First in Human Study to Test the Safety and Preliminary Efficacy of PPSGG in Patients With Anti-MAG Neuropathy
NCT ID: NCT04568174
Last Updated: 2021-10-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE1/PHASE2
1 participants
INTERVENTIONAL
2020-11-17
2021-09-23
Brief Summary
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This is the first research of PPSGG (PN-1007) in people and its main purpose is to test its safety and acceptability in patients. In this study it will be examined how the drug is changed by and removed from the body and checked for signs that the drug may be truly effective against anti-MAG neuropathy. PPSGG (PN-1007) will be tested at several different doses.
Detailed Description
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This is a Phase I/IIa, First in Human (FiH), multicenter, single and multiple ascending dose escalation trial of PPSGG (PN-1007), an antibody scavenger of pathogenic anti-MAG immunoglobulin M (IgM) autoantibodies for treatment of anti-MAG neuropathy. The aim of the study is to assess the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of PPSGG (PN-1007) in a SAD and a MAD phase in an adaptive trial in anti-MAG neuropathy patients.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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PPSGG
sterile liquid, one 1-hour infusion in SAD and multiple infusions in MAD. In SAD multiple cohorts being tested. Dosage and regime in MAD to be defined based on SAD outcome.
PPSGG
an antibody scavenger of pathogenic anti-MAG immunoglobulin M (IgM) autoantibodies
Placebo
standard PBS solution, pH 7.4, composed of disodium hydrogen phosphate dodecahydrate, potassium dihydrogen phosphate, sodium chloride, and water for injection
Placebo
A standard PBS solution, pH 7.4, composed of disodium hydrogen phosphate dodecahydrate, potassium dihydrogen phosphate, sodium chloride, and water for injection
Interventions
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PPSGG
an antibody scavenger of pathogenic anti-MAG immunoglobulin M (IgM) autoantibodies
Placebo
A standard PBS solution, pH 7.4, composed of disodium hydrogen phosphate dodecahydrate, potassium dihydrogen phosphate, sodium chloride, and water for injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Clear clinical signs of disability
* Adequate hepatic and renal function
Exclusion Criteria
* Hematological malignancy, prior malignancy of any organ system (except BCC)
* Prior immunosuppression: No IVIG in previous 3 months, no previous cyclophosphamide or biologicals in prior 6 months.
* Other neurological, neuromuscular, rheumatologic or orthopedic condition with significant impact on the capabilities of walk preventing evaluation of neurological scores
18 Years
80 Years
ALL
No
Sponsors
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Polyneuron Pharmaceuticals AG
INDUSTRY
Responsible Party
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Principal Investigators
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Hedvika Lazar
Role: STUDY_CHAIR
Polyneuron Pharmaceuticals AG
Locations
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Service de Neurologie Centre de Référence Neuropathies Périphériques Rares, CHU Limoges
Limoges, , France
Referral centre for neuromuscular diseases and ALS, hôpital La Timone
Marseille, , France
Département de Neurologie Pôle Neurosciences Centre de Référence des Neuropathies Amyloïdes Familiales et autres Neuropathies Périphériques Rares Centre Hospitalier Universitaire de Bicêtre
Paris, , France
UMC Utrecht Cancer Center
Utrecht, , Netherlands
Barcelona
Barcelona, , Spain
Lausanne
Lausanne, , Switzerland
National hospital for neurology and neurosurgery, Queen London
London, , United Kingdom
Countries
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Other Identifiers
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PN-1007-001
Identifier Type: -
Identifier Source: org_study_id