Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients

Nestlé

Status and phase: Withdrawn; Phase 1

Conditions: Phenylketonurias

Treatments: Drug: Cohort 2 0.75g; Drug: cohort 1 0.225g; Drug: Cohort 3 2.25 g

Study type: Interventional

Funder types: Industry

Identifiers

NCT04256655

19.05.CLI

Details and patient eligibility

About

The primary objective is of this Phase 1 study is to evaluate the safety and tolerability of daily, multiple, oral doses of CDX-6114 when administered to patients with PKU for 14 days. The aim is to check if administration of daily, multiple, oral doses of CDX-6114 to patients with PKU for 14 days shows a clinically acceptable safety and tolerability profile.

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

1. Male and female patients between the ages of 18 and 65 years, with a diagnosis of classical PKU
2. Patients with a blood phenylalanine concentration > 600mol/L at screening as an indicator of sub-optimal dietary management
3. Body mass index (BMI) between 18 and 35 kg/m2 at screening.
4. Male patients must agree not to donate sperm starting at screening and continuing throughout the clinical study period up to 90 days after last study drug administration
5. Female patients of childbearing potential and their spouse/partner
6. Female patients of non-childbearing potential:
7. Female patients must agree not to breastfeed. This includes the period starting at screening and continuing throughout the clinical study period up to 90 days after last study drug administration.
8. Female patients must agree not to donate ova. This includes the period starting at screening and continuing throughout the clinical study period up to 90 days after last study drug administration.
9. Patients must be deemed competent to understand the nature of the study and capable of giving written informed consent. Patients must also be willing to attend scheduled study visits in person and to reliably communicate to study personnel on adverse events and concomitant medication use.
10. Patients must agree not to participate in another interventional study while participating in the present clinical study.

Exclusion Criteria

1. Presence or history of clinically significant hematological, renal, endocrine, pulmonary, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease/condition (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies and childhood asthma).
2. Presence or history of gastrointestinal illness or conditions interfering with normal gastrointestinal anatomy. Examples include gastrointestinal bypass surgery, cholecystectomy, partial or total gastrectomy, gastric band surgery, small bowel resection, vagotomy, malabsorption, Crohn's disease, ulcerative colitis, or coeliac disease.
3. Active treatment with any platelet aggregation inhibitor and/or active treatment (or within the last 4 weeks) with anticoagulant medication.
4. Presence or history of specific food intolerance. Examples include coeliac disease, severe lactose or dairy food intolerance.
5. Positive result for serum hepatitis B surface antigen (HBsAg), hepatitis A virus antibodies (HAV), hepatitis C virus antibodies (HCV), or antibodies to human immunodeficiency virus type 1 (HIV-1) and/or type 2 (HIV-2) at Screening.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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0 participants in 3 patient groups

Cohort 1 0.225 g

Experimental group

Description:

Randomized to treatment with either CDX-6114 0.225g or matching Placebo

Treatment:

Drug: cohort 1 0.225g

Cohort 2 0.75g

Experimental group

Description:

Randomized to treatment with either CDX-6114 0.75g or matching Placebo

Treatment:

Drug: Cohort 2 0.75g

Cohort 3 2.25g

Experimental group

Description:

Randomized to treatment with either CDX-6114 2.25 g or matching Placebo

Treatment:

Drug: Cohort 3 2.25 g