First in Human Study to Test the Safety and Preliminary E... | NCT04568174

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

NCT04568174

Sponsor

Polyneuron Pharmaceuticals AG

Status

Terminated

Last Update Posted

Oct 15, 2021Actual

Enrollment

1Actual

Phase

Phase 1Phase 2

Conditions

Anti-MAG Neuropathy

Interventions

PPSGG
Placebo

Countries

France
Netherlands
Spain
Switzerland
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Oct 2021

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Data don't support further development

Expanded Access Info

Start Date

Nov 17, 2020Actual

Primary Completion Date

Sep 23, 2021Actual

Completion Date

Sep 23, 2021Actual

First Submitted Date

Sep 8, 2020

First Submission Date that Met QC Criteria

Sep 23, 2020

First Posted Date

Sep 29, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Oct 7, 2021

Last Update Posted Date

Oct 15, 2021Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

PPSGG (PN-1007) is intended to bind anti-MAG IgM autoantibodies, the underlying cause of anti-MAG neuropathy, in a highly selective manner, resulting in their neutralization and removal from the circulation. This allows specific targeting of anti-MAG IgM in the circulation and circumvents unspecific immunosuppression associated with current treatment strategies.

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

1Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
PPSGG	Drug	an antibody scavenger of pathogenic anti-MAG immunoglobulin M (IgM) autoantibodies	PPSGG

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
Tmax	Time of peak concentration of PPSGG (PN-1007)	Day 1 to Day 42
Cmax	Maximum Plasma Concentration of PPSGG (PN-1007)	Day 1 to Day 42

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

80 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Hedvika Lazar	Polyneuron Pharmaceuticals AG	Study Chair

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Service de Neurologie Centre de Référence Neuropathies Périphériques Rares, CHU Limoges		Limoges		87 042	France

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D010523	Peripheral Nervous System Diseases

Ancestor Terms

ID	Term
D009468	Neuromuscular Diseases
D009422	Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Masking Info

Masking

Triple

Masking Description

The SAD phase is open label and MAD is randomized, dose escalation, double blind (patient and investigator blinded), placebo-controlled

Who Masked

ParticipantInvestigatorOutcomes Assessor