PhenylketonuriaPhase 1NCT04085666

Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)

A Phase 1 study testing CDX 6114 in people with Phenylketonuria, run by Société des Produits Nestlé (SPN) at 2 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This study is an international, multi-center, randomized, double-blind, placebo-controlled, two-treatment, two-period cross-over study to evaluate the pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral dose of CDX-6114 in patients with phenylketonuria (PKU). Read more about this study

Who is it for?: Ages 18 to 55 years
Conditions the study is for: Phenylketonuria
What is being tested?: CDX 6114
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 18 participants
Where: 2 sitesin 2 countries
Study dates: 1 Jun 2019to 30 Aug 2020
Phase: Phase 1
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 55 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Male and female patients between the ages of 18 and 55 years (inclusive) with a diagnosis of classical PKU by either a historical blood Phe concentration of > 1200 mol/L at any time or a genetic diagnosis of PKU.
Patients capable of following dietary instructions to maintain protein intake stable throughout the study duration based on principal investigator and dietician assessment.
Patients with a blood Phe concentration < 1200mol/L at screening.
Have a body mass index (BMI) between 18.0 and 30.0 kg/m2 inclusive and weigh at least 50 kg and no more than 100 kg inclusive.
Patients must be in good general health, as determined by the PI or delegate, based on a medical evaluation including detailed medical history, full physical examination, including blood pressure and pulse rate measurement, 12-lead electrocardiogram (ECG) and clinical laboratory tests.
Male patients (unless surgically sterilised) and their female spouse/partner(s) who are of childbearing potential:

Show all 28Show fewer

Must agree to stay abstinent (where abstinence is the preferred and usual life-style of the patient), starting at screening and continuing throughout the clinical study period, and for 90 days after last study drug administration.
Or
Must be using highly effective contraception starting at screening and continuing throughout the clinical study period, and for 90 days after last study drug administration. Highly effective contraception is defined as follows:
Injectable or implantable hormones ii. Intrauterine device iii. Surgical sterilisation iv. Sterilisation implant device v. Combined oral contraceptives vi. Use of a condom plus oral or injectable or implantable or intrauterine contraception
These requirements do not apply to participants in a same sex relationship.
Male patients must agree not to donate sperm starting at screening and continuing throughout the clinical study period, and for 90 days after last study drug administration.
Female patients of childbearing potential and their spouse/partner(s):
Must agree not to become pregnant during the clinical study period and for 30 days after last study drug administration.
Must have a negative serum pregnancy test at screening.
If heterosexually active, must agree to consistently use a form of highly effective contraception, starting at screening and continuing throughout the clinical study period, and for 30 days after last study drug administration. Highly effective contraception is defined as follows:
Injectable or implantable hormones ii. Intrauterine device iii. Surgical sterilisation iv. Sterilisation implant device v. Surgical sterilisation of the male partner vi. Combined oral contraceptives vii. Use of a condom plus oral or injectable or implantable or intrauterine contraception
Or
Must agree to stay abstinent (where abstinence is the preferred and usual life-style of the patient), starting at screening and continuing throughout the clinical study period, and for 30 days after last study drug administration.
These requirements do not apply to participants in a same sex relationship.
Female patients of non-childbearing potential:
Must have a confirmed clinical history of sterility (e.g. the patient is without a uterus).
Or
Must be postmenopausal as defined as amenorrhea for at least 1 year prior to screening and a laboratory confirmed serum follicle stimulating hormone (FSH) level ≥ 40mIU/mL or similar value considered to be in the menopausal range.
Female patients must agree not to breastfeed from screening, throughout the clinical study period, and until 90 days after last study drug administration.
Female patients must agree not to donate ova from screening, throughout the clinical study period, and until 90 days after last study drug administration.
Patient must be competent to understand the nature of the study and capable of giving written informed consent. Be willing to report for the scheduled study visits and communicate to study personnel about adverse events and concomitant medication use.
Patient agrees not to participate in another interventional study while participating in the present clinical study.

Who cannot join

Female patient who has been pregnant within the 6 months prior to screening or breastfeeding within the 3 months prior to screening.
Evidence or history of clinically significant haematological, renal, endocrine, pulmonary, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies and childhood asthma) at time of screening.
Current or chronic history of gastrointestinal illness or conditions interfering with normal gastrointestinal anatomy or motility. Examples include gastrointestinal bypass surgery, partial or total gastrectomy, small bowel resection, vagotomy, malabsorption, Crohn's disease, ulcerative colitis, irritable bowel syndrome (IBS) or celiac sprue.
Evidence or history of gastrointestinal symptoms that could lead to the assumption of an underlying gastrointestinal impairment.
Treatment with any anti-platelet and/or anticoagulant medication.
Evidence or history of specific food intolerance. Examples include coeliac disease, severe lactose or dairy food intolerance or a severe intolerance to any food/ingredient included in the standard breakfast.

Show all 20Show fewer

Any positive result, on screening, for serum hepatitis B surface antigen (HBsAg), hepatitis C virus antibodies (HCV) or antibodies to human immunodeficiency virus type 1 (HIV-1) and/or type 2 (HIV-2).
A positive drug/alcohol result.
Patient has a history of exceeding > 21 units of alcohol/week for male patients or > 14 units of alcohol/week for female patients within the 3 months prior to screening. One unit of alcohol is equivalent to 5 ounces (150 mL) of wine or 12 ounces (360 mL) of beer or 1.5 ounces (45 mL) of spirits.
Patient has a history of regular smoking (daily or most days in a week) or the use of nicotine products (3 or more nicotine-containing products) who would be unable to abstain from smoking during the confinement periods in the Phase 1 Unit.
Patient has used any recreational drugs of abuse within the 3 months prior to screening.
Patient has a pulse rate ≤ 50 or ≥ 100 bpm; mean systolic blood pressure (SBP) > 140 mmHg; mean diastolic blood pressure (DBP) > 90 mmHg at screening (excluding white-coat hypertension; therefore, a repeat test showing results within range will be acceptable).
Patient has any clinically significant abnormalities at screening in rhythm, conduction or morphology of the resting ECG and any clinically significant abnormalities in the 12-lead ECG, as considered by the PI or delegate, which may interfere with the interpretation of QTc interval changes including abnormal ST-T wave morphology.
Patient has prolonged QTcF (QT interval corrected for heart rate using Fridericia's formula) > 450 ms for male patients or > 470 ms for female patients, or a shortened QTcF < 300 ms or a family history of prolonged QT syndrome, at screening.
Patient has any clinically significant abnormalities in clinical chemistry, haematology, or urinalysis at screening as judged by the PI or delegate, including: Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), alkaline phosphatase (ALP), gamma glutamyl transferase (GGT) or Total bilirubin (TBL) more than 2.0 times above the Upper Limit of Normal (ULN).
Plasma donation within the 14 days prior to screening or any whole blood donation/significant blood loss > 500 mL during the 3 months prior to screening.
Treatment with any Investigational Drug or Device/Treatment within the 30 days prior to the first administration of study drug.
Use of any prescribed or non-prescribed medication including herbal medicines, antacids and analgesics (other than anti-hypertensive drugs, oral contraceptives, paracetamol or multi-vitamins) for the two weeks prior to the initial administration of the study medication or up to a minimum of 5 times the half-life of the medication if it has a long half-life.
Known allergy or adverse reaction history to any of the oral dose formulation components e.g. mannitol.
Use of Sapropterin (KUVAN) or pegylated phenylalanine ammonia lyase (Palinzyk) or SYNB1618 or any Phenylalanine-lowering drug within the last 4 weeks.

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

Australia1 site

Linear Clinical Research Ltd
Nedlands, Western Australia, 6009

Other trials in Australia

Germany1 site

Profil Institut für Stoffwechselforschung GmbH
Neuss, D-41460

Other trials in Germany

About this study

Official title: A Phase 1, Multi-center, Randomized, Double-blind, Placebo-controlled, Cross-over Study to Evaluate the Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of a Single Oral Dose of CDX-6114 in Patients With Phenylketonuria (PKU).

Conditions: Phenylketonuria
Being tested: CDX 6114, Matching Placebo
Medical subject headings: Phenylketonurias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

CDX 6114Drug
CDX-6114 for oral administration is formulated in phosphate buffer, which also includes mannitol and poloxamer.The vehicle solution provided is identical to the CDX-6114 oral solution except for the active drug.
Matching PlaceboOther
The placebo oral dosing solution will also be supplied as an approximately 240 mL oral solution and will be made up of the phosphate buffer diluent and the caramel flavoring.

Study groups in NCT04085666
Study group	Role	What they receive
1st Confinement Period in Unit	Experimental	Randomized to treatment with either CDX-6114 or matching Placebo Receives: Drug: CDX 6114; Other: Matching Placebo
2nd Confinement Period in Unit	Experimental	Randomized to treatment with either CDX-6114 or matching Placebo Receives: Drug: CDX 6114; Other: Matching Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in concentration of post parandial plasma level of Phe will be summarized over time for each treatment
Blood samples will be collected at the following time points to determine the postprandial plasma levels of Phe following single does of CDX-6114
Measured: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8
Change in concentration of post parandial plasma level of CA will be summarized over time for each treatment
Blood samples will be collected at the following time points to determine the postprandial plasma levels of CA following single does of CDX-6114
Measured: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8
Change in the peak Phe concentration in Plasma will be summarized by treatment
Blood samples will be collected at the following time points to determine the Peak Phe plasma concentration follwoing a single, oral dose of CDX-6114
Measured: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Change in the peak CA concentration in Plasma will be summarized by treatment
Blood samples will be collected at the following time points to determine the Peak CA plasma concentration following a single, oral dose of CDX-6114
Measured: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Phe Area under the plasma concentration versus time curve (AUC) , over a 5-hour period, following dosing and the standardized breakfast
Blood samples will be collected at the following time points to determine the Phe AUC following a single, oral dose of CDX-6114
Measured: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
CA Area under the plasma concentration versus time curve (AUC) , over a 5-hour period, following dosing and the standardized breakfast
Blood samples will be collected at the following time points to determine the CA AUC following a single, oral dose of CDX-6114
Measured: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8

Secondary outcomes (12)

Incidence of Treatment-Emergent Adverse Events (AEs) will be measured
The safety and tolerability of CDX-6114 following single dose oral administration assesed by Adverse events monitoring
Measured: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
The serum levels of CDX-6114 will be summarized descriptively over time
Blood will be collected for pharmacokinetic analysis of CDX-6114 at the following time points follwoing a single oral dose of CDX-6114
Measured: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Absolute values and changes from baseline in blood pressure measurements will be summarized over time for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by blood pressure monitoring
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in Heart rate measurements will be summarized over time for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by Heart rate monitoring
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in Respiratory rate measurements will be summarized over time for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by Respiratory rate monitoring
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in body temperature (in Fahrenheit or Celsius) measurements will be summarized over time for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by Body temperature monitoring
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in 12 lead Electrocardiogram (ECG) measurements will be summarized over time for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by 12 lead ECG including P Wave, QRS Complex, QT Interval
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values of Weight measurements will be summarized over time for each treatment using a weighing scale in Kg or pounds
The safety and tolerability of CDX-6114 following single dose oral administration assesed by weight monitoring
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1hr, 1.5hr, 2hr, 4hr and 5hr after dosing on both Day 1 and Day 8.
Absolute blood composition values and changes from baseline to the last post-dose time-point will be summarized for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by laboratory assessments as Haematology and Coagulation
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute urine composition values and changes from baseline to the last post-dose time-point will be summarized for each treatment
The safety and tolerability of CDX-6114 following single dose oral administration assesed by routine urinalysis laboratory assessments
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Assesment of the incidence of Treatment-Emergent Antibodies
The safety and tolerability of CDX-6114 following single dose oral administration assesed by assessment for development of anti-CDX-6114 antibodies
Measured: Assesments will be done on day 1 and day 8. Blood will be collected for analysis within 30 minutes prior to dosing on Day 1 and Day 8 and again at the End of Study Visit ( through study completion , an avearge of 8 to 10 weeks)
absolute values of height measurements will be summarized over time for each treatment using length measurement scale in centimeters or inches
The safety and tolerability of CDX-6114 following single dose oral administration assesed by height examination using Lenght scale
Measured: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1hr, 1.5hr, 2hr, 4hr and 5hr after dosing on both Day 1 and Day 8.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Jun 201930 Aug 2020

First posted11 Sep 2019
Study start1 Jun 2019
Primary completion30 Jun 2020
Study completion30 Aug 2020

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for PhenylketonuriaStudies of the same condition, open and under way.
More trials run by Société des Produits Nestlé (SPN)The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Other
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04085666
Sponsor's protocol ID: CDX6114-002
Status last verified: Sep 2020
First submitted: 3 Aug 2019
Last updated: 14 Sep 2020
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria

All Phenylketonuria trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1, Multi-center, Randomized, Double-blind, Placebo-controlled, Cross-over Study to Evaluate the Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of a Single Oral Dose of CDX-6114 in Patients With Phenylketonuria (PKU).
ClinicalTrials.gov ID: NCT04085666
Acronym / sponsor ID: CDX6114-002
Conditions: Phenylketonuria
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: Société des Produits Nestlé (SPN)Industry (pharma, biotech, devices)
Study start: 1 Jun 2019 (actual)
Primary completion: 30 Jun 2020 (actual)
Study completion: 30 Aug 2020 (actual)
First posted: 11 Sep 2019 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Other
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
1st Confinement Period in Unit	Experimental	Drug: CDX 6114, Other: Matching PlaceboRandomized to treatment with either CDX-6114 or matching Placebo
2nd Confinement Period in Unit	Experimental	Drug: CDX 6114, Other: Matching PlaceboRandomized to treatment with either CDX-6114 or matching Placebo

Who may qualify

Ages: 18 to 55 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (28 listed)

Key exclusion criteria (20 listed)

Female patient who has been pregnant within the 6 months prior to screening or breastfeeding within the 3 months prior to screening.
Evidence or history of clinically significant haematological, renal, endocrine, pulmonary, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies and childhood asthma) at time of screening.
Current or chronic history of gastrointestinal illness or conditions interfering with normal gastrointestinal anatomy or motility. Examples include gastrointestinal bypass surgery, partial or total gastrectomy, small bowel resection, vagotomy, malabsorption, Crohn's disease, ulcerative colitis, irritable bowel syndrome (IBS) or celiac sprue.
Evidence or history of gastrointestinal symptoms that could lead to the assumption of an underlying gastrointestinal impairment.
Treatment with any anti-platelet and/or anticoagulant medication.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
CDX 6114CDX-6114 for oral administration is formulated in phosphate buffer, which also includes mannitol and poloxamer.The vehicle solution provided is identical to the CDX-6114 oral solution except for the active drug.	Drug	1st Confinement Period in Unit, 2nd Confinement Period in Unit
Matching PlaceboThe placebo oral dosing solution will also be supplied as an approximately 240 mL oral solution and will be made up of the phosphate buffer diluent and the caramel flavoring.	Other	1st Confinement Period in Unit, 2nd Confinement Period in Unit

Primary outcome

Change in concentration of post parandial plasma level of Phe will be summarized over time for each treatment
Time frame: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8
Blood samples will be collected at the following time points to determine the postprandial plasma levels of Phe following single does of CDX-6114
Change in concentration of post parandial plasma level of CA will be summarized over time for each treatment
Time frame: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8
Blood samples will be collected at the following time points to determine the postprandial plasma levels of CA following single does of CDX-6114
Change in the peak Phe concentration in Plasma will be summarized by treatment
Time frame: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Blood samples will be collected at the following time points to determine the Peak Phe plasma concentration follwoing a single, oral dose of CDX-6114
Change in the peak CA concentration in Plasma will be summarized by treatment
Time frame: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Blood samples will be collected at the following time points to determine the Peak CA plasma concentration following a single, oral dose of CDX-6114
Phe Area under the plasma concentration versus time curve (AUC) , over a 5-hour period, following dosing and the standardized breakfast
Time frame: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Blood samples will be collected at the following time points to determine the Phe AUC following a single, oral dose of CDX-6114
CA Area under the plasma concentration versus time curve (AUC) , over a 5-hour period, following dosing and the standardized breakfast
Time frame: Within 30 minutes, within 10 minutesand immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Blood samples will be collected at the following time points to determine the CA AUC following a single, oral dose of CDX-6114

12 secondary outcomes

Incidence of Treatment-Emergent Adverse Events (AEs) will be measured
Time frame: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
The serum levels of CDX-6114 will be summarized descriptively over time
Time frame: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8
Absolute values and changes from baseline in blood pressure measurements will be summarized over time for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in Heart rate measurements will be summarized over time for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in Respiratory rate measurements will be summarized over time for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in body temperature (in Fahrenheit or Celsius) measurements will be summarized over time for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values and changes from baseline in 12 lead Electrocardiogram (ECG) measurements will be summarized over time for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute values of Weight measurements will be summarized over time for each treatment using a weighing scale in Kg or pounds
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1hr, 1.5hr, 2hr, 4hr and 5hr after dosing on both Day 1 and Day 8.
Absolute blood composition values and changes from baseline to the last post-dose time-point will be summarized for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Absolute urine composition values and changes from baseline to the last post-dose time-point will be summarized for each treatment
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1, 1.5, 2, 4 and 5hours after dosing on both Day 1 and Day 8.
Assesment of the incidence of Treatment-Emergent Antibodies
Time frame: Assesments will be done on day 1 and day 8. Blood will be collected for analysis within 30 minutes prior to dosing on Day 1 and Day 8 and again at the End of Study Visit ( through study completion , an avearge of 8 to 10 weeks)
absolute values of height measurements will be summarized over time for each treatment using length measurement scale in centimeters or inches
Time frame: Within 30 minutes prior to dosing, and then at 15 minutes, 30 minutes, 1hr, 1.5hr, 2hr, 4hr and 5hr after dosing on both Day 1 and Day 8.

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2020.

Related trials for Phenylketonuria

RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Damon Bell principal investigator

Linear Clinical Research

Tim Heise principal investigator

Profil Institut für Stoffwechselforschung GmbH

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CDX 6114 in people with Phenylketonuria, run by Société des Produits Nestlé (SPN) at 2 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 28 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 14 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Australia, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in concentration of post parandial plasma level of Phe will be summarized over time for each treatment (time frame: Within 30 minutes, within 10 minutes and immediately prior to dosing, and then at 15 minutes, 30 minutes, 1hours, 1.5, 2,4 and 5hours after dosing on both Day 1 and Day 8). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04085666.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2020.