Cystinosis
Conditions
Interventions
Investigational drug (PO-001) and comparable drugs (Cystagon® and Procysbi®)
Sponsors
Patient One
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Subjects must meet all of the following criteria to be considered eligible to participate in the study: 1. Healthy male, and 18-55 years of age (inclusive); 2. Body Mass Index between 18 and 27 kg/m2 (inclusive) and body weight minimal 50 kg (inclusive); 3. Ability to read and understand the written consent form, complete study-related procedures, and communicate with the study staff; 4. All males must practice effective contraception during the study and be willing and able to continue contraception for at least 90 days after their last dose of study treatment; 5. Subjects must be able to swallow the drug-administered capsules with the capsule intact; 6. Willingness to comply with study restrictions and requirements.
Exclusion criteria
Exclusion criteria: Subjects are not allowed to participate in the study if any of the following exclusion criteria apply: 1. Clinically relevant abnormal history or presence of physical and mental health as determined by medical history taking and physical examinations obtained during the screening visit and/or at the start of the first study day (as judged by the investigator); 2. Clinically relevant abnormal laboratory results, ECG and vital signs at screening and/or at the start of the first study day (as judged by the investigator); 3. Acute disease state (e.g. nausea, vomiting, fever, or diarrhea) within 7 days before the first study day; 4. Abnormal renal function (eGFR (MDRD)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability endpoints Treatment-emergent (serious) adverse events ((S)AEs) will be documented, regarding incidence, nature and severity from the time the subject signs the consent until the follow-up visit (End of Study Visit). The following endpoints will be determined at the time points indicated in the Schedule of Assessments. Clinical laboratory tests o Haematology o Chemistry o Urinalysis Vital signs o Pulse Rate (bpm) o Systolic blood pressure (mmHg) o Diastolic blood pressure (mmHg) o Temperature ECG o Heart Rate (HR) (bpm), PR, QRS, QT, QTcF Abdominal Visual Analog Scales (VAS) to assess: o Abdominal fullness (completely empty-intolerably full) o Nausea (no nausea-intolerance nausea) o Epigastric pain (no pain-inbearable pain) o Hunger (not at all-intolerable) o Desire to eat (very weak-intolerably strong) Pharmacokinetic endpoints The following endpoints will be determined for the study drug at time points indicated in the Schedule of Assessments. They will be derived by non-compartmental analysis of the plasma concentration-time data: Plasma maximum cysteamine concentration (Cmax) The time to reach maximum plasma concentration (Tmax) The area under the plasma concentration-time curve from zero to infinity (AUC0-inf) The area under the plasma concentration-time curve from zero to t of the last measured concentration above the limit of quantification (AUC0-last) The terminal disposition rate constant (*z) with the respective Half-life (T1/2) Clearance (CL/F) apparent oral clearance calculated from Dose/AUC0-inf) | — |
Secondary
| Measure | Time frame |
|---|---|
| N.A. | — |
Countries
Netherlands
Outcome results
None listed