Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14

Orpha Labs

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Glycogen Storage Disease, Type 14

Treatments: Drug: ORL-1G - D-galactose

Study type: Interventional

Funder types: Industry

Identifiers

NCT03404856

GALA-1

Details and patient eligibility

About

Study of ORL-1G in Patients With Glycogen Storage Disease Type 14

Enrollment: 5 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of Glycogen Storage Disease Type 14.
• Less than 18 years old.

Exclusion criteria

• Diagnosis of any other disease that is not a manifestation of Glycogen Storage Disease Type 14.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Treatment with ORL-1G - D-galactose

Other group

Treatment:

Drug: ORL-1G - D-galactose