A Trial of Hydroxyurea in Spinal Muscular Atrophy

Plain-language summary

This study looks at the use of Hydroxyurea in spinal muscular atrophy. Patients older than 4 with a homozygous deletion of the SMN1 gene who still carried the SMN2 gene were able to take part. Sponsored by Kaohsiung Medical University Chung-Ho Memorial Hospital, the study was conducted in Phase 2/Phase 3 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE2/PHASE3

Status

Completed

Sponsor

Kaohsiung Medical University Chung-Ho Memorial Hospital

Intervention

Hydroxyurea

Start date

6/1/2007

Estimated completion

6/1/2009

Countries

Taiwan

Related genes

Locations

• Kaohsiung City, Taiwan

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Sixty SMA patients fulfilled the diagnostic criteria of SMA as defined by the International SMA Consortium (Munsat, 1992), and all had homozygous deletion of SMN1 gene but carried SMN2 gene (Chang, 1995; 1997)
* The subjects shall each have the age older than 4 years.
* The subjects (if age \> 20 years old) or their parents (if subject age \< 20 years old) shall agree with the trial and have signed the informed consents.

Exclusion Criteria:

* The subjects shall not have hematological diseases, other severe systemic diseases and congenital anomalies except for SMA.
* The subjects shall not have severe perinatal asphyxia history.
* The subjects shall not be with respiratory assists.
* The subjects shall have normal hepatic and renal functions in the pre-trial examination.
* The subjects shall not have received operation with generalized anesthesia in past half a year.
* The subjects shall not have acceded to other clinical trials in past half a year.
* The subjects shall not get in this trial if they could not complete the course.