CompletedPhase 1

Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects Under Fed Conditions

Canada42 participantsStarted 2025-08-20

Plain-language summary

This is a single center, Phase 1, randomized, open-label, single-dose, 2 treatment, 2-period,2-sequence, crossover study designed to compare the pharmacokinetics (PK) of sapropterin between the Test and Reference products in healthy Participants.

Who can participate

Age range18 Years – 50 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Healthy male or female participants, light smokers (no more than 10 cigarettes daily) or non-smokers, from 18 to 50 years of age.
✓. BMI ≥18.5 and ≤30 kg/m2 and weight ≥50.0 and ≤100.0 kg for males and ≥45.0 and ≤100.0 kg for females
✓. Females may be of childbearing or non-childbearing potential:
✓. Willing to use acceptable, effective methods of contraception.
✓. Able to tolerate venipuncture.
✓. Be informed of the nature of the study and give written consent prior to any study procedure.

Exclusion criteria

✕. History of clinically significant neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, genitourinary, psychiatric, or cardiovascular disease or any other condition which, in the opinion of the Investigator, would jeopardize the safety of the participant or impact the validity of the study results.
✕. Clinically significant illness and/or surgery.
✕. Known or suspected carcinoma.
✕. History of hypersensitivity or idiosyncratic reaction to sapropterin dihydrochloride or any other drug substances with similar activity.
✕. History of or predisposition to seizure which, in the opinion of the Investigator, would jeopardize the safety of the participant or impact the validity of the study results.

What they're measuring

Uncorrected and Baseline-corrected Sapropterin AUCt

Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)

Uncorrected and Baseline-corrected Sapropterin AUCinf

Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)

Uncorrected and Baseline-corrected Sapropterin Cmax

Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)

Trial details

NCT IDNCT06987773

SponsorAPR Applied Pharma Research s.a.

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-08-29

Results submitted2026-02-18

View on ClinicalTrials.gov More Bioequivalence trials

Who can participate

Age range18 Years – 50 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Healthy male or female participants, light smokers (no more than 10 cigarettes daily) or non-smokers, from 18 to 50 years of age.
✓. BMI ≥18.5 and ≤30 kg/m2 and weight ≥50.0 and ≤100.0 kg for males and ≥45.0 and ≤100.0 kg for females
✓. Females may be of childbearing or non-childbearing potential:
✓. Willing to use acceptable, effective methods of contraception.
✓. Able to tolerate venipuncture.
✓. Be informed of the nature of the study and give written consent prior to any study procedure.

Exclusion criteria

✕. History of clinically significant neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, genitourinary, psychiatric, or cardiovascular disease or any other condition which, in the opinion of the Investigator, would jeopardize the safety of the participant or impact the validity of the study results.
✕. Clinically significant illness and/or surgery.
✕. Known or suspected carcinoma.
✕. History of hypersensitivity or idiosyncratic reaction to sapropterin dihydrochloride or any other drug substances with similar activity.
✕. History of or predisposition to seizure which, in the opinion of the Investigator, would jeopardize the safety of the participant or impact the validity of the study results.

What they're measuring

Uncorrected and Baseline-corrected Sapropterin AUCt

Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)

Uncorrected and Baseline-corrected Sapropterin AUCinf

Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)

Uncorrected and Baseline-corrected Sapropterin Cmax

Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)