Not yet recruitingNCT07927608ClinicalTrials.gov record

A First-in-Human Study of Phelimin in Healthy Volunteers and Adults With PKU

Phenylketonuria (PKU) · Phenylketonuria

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THE QUICK READ

What this study involves

The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.

Study type: Interventional
Phase: Phase 1
Age range: 18 years - 60 years
Sex eligible: Male
Healthy volunteers: Accepted
Participants: 7 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

This first-in-human study will evaluate the safety and tolerability of phelimin, an investigational oral treatment being developed for people with phenylketonuria (PKU). Phelimin is designed to bind the amino acid phenylalanine in the gut, which may reduce its absorption from food and support its removal from the body in stool. The study will include healthy volunteers and adults with classical PKU. Participants will receive single doses of phelimin at increasing dose levels and will undergo safety assessments, including monitoring for side effects, laboratory tests, and gastrointestinal symptoms. The study will also explore whether phelimin affects blood phenylalanine levels after a standardised protein challenge.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

Inclusion Criteria:

COMMON FOR BOTH PARTICIPANT GROUPS

1. Age 18-60 years at screening.
2. Male gender.
3. Body weight 60-90 kg.
4. Body mass index (BMI): ≤32 kg/m\^2.
5. Negative alcohol breath test and urine drugs of abuse test on the day of screening.
6. Males, and their female partners of childbearing potential, must be willing to use highly effective methods of birth control during the study period and until 90 days after end of treatment. Highly effective methods of birth control are defined as those which result in a low failure rate (i.e. less than 1% per year) when used consistently and correctly. Such methods include:

i. combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation

oral
intravaginal
transdermal ii. progestogen-only hormonal contraception associated with inhibition of ovulation
oral
injectable
implantable iii. intrauterine device iv. intrauterine hormone-releasing system v. bilateral tubal occlusion vi. vasectomized partner vii. sexual abstinence (if this is the preferred and usual lifestyle of the patient) g. Signed and dated informed consent form (ICF).

For HEALTHY VOLUNTEERS h. Good health in the opinion of the Investigator based on medical history, physical examination, vital signs, electrocardiogram (ECG) and laboratory measurements of standard blood and urine parameters.

For PKU PATIENTS i. Diagnosis of classical PKU based on medical history (genotyping and/or p-Phe concentration \>1200 μmol/L at any time in life).

j. Currently, and prior to screening, reasonably well controlled (see below) on low protein diet; with no single p-Phe measurement \>1200 μmol/L during last 3 months prior to screening k. Within study, p-Phe ≤900 μmol/L taken in the clinic at screening and confirmed at the baseline visit (visit 2) prior to protein challenge and at the 'Day -3' sampling prior to each clinic visit l. With the exception of the underlying PKU diagnosis, good health in the opinion of the Investigator based on medical history, physical examination, vital signs, ECG and laboratory measurements of standard blood and urine parameters.

Exclusion Criteria:

COMMON

1. Any condition (such as irritable bowel syndrome, inflammatory bowel disease, chronic gastritis, peptic ulcer disease, celiac disease) or receiving prescription medication or any over-the-counter (OTC) product that may possibly affect absorption of nutrients or IMP.
2. History of severe GI problems, incl. gastrectomy, bypass surgery or ileostomy.
3. Active or past history of any GI bleeding within 60 days prior to the screening visit.
4. Active laxative use within 14 days prior to baseline, and during trial.
5. Abnormal liver function tests defined by:

* Aspartate aminotransferase (AST), alanine aminotransferase (ALT) or alkaline phosphatase (ALP) \>3x the upper limit of the normal range (ULN). Elevated gamma-glutamyl transferase (GGT) values exceeding \>3x ULN are allowed but these GGT cases will be carefully assessed alongside other clinical and laboratory data by the investigator.
6. Any other clinically significant laboratory abnormality as determined by the investigator.
7. Ongoing, active infectious disease.
8. Known human immunodeficiency virus (HIV)-positive status or suffering from any other immunosuppressive disease.
9. Current participation in any other investigational drug trial or use of any investigational agent within 30 days or 5 half-lives before screening, whichever is longer.
10. Any acute or chronic medical, surgical or social condition (e.g. history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, GI, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that may increase the patient's risk associated with study participation, compromise adherence to study procedures and requirements, or may confound interpretation of study results, and in other ways, in the judgement of the investigator, would make the patient inappropriate for enrolment.
11. Patients with documented or suspected, clinically significant, alcohol (i.e., \> 24 g/d for men) or drug abuse within the past 12 months.
12. Any other significant, unstable medical condition that would interfere with the completion of the study or interpretation of results.
13. Is unable, in the opinion of the investigator, to comply with the requirements of the study.

For PKU PATIENTS, only
14. Use of any medication including sapropterin, sepiapterin, pegvaliase or LNAAs intended to treat PKU.

Understand screening

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment: Not applicable
Masking: No masking

Protein/water followed by protein/phelimin

Not reported in this registry record.

Drug: Phelimin

Phelimin DRUG

Phelimin is an orally administered investigational medicinal product based on molecular imprinted polymer technology designed to bind phenylalanine and phenylalanine-containing oligopeptides in the GI tract, facilitating their elimination without systemic absorption.

Prepare your questions

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Treatment-Emergent Adverse Events

Time frame: From enrollment to 4 weeks after last dose.

Frequency of any TEAEs
Frequency of any TEAE by system organ class (SOC),
Frequency of any TEAE by preferred term (PT),
Frequency of any TEAE by severity, and
Frequency of any TEAE by relationship of TEAEs to IMP.

AEs

Time frame: From enrollment to 4 weeks after last dose

Frequency of any AE
Frequency of any AE by SOC,
Frequency of any AE by PT,
Frequency of any AE by severity, and
Frequency of any AE by relationship of AEs to IMP.

Serious adverse events (SAE)

Time frame: From enrollment to 4 weeks after the last dose.

Frequency of any SAE
Frequency of any SAE by SOC,
Frequency of any SAE by PT,
Frequency of any SAE by severity and
Frequency of any SAE by relationship of SAEs to IMP.

Secondary measures

Adverse Events of Special Interest (AESIs; related to GI symptoms)

Time frame: From enrollment to 4 weeks after last dose.

Frequency of any AESI
Frequency of any AESIs by SOC,
Frequency of any AESIs by PT,
Frequency of any AESIs by severity, and
Frequency of any AESIs by relationship of AESIs to IMP.

Discontinuation rate

Time frame: From enrollment to 4 weeks after last dose.

Overall
Due to TEAEs

Safety laboratory variables

Time frame: From enrollment to 4 weeks after last dose

For each safety laboratory variable:

Frequency of below normal range (clinically significant)
Frequency of below normal range (not clinically significant)
Frequency of within normal range
Frequency of above normal range (not clinically significant)
Frequency of above normal range (clinically significant)