First in Human Study to Test the Safety and Preliminary Efficacy of PPSGG in Patients With Anti-MAG Neuropathy

NCT04568174 | Terminated Phase 1 DMC

• 1 other identifier: PN-1007-001
• Study Type: interventional
• Target: 1
• Locations: 5 countries
• Sites: 7

Brief Summary

In this study, the new drug called PPSGG (PN-1007) will be tested. Preliminary studies conducted in animals suggest PPSGG (PN-1007) might be a good treatment for reducing levels of anti-MAG antibodies in patients with anti-MAG neuropathy. 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.

Conditions

Anti-MAG Neuropathy

Keywords

peripheral neuropathy

Outcome Measures

Primary Outcomes (2)

• Adverse Events (AEs) and Serious Adverse Events (SAEs)

  All AEs will be recorded, whether considered minor or serious, drug-related or not

  1 month

• anti-drug-antibodies ADA

  Potential ADAs (immunogenicity) resulting from exposure of patients to PPSGG (PN-1007) will be measured by ELISA

  1 month in SAD

Secondary Outcomes (6)

• Tmax

  Day 1 to Day 42

• Cmax

  Day 1 to Day 42

• AUCinf

  Day 1 to Day 42

• t1/2

  Day 1 to Day 42

• Pharmacodynamic

  up to Day 28

Study Arms (2)

PPSGG

ACTIVE COMPARATOR

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.

Drug: PPSGG

Placebo

PLACEBO COMPARATOR

standard PBS solution, pH 7.4, composed of disodium hydrogen phosphate dodecahydrate, potassium dihydrogen phosphate, sodium chloride, and water for injection

Drug: Placebo

Interventions

PPSGG DRUG

an antibody scavenger of pathogenic anti-MAG immunoglobulin M (IgM) autoantibodies

Also known as: PN-1007

PPSGG

Placebo DRUG

A standard PBS solution, pH 7.4, composed of disodium hydrogen phosphate dodecahydrate, potassium dihydrogen phosphate, sodium chloride, and water for injection

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with a confirmed diagnosis of monoclonal IgM associated with MGUS with anti-MAG activity (titer of \> 10'000 BTU) and demyelinating neuropathy defined by electrophysiological criteria according to EFNS/PNS PDN guideline, 2010.
• Clear clinical signs of disability
• Adequate hepatic and renal function

You may not qualify if:

• Patients with total serum IgM levels \>30 g.
• 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

Sponsors & Collaborators

• Polyneuron Pharmaceuticals AG: lead

Study Sites (7)

Service de Neurologie Centre de Référence Neuropathies Périphériques Rares, CHU Limoges

Limoges, 87 042, France

Location

Referral centre for neuromuscular diseases and ALS, hôpital La Timone

Marseille, 13385, France

Location

Département de Neurologie Pôle Neurosciences Centre de Référence des Neuropathies Amyloïdes Familiales et autres Neuropathies Périphériques Rares Centre Hospitalier Universitaire de Bicêtre

Paris, 94275, France

Location

UMC Utrecht Cancer Center

Utrecht, Netherlands

Location

Barcelona

Barcelona, Spain

Location

Lausanne

Lausanne, Switzerland

Location

National hospital for neurology and neurosurgery, Queen London

London, WC1N 3bg, United Kingdom

Location

MeSH Terms

Conditions

Peripheral Nervous System Diseases

Condition Hierarchy (Ancestors)

Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Hedvika Lazar

  Polyneuron Pharmaceuticals AG

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The SAD phase is open label and MAD is randomized, dose escalation, double blind (patient and investigator blinded), placebo-controlled
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single Group in SAD and parallel in MAD

Study Record Dates

• First Submitted: September 8, 2020
• First Posted: September 29, 2020
• Study Start: November 17, 2020
• Primary Completion: September 23, 2021
• Study Completion: September 23, 2021
• Last Updated: October 15, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1); NL (1); FR (3); ES (1); GB (1)