Clinical trial • Phase III • Neurology

Pyridoxal phosphate for Pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency | Seizures associated with PNPO deficiency

Phase III trial of Pyridoxal phosphate for Pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency | Seizures associated with PNPO deficiency.

Overview

Trial Therapeutic Area: Neurology
Trial Disease: Pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency | Seizures associated with PNPO deficiency
Trial Stage: Phase III
Drug Modality: Small molecule
Paediatric Trial: Yes
Orphan Drug: Yes

External control group (without any P5P treatment). Phase III trial in Poland.

Recruits 3 paediatric patients.

Pregnancy Exclusion: Pregnant female, or at risk of becoming pregnant (a negative pregnancy test and documented confirmation of contraception is required for females of child- bearing potential).
Vulnerable Population: Vulnerable population (minors) are included. Written informed consent must be provided by parent or guardian if participant is under 18. Assent documents are provided: 'L1_SIS and ICF Adolescent and Adult Assent 13 PL' and 'L1_SIS and ICF Child Assent 7-12 PL' are listed among trial documents, and subject information and consent forms for adults, responsible persons, and pregnant participants are provided (documents labelled PL indicating Polish language materials).

{"criterion_text":"-Patients with confirmed PNPO deficiency via genetic analysis, whose seizures are typically controlled on P5P (oral) therapy. a. Typically controlled is defined as receiving multiple doses of P5P daily to control seizures. Receiving P5P for a minimum of 30 days.\n-Male and/or female patients\n-Aged ≥2 years.\n-Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).\n-Written informed consent (by parent or guardian if under the age of 18)."}

{"criterion_text":"-The patient has any condition or abnormality which may, in the opinion of the Investigator, compromise the safety of the patient, or influence their ability to comply with study procedures.\n-Known or suspected allergy to the trial drug or the relevant drugs given in the trial.\n-Involvement in a clinical research study within 4 weeks prior to screening and/or prior enrollment in the study. Participation in observational registry studies is permitted.\n-Pregnant female, or at risk of becoming pregnant (a negative pregnancy test and documented confirmation of contraception is required for females of child- bearing potential)."}

{"endpoint_text":"-Incidence of death at 12 months. The incidence of death at 12 months will be compared between the study group and an external control group (without any P5P treatment).","definition_or_measurement_approach":"Incidence of death at 12 months compared between the study group and an external control group without P5P treatment."}

{"endpoint_text":"-Frequency of seizures (including but not limited to status epilepticus)","definition_or_measurement_approach":"Not specified in available source."}

Planned Sample Size: 3
Recruitment Window Months: 12
Consent Approach: Written informed consent is required; for participants under 18 consent must be provided by a parent or guardian. Assent is obtained from minors where appropriate (documents listed: Adolescent and Adult Assent 13 PL; Child Assent 7-12 PL). Subject information and informed consent forms (SIS and ICF) and responsible person forms are provided; available documents are labelled PL (Polish).

Name: Global Medical Services Polska Sp. z o.o.
Responsibilities: Listed as sponsor public contact and listed contacts for scientific correspondence (public and scientific contact functions for the sponsor).

Investigational Product Name: MC-1
Active Substance: Pyridoxal phosphate
Modality: Small molecule
Routes Of Administration: Oral
Route: Oral
Authorisation Status: prodAuthStatus=1
Orphan Designation: Yes
Maximum Dose: 3 g per day (maxDailyDoseAmount = 3 g); max total amount reported 1095 g

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