Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases

Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases - HSCT for ataxia telangiectasia and related diseases

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000019532

Enrollment

Registered

2015-12-01

Start date

2015-12-01

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

ataxia telangiectasia DNA damage response disorder

Interventions

fludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Ataxia Telanogiectasia Nijmegen Syndrome LIG4 Syndrome Cernunnos deficiency PRKDC mutated SCID RIDDLE Syndrome other DDR associated diseases

Exclusion criteria

Exclusion criteria: multiple organs failure intra cranial hemorrhage heart failure renal failure uncontrolled infection uncontrolled diabetes lever cirrhosis serious psychological problem

Design outcomes

Primary

Measure	Time frame
overall survival for 1 year	—

Secondary

Measure	Time frame
percentage of adverse effect	—

Countries

Japan

Contacts

Public ContactKohsuke Imai

Tokyo Medical and Dental University Dept. of Community Pediatrics, Perinatal and Maternal Medicine

kimai.ped@tmd.ac.jp03-5803-4705

Outcome results

None listed

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