A study to learn more about the long-term safety and efficacy of AB-1005 gene therapy in people with Parkinson’s disease or multiple system atrophy

2025-522653-19-00 Protocol ASK-PD0-CS002 Therapeutic use (Phase IV) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 2 sites · Protocol ASK-PD0-CS002

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)
Status Authorised, recruitment pending
Participants planned 130
Countries 1
Sites 2

Multiple System Atrophy

To evaluate the long-term safety and tolerability of AB-1005 in participants with Parkinson's Disease (PD) or Multiple System Atrophy-Parkinsonian subtype (MSA-P)

Key facts

Sponsor
Askbio Inc.
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10]
Decision date (initial)
2025-12-22
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
No
Funding sources
AskBio Inc.

External identifiers

EU CT number
2025-522653-19-00
ClinicalTrials.gov
NCT07081841

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To evaluate the long-term safety and tolerability of AB-1005 in participants with Parkinson's Disease (PD) or Multiple System Atrophy-Parkinsonian subtype (MSA-P)

Secondary objectives 1

  1. To evaluate the long-term efficacy of AB-1005 in participants with PD or MSA-P

Conditions and MedDRA coding

Multiple System Atrophy

Regulatory references

Plan to share IPD
No
EU CT numberTitleSponsor
2023-506519-16-00 (ASK-PD5-CS201) A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults with Moderate Stage Parkinson’s Disease Asklepios Biopharmaceutical Inc.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

  1. PD and MSA-P participants that are or were enrolled in an interventional AB-1005 study and provide informed consent.

Exclusion criteria 1

  1. There are no exclusion criteria for this study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Incidence and severity of AEs (serious and non-serious)

Secondary endpoints 9

  1. For PD participants - Normalized PD motor diary times (Good ON, ON with troublesome dyskinesia, OFF)
  2. For PD participants - MDS-UPDRS part III (OFF and ON)
  3. For PD participants - MDS-UPDRS part II (OFF and ON)
  4. For PD participants - MDS-UPDRS part I
  5. For PD participants - MDS-UPDRS part IV
  6. For PD participants - Levodopa equivalent daily dose (LEDD)
  7. For PD participants - 18F-DOPA PET
  8. For MSA participants - UMSARS parts I, II (ON), III, IV
  9. For MSA participants - MSA-QoL

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

AAV2-GDNF

PRD11008558 · Product

Active substance
Adeno-Associated Viral Vector Serotype 2 Encoding Glial Cell Line-Derived Neurotrophic Factor
Substance synonyms
Adeno-associated viral vector serotype 2 encoding GDNF, AAV2-GDNF
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAPUTAMINAL USE
Max daily dose
12000000000000 Other
Max total dose
12000000000000 Other
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
MA holder
ASKLEPIOS BIOPHARMACEUTICAL INC.
Paediatric formulation
No
Orphan designation
No

Auxiliary 2

Fluorodopa (18F)

PRD11201945 · Product

Active substance
Fluorodopa (18F)
Other product name
FDOPA
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRAVENOUS
Max daily dose
222 MBq megabecquerel(s)
Max total dose
888 MBq megabecquerel(s)
Max treatment duration
4 Day(s)
Authorisation status
Not Authorised
ATC code
V09IX05 — -
MA holder
ASKLEPIOS BIOPHARMACEUTICAL INC.
Paediatric formulation
No
Orphan designation
No

Carbidopa

PRD11202056 · Product

Active substance
Carbidopa
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
200 mg milligram(s)
Max total dose
200 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Not Authorised
ATC code
N04BA02 — LEVODOPA AND DECARBOXYLASE INHIBITOR
MA holder
ASKLEPIOS BIOPHARMACEUTICAL INC.
Paediatric formulation
No
Orphan designation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Askbio Inc.

3 Total trials 1 Ended
Commercial
Sponsor organisation
Askbio Inc.
Address
20 Tw Alexander Drive Suite 110
City
Durham
Postcode
27713-2928
Country
United States

Scientific contact point

Organisation
Askbio Inc.
Contact name
Alexander Neumeister

Public contact point

Organisation
Askbio Inc.
Contact name
Alexander Neumeister

Third parties 1

OrganisationCity, countryDuties
Bayer AG
ORG-100000011
 Berlin, Germany On site monitoring, Code 10, Code 11, Code 12, Code 13, Code 5, Data management, E-data capture, Code 8, Code 9

Sponsor responsibilities

Article 77 compliance
Askbio Inc.
Contact point sponsor
Askbio Inc.
Article 77 implementation
Askbio Inc.

Locations

1 EU/EEA country · 2 investigational sites

By country

CountryMS statusPlanned subjectsSites
Poland Authorised, recruitment pending 40 2
Rest of world
United States
 — 90 —

Investigational sites

Poland

2 sites · Authorised, recruitment pending
Copernicus Podmiot Leczniczy Sp. z o.o.
Oddział Neurologiczny i Udarowy, Al. Jana Pawla II 50, 80-462, Gdansk
Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala
Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala, ul. Fitelberga 34/1, 40-588, Katowice

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol_EN_2025-522653-19-00_public 3
Recruitment arrangements (for publication) K1_Recruitment arrangements_Public_PL_PL_Procedure Template 2
Subject information and informed consent form (for publication) L1_ICF_PL_PL_public 2-1
Synopsis of the protocol (for publication) D1_Synopsis of Protocol_EN_2025-522653-19-00_public 1
Synopsis of the protocol (for publication) D1_Synopsis of Protocol_PL_PL_2025-522653-19-00_public 1

Application history

3 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-08-26 Poland Acceptable
2025-12-15
 2025-12-22
2 SUBSTANTIAL MODIFICATION SM-1 2026-01-29 Poland Acceptable
2026-03-16
 2026-03-23
3 NON SUBSTANTIAL MODIFICATION NSM-1 2026-03-26 Poland Acceptable
2026-03-16
 2026-03-26

Related clinical trials