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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 16 December 2017
Main ID:  NCT02750332
Date of registration: 21/04/2016
Prospective Registration: No
Primary sponsor: Cycle Pharmaceuticals Ltd.
Public title: Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
Scientific title: A Single Center, Single-Dose, Open-Label, Randomized Study to Compare the Bioavailability of an Oral Test Formulation Containing Nitisinone 10 mg in at Least 16 Healthy Male and Female Subjects Under Fasting and Fed Conditions
Date of first enrolment: November 2015
Target sample size: 20
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02750332
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Basic Science. Masking: None (Open Label).  
Phase:  Phase 1
Countries of recruitment
South Africa
Contacts
Name:     AndrĂ© Nell
Address: 
Telephone:
Email:
Affiliation:  +27 51 410 3046
Key inclusion & exclusion criteria

Inclusion Criteria:

- Healthy male and female subjects, 18 to 55 years (both inclusive) at signing of
informed consent.

- Body Mass Index (BMI) between 18.5 and 30 kg/m2 (inclusive).

- Body mass not less than 50 kg.

- Medical history, vital signs, physical examination, standard 12-lead electrocardiogram
(ECG) and laboratory investigations must be clinically acceptable or within laboratory
reference ranges for the relevant laboratory tests, unless the investigator considers
the deviation to be irrelevant for the purpose of the study.

- Non-smokers.

- Females, if:

Of childbearing potential, the following conditions are to be met:

- Negative pregnancy test If this test is positive, the subject will be excluded from
the study. In the rare circumstance that a pregnancy is discovered after the subject
received IMP, every attempt must be made to follow her to term.

- Not lactating

- Abstaining from sexual activity (if this is the usual lifestyle of the subject) or
must agree to use an accepted method of contraception, and agree to continue with the
same method throughout the study Examples of reliable methods of contraception include
non-hormonal intrauterine device, and barrier methods combined with an additional
contraceptive method. In this study the concomitant use of hormonal contraceptives is
NOT allowed. Other methods, if considered by the investigator as reliable, will be
accepted.

- Written consent given for participation in the study.

- Subjects must be willing to consume the meal prescribed with administration of
the IMP in full and within the required time.

Exclusion Criteria:

- Evidence of psychiatric disorder, antagonistic personality, poor motivation, emotional
or intellectual problems likely to limit the validity of consent to participate in the
study or limit the ability to comply with protocol requirements.

- Current alcohol use > 21 units of alcohol per week for males and > 14 units of alcohol
per week for females.

- Consumption of more than 5 cups of coffee (or equivalent amounts of caffeine) per day.

- Regular exposure to substances of abuse (other than alcohol) within the past year.

- Use of any medication, prescribed or over-the-counter or herbal remedies, within 2
weeks before the first administration of investigational medicinal product (IMP)
except if this will not affect the outcome of the study in the opinion of the
investigator.

In this study the concomitant use of hormonal contraceptives is NOT allowed.

- Participation in another study with an experimental drug, where the last
administration of the previous IMP was within 8 weeks (or within 10 elimination
half-lives for chemical entities or 2 elimination half-lives for antibodies or
insulin), whichever is the longer) before administration of IMP in this study, at the
discretion of the investigator.

- Treatment within the previous 3 months before the first administration of IMP with any
drug with a well-defined potential for adversely affecting a major organ or system.

- A major illness during the 3 months before commencement of the screening period.

- History of hypersensitivity or allergy to the IMP or its excipients or any related
medication.

- History of bronchial asthma or any other bronchospastic disease.

- History of convulsions.

- History of porphyria.

- Relevant history or laboratory or clinical findings indicative of acute or chronic
disease, likely to influence study outcome.

- Donation or loss of blood equal to or exceeding 500 mL during the 8 weeks before the
first administration of IMP.

- Diagnosis of hypotension made during the screening period.

- Diagnosis of hypertension made during the screening period or current diagnosis of
hypertension.

- Resting pulse of > 100 beats per minute or < 40 beats per minute during the screening
period, either supine or standing.

- Positive testing for human immunodeficiency virus (HIV), Hepatitis B and Hepatitis C.

- Positive urine screen for drugs of abuse. In case of a positive result the urine
screen for drugs of abuse may be repeated once at the discretion of the investigator.

- Positive urine screen for tobacco use.

- Positive pregnancy test.

- Female subjects that are pregnant or breastfeeding.

- Difficulty in swallowing.

- Any specific investigational product safety concern.

- Vulnerable subjects, e.g. persons in detention.

- Subjects with current keratopathy, or other clinically significant abnormalities found
by slit-lamp examination (cataracts) at the discretion of the investigator.

- Concomitant use of medications that are metabolized by CYP2C9 (ibuprofen, diclofenac
and indomethacin).



Age minimum: 18 Years
Age maximum: 55 Years
Gender: All
Health Condition(s) or Problem(s) studied
Hereditary Tyrosinemia, Type I
Intervention(s)
Drug: Nitisinone
Primary Outcome(s)
Area Under the Plasma Concentration Versus Time Curve (AUC(0-120)) [Time Frame: 0 - 120 hours post-dose]
Maximum Observed Plasma Concentration (Cmax) [Time Frame: 0 - 120 hours post-dose]
Secondary Outcome(s)
Apparent Terminal Elimination Half-life (t1/2) [Time Frame: 0 - 120 hours post-dose]
Area Under the Plasma Concentration Versus Time Curve (AUC(0-72)) [Time Frame: 0 - 72 hours post-dose]
Terminal Elimination Rate Constant (?z) [Time Frame: 0 - 120 hours post-dose]
Area Under the Plasma Concentration Versus Time Curve, With Extrapolation to Infinity (AUC(0-8)) [Time Frame: 0 - 120 hours post-dose]
Time to Maximum Observed Plasma Concentration (Tmax) [Time Frame: 0 - 120 hours post-dose]
Secondary ID(s)
CT-002
PXL225421
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Parexel
Ethics review
Results
Results available: Yes
Date Posted: 13/12/2016
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT02750332
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