Phase 3 clinical study of STM-279 in patients with adenosine deaminase (ADA) deficiency

Multicenter, open-label, uncontrolled clinical study of STM-279 in patients with adenosine deaminase (ADA) deficiency (Phase III clinical study)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080223149

Enrollment

Registered

2016-03-22

Start date

2016-03-02

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

ADA deficiency

Interventions

investigational material(s) Generic name etc : Elapegademase (Genetical Recombination) INN of investigational material : elapegademase Therapeutic category code : 639 Other biological preparations Dos

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Main Inclusion Criteria (1) Patients diagnosed with ADA deficiency (2) Patients who need the enzyme therapy according to the investigator's judgment

Exclusion criteria

Exclusion criteria: Main Exclusion Criteria (1) Patients with severe thrombocytopenia(platelet count less than 50,000,000,000/L) (2) Female patients who are pregnant or lactating (3) Female patients of childbearing potential who are unwilling to practice adequate contraception by use of a condom from the time of obtaining informed consent to the completion of the continuous administration (extension) phase

Design outcomes

Primary

Measure	Time frame
other -	—

Secondary

Measure	Time frame
safety efficacy pharmacokinetics Efficacy:deoxyadenosine nucleotide(dAXP), ADA activity, Immune status, Body height and body weight Safety:Adverse events, Laboratory data, Vital signs Pharmacokinetics	—

Countries

Japan

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026