Study identification

PURI

https://redirect.ema.europa.eu/resource/11889

EU PAS number

EUPAS3750

Study ID

11889

Official title and acronym

Glybera Registry, long-term safety and efficacy follow-up in Lipoprotein Lipase deficient (LPLD) patients treated with alipogene tiparvovec (GLYBERA®)

DARWIN EU® study

No

Study countries

Canada
Germany
Italy
Netherlands

Study description

The Glybera Registry is an international, prospective, non-interventional, longitudinal, observational PASS open to patients with genetically confirmed LPLD who had been treated with GLYBERA and participated in the LPLD Registry. The Glybera Registry was a continuation of this LPLD Registry, albeit with fewer assessments and decreased burden to the patient, if any. Physicians collected data from participating patients during their routine care, i.e. at entry in the LPLD Registry, on regular intervals during the first 2 years after dosing and subsequently once every 2 years, up to 15 years, or until the patients was lost to follow-up.

Study status

Finalised
Research institutions and networks

Institutions

Contact details

Elisabeth Steinhagen-Thiessen

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:

Study start date

Planned:
Actual:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

uniQure biopharma B.V.
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

Not applicable