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

NCT06987773 | Completed Phase 1 Results FDA Drug

• 1 other identifier: 2024-5705
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Bioequivalence

Outcome Measures

Primary Outcomes (3)

• Uncorrected and Baseline-corrected Sapropterin AUCt

  The area under the analyte concentration versus time curve from time zero to the time of the last measurable analyte concentration (t)

  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

  The area under the analyte concentration versus time curve from time zero to infinity

  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

  Maximum measured plasma analyte concentration over the sampling period

  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)

Secondary Outcomes (2)

• Uncorrected and Baseline-corrected Sapropterin Tmax

  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 Tlag

  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)

Other Outcomes (6)

• Uncorrected and Baseline-corrected Sapropterin Thalf

  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 Residual Area

  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 Kel

  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)

Study Arms (2)

AB

OTHER

A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water followed by B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water

Drug: RLF-OD032 100 mg/mL oral suspension; Drug: Kuvan 100 MG Powder for Oral Solution

BA

OTHER

B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water followed by A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032), administered under fed conditions without water

Drug: RLF-OD032 100 mg/mL oral suspension; Drug: Kuvan 100 MG Powder for Oral Solution

Interventions

RLF-OD032 100 mg/mL oral suspension DRUG

Sapropterin dihydrochloride

AB; BA

Kuvan 100 MG Powder for Oral Solution DRUG

Sapropterin dihydrochloride

AB; BA

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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:
• Childbearing potential:
• Physically capable of becoming pregnant
• Non-childbearing potential:
• Surgically sterile (i.e., both ovaries removed, uterus removed, or bilateral tubal ligation) at least three (3) months prior to first drug administration; and/or;
• Postmenopausal (no menstrual period for at least 12 consecutive months without any other medical cause and a FSH value consistent with being postmenopausal).
• 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.

You may not qualify if:

• 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.
• History of upper gastrointestinal (GI) mucosal inflammation, esophagitis, gastritis, pharyngitis, or oropharyngeal pain, which, in the opinion of the Investigator, would jeopardize the safety of the participant or impact the validity of the study results.
• History of abuse of medicinal product or drugs within the last three (3) years.
• History of alcohol addiction requiring treatment.
• History or presence of alcoholism within the last three (3) years. (\>40 g ethanol/day or \>10 units per week \[one (1) unit =150 mL of wine, or 360 mL of beer, or 45 mL of 45% alcohol\])
• History of recreational use of soft drugs (such as marijuana) within one (1) month or hard drugs (such as cocaine, phencyclidine \[PCP\], crack, opioid derivatives including heroin, and amphetamine derivatives) within three (3) months prior to screening.
• Use of St. John's wort within 30 days prior to the first drug administration.
• History of clinically significant lactose, galactose, or fructose intolerance.
• Presence of hepatic or renal dysfunction.
• Presence of mouth piercings (object or hole), presence of non-removable dentures and orthodontic appliances (e.g., braces, retainers), or any other alteration to the mouth that may be deemed by the Investigator to compromise drug delivery.
• Note: Dental fillings, crowns, bridges, and implants are permitted as they are not considered to compromise drug delivery.

Sponsors & Collaborators

• APR Applied Pharma Research s.a.: lead

Study Sites (1)

Pharma Medica Research Inc. 4770 Sheppard Ave E,

Toronto, Ontario, M1S3V6, Canada

Location

MeSH Terms

Interventions

Suspensions; sapropterin; Powders; Solutions

Intervention Hierarchy (Ancestors)

Colloids; Complex Mixtures; Dosage Forms; Pharmaceutical Preparations

Results Point of Contact

• Title: Giorgio Reiner Chief Scientific Officer
• Organization: APR Applied Pharma Research s.a.

giorgio.reiner@apr.ch

+41.91.6957020

Study Officials

• Mark L. Freedman, MD,FRCPC

  Canada, Ontario - Pharma Medica Research Inc. 4770 Sheppard Ave E

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Adaptative design In each period, subjects will receive one of the following treatments, according to the randomization scheme: Treatment A: 1 x 10 mg/kg dose of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. Treatment B: 1 x 10 mg/kg dose of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions

Study Record Dates

• First Submitted: May 7, 2025
• First Posted: May 23, 2025
• Study Start: August 20, 2025
• Primary Completion: August 29, 2025
• Study Completion: October 28, 2025
• Last Updated: April 28, 2026
• Results First Posted: April 28, 2026

Record last verified: 2026-02

Locations

CA (1)