Use of Idursulfase by a specific Patient: Alternative Access to Treatment among Patients with Selected MPS II who participate in the study TKT024EXT

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

REPEC

Registry ID

PER-033-06

Enrollment

Registered

2006-07-18

Start date

2006-09-11

Completion date

Unknown

Last updated

2023-09-04

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

Conditions

None listed

Interventions

ONLY GROUP Type of group

Patients will be treated with idursulfase, at a dose of 0.5 mg / kg, IV in a continuous infusion for 3 hours, once a week for 2 years.

Eligibility

Inclusion criteria

Inclusion criteria: Patients who are currently participating in the TKT024EXT Study and who meet the entry criteria described below are eligible to participate in this protocol: 1) Patients for whom the requirements of the TKT024EXT Study are considered to be very onerous. 2) Patients should not have experienced an adverse reaction to idursulfase, which would contraindicate subsequent treatment with idursulfase.

Exclusion criteria

Exclusion criteria: The same as the protocol TKT024EXT.

Design outcomes

Primary

Measure	Time frame
Outcome name:Clinical Assessment of Adverse Events (AA): Which will be defined by any of the following: 1) Worsening of the conditions present at the beginning of the study. 2) Intercurrent diseases. 3) Drug interactions. 4) Events related or possibly related to concomitant medications. 5) Alteration of laboratory analysis values. 6) Clinically significant abnormalities in physical examination, vital signs, weight and ECG. In addition to cataloging the AA according to their relationship with the treatment and their severity. Measure:Safety of the treatment. Timepoints:30 days after the patient s last infusion of idursulfase under this protocol, whether the patient has completed the 2-year treatment period or has abandoned their participation in advance.	—

Measure

Time frame

Outcome name:Clinical Assessment of Adverse Events (AA): Which will be defined by any of the following: 1) Worsening of the conditions present at the beginning of the study. 2) Intercurrent diseases. 3) Drug interactions. 4) Events related or possibly related to concomitant medications. 5) Alteration of laboratory analysis values. 6) Clinically significant abnormalities in physical examination, vital signs, weight and ECG. In addition to cataloging the AA according to their relationship with the treatment and their severity. Measure:Safety of the treatment. Timepoints:30 days after the patient s last infusion of idursulfase under this protocol, whether the patient has completed the 2-year treatment period or has abandoned their participation in advance.

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Countries

Brazil, Germany, Peru, United Kindgdom, United States

Outcome results