' section: "observe aseptic technique throughout placement and use of the ventriculostomy reservoir" and in 'sterility' section: "use aseptic technique in all phases of handling".Complaint will be closed as 'incident not related to device'.Additional information received: the device was implanted on june 20, 2017.It was explanted and disposed.The patient started to experience symptoms on april 12, 2021 and on april 16, 2021 the antibiotic therapy with cefotaxime was stopped, monotherapy with vancomycin was continued until (b)(6) 2021.The central venous catheter was removed without any complications.Reimplantation was not scheduled at the time of the report.On (b)(6) 2021, the event was considered resolved.On (b)(6) 2021, the subject was discharged in good clinical condition.The investigator assessed the intracerebroventricular infection as nci ctcae grade 3 in severity and not related to study drug.The investigator assessed the intracerebroventricular infection as related to the non-study device.Comments from surgeon: "just for the notification, the intracerebroventricular infection occurred due to the process of the puncture and the drug administration and is therefore not directly related with the device.But of course the icv-infection wouldn¿t have occurred if the patient haven¿t had the device.".
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