Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - Other
Status
Ongoing, recruiting
Participants planned
13
Countries
1
Sites
2
Phenylketonuria
Key facts
- Sponsor
- Agios Pharmaceuticals Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Trial duration
- 2 Jun 2026 → ongoing
- Decision date (initial)
- 2026-04-03
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
Trial design
CTIS Part I — objectives, methods, condition coding
Conditions and MedDRA coding
Phenylketonuria
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10034872 | Phenylketonuria | 100000004850 |
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Agios Pharmaceuticals Inc.
- Sponsor organisation
- Agios Pharmaceuticals Inc.
- Address
- 88 Sidney Street
- City
- Cambridge
- Postcode
- 02139-4137
- Country
- United States
Scientific contact point
- Organisation
- Agios Pharmaceuticals Inc.
- Contact name
- Scientific Communications
Public contact point
- Organisation
- Agios Pharmaceuticals Inc.
- Contact name
- Scientific Communications
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Poland | Ongoing, recruiting | 10 | 2 |
| Rest of world
United States
|
— | 3 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Poland | 2026-06-02 | 2026-06-02 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-11-28 | Poland | Acceptable 2026-03-30
|
2026-04-03 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2026-04-30 | Poland | Acceptable 2026-06-15
|
2026-06-18 |