NCT02904772

An Open Label, Multi-centre Trial of Alipogene Tiparvovec for the Treatment of LPLD Patients

Withdrawn Phase 2 Last updated 14 August 2017

What this trial tests

Phase 2 trial testing alipogene tiparvovec in LPL Deficiency. Withdrawn.

Timeline

1 October 2016

Primary endpoint
1 June 2020

1 September 2020

Quick facts

Lead sponsor	UniQure Biopharma B.V.
Phase	Phase 2
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Start date	1 October 2016
Primary completion	1 June 2020
Estimated completion	1 September 2020
Sites	2 locations across United States, Canada

Drugs / interventions tested

• alipogene tiparvovec (ALIPOGENE TIPARVOVEC) — full drug profile →
• Prednisolone — full drug profile →
• Cyclosporins — full drug profile →
• Mycophenolate mofetil (MYCOPHENOLATE MOFETIL) — full drug profile →

Conditions studied

• LPL Deficiency — all drugs for LPL Deficiency →

Sponsor

UniQure Biopharma B.V. — full company profile →

Who can join

18 and older, any sex, with LPL Deficiency. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• The Clinical Response of alipogene tiparvovec in LPLD patients
  Time frame: 2 years
  The overall clinical response of alipogene tiparvovec in LPLD patients will be assessed compared to baseline, by a combination of measurements, of which each gives relevant information to obtain enough and solid evidence in a small trial. Each of these outcome measures will be evaluated in combination with the results of other measures (to get an overall conclusion relating the clinical response).
• The long term effect of alipogene tiparvovec on post prandial metabolism of chylomicrons (ppCM) in LPLD patients.
  Time frame: 2 years
  CM \[3H\]-activity will be assessed during ppCM testing pre- and post-dose, compared to baseline.

Sponsor's own description

The aim of the study is to provide further confirmatory evidence of clinical benefit in LPLD patients treated with alipogene tiparvovec by assessing both the "clinical response" (as defined by a range of parameters), and "the metabolic response" (postprandial CM metabolism) in LPLD patients with and without an immunosuppressant regimen.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. A versatile toolkit for overcoming AAV immunity.
   Li X, Wei X, Lin J, Ou L. · · 2022 · cited 31× · PMID 36119036 · DOI 10.3389/fimmu.2022.991832
2. A Novel APOC2 Missense Mutation Causing Apolipoprotein C-II Deficiency With Severe Triglyceridemia and Pancreatitis.
   Ueda M, Dunbar RL, Wolska A, Sikora TU, et al · · 2017 · cited 30× · PMID 28201738 · DOI 10.1210/jc.2016-3903
3. The significance of triple-capsid-mutant AAV8 for treatment of Sanfilippo Syndrome Type B.
   Ashby F, Heldermon C. · · 2022 · cited 2× · PMID 38596745 · DOI 10.46439/stemcell.3.013