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Clinical Trials

Date: 2022-05-16

Type of information: Clinical trial authorisation

phase: 1-2

Announcement: clinical trial authorization

Company: Atamyo Therapeutics (France)

Product: ATA-100

Action mechanism: gene therapy. ATA-100 is a single-administration gene therapy candidate that delivers a normal copy of the gene for production of fukutin-related protein (FKRP). LGMD2I/R9 is a rare genetic disease caused by mutations in the gene that produces FKRP. This disease affects an estimated 5,000 people in the US and Europe.

Disease: limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Country: France, Denmark, UK

Trial details: This file contains full details on each clinical trial selected for download. Where multi-state trials have been downloaded full information for each of the member states/countries involved in the trial are included separately. Summary EudraCT Number: 2021-004276-33 Sponsor's Protocol Code Number: ATA-001-FKRP National Competent Authority: Denmark - DHMA Clinical Trial Type: EEA CTA Trial Status: Ongoing Date on which this record was first entered in the EudraCT database: 2021-10-01 Link: https://www.clinicaltrialsregister.eu/ctr-search/trial/2021-004276-33/DK/ A. Protocol Information A.1 Member State Concerned: Denmark - DHMA A.2 EudraCT number: 2021-004276-33 A.3 Full title of the trial: A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I) A.3.1 Title of the trial for lay people, in easily understood, i.e. non-technical, language: GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophy A.4.1 Sponsor's protocol code number: ATA-001-FKRP A.7 Trial is part of a Paediatric Investigation Plan: No A.8 EMA Decision number of Paediatric Investigation Plan: B. Sponsor Information Sponsor 1 B.1.1 Name of Sponsor: Atamyo Therapeutics B.1.3.4 Country: France B.3.1 and B.3.2 Status of the sponsor: Commercial B.4 Source(s) of Monetary or Material Support for the clinical trial: B.4.1 Name of organisation providing support: Atamyo Therapeutics B.4.2 Country: France B.5 Contact point designated by the sponsor for further information on the trial B.5.1 Name of organisation: Atamyo Therapeutics B.5.2 Functional name of contact point: Sophie Olivier B.5.3 Address B.5.3.1 Street Address: 1bis, rue de l’Internationale B.5.3.2 Town/ city: Evry-Courcouronnes B.5.3.3 Post code: 91000 B.5.3.4 Country: France B.5.6 E-mail: s.olivier@atamyo.com D. IMP Identification D.IMP: 1 D.1.2 and D.1.3 IMP Role: Test D.2 Status of the IMP to be used in the clinical trial D.2.1 IMP to be used in the trial has a marketing authorisation: No D.2.5 The IMP has been designated in this indication as an orphan drug in the Community: No D.2.5.1 Orphan drug designation number: D.3 Description of the IMP D.3.1 Product name: rAAV9-hFKRPco_miR-208a D.3.2 Product code: GNT0006 D.3.4 Pharmaceutical form: Concentrate for solution for infusion D.3.4.1 Specific paediatric formulation: No D.3.7 Routes of administration for this IMP: Intravenous use D.3.8 to D.3.10 IMP Identification Details (Active Substances) D.3.8 INN - Proposed INN: not yet available D.3.9.2 Current sponsor code: GNT0006 D.3.9.3 Other descriptive name: Adeno-associated virus serotype 9 carrying the human fukutin-related protein and target sequence of the miR-208a D.3.9.4 EV Substance Code: SUB235392 D.3.10 Strength D.3.10.1 Concentration unit: vector genomes (vg)/mL D.3.10.2 Concentration type: not less then D.3.10.3 Concentration number: 1.7E+13 D.3.11 The IMP contains an D.3.11.1 Active substance of chemical origin: No D.3.11.2 Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP): No D.3.11.3 Advanced Therapy IMP (ATIMP): Yes D.3.11.3.1 Somatic cell therapy medicinal product: No D.3.11.3.2 Gene therapy medical product: Yes D.3.11.3.3 Tissue Engineered Product: No D.3.11.3.4 Combination ATIMP (i.e. one involving a medical device): No D.3.11.3.5 Committee on Advanced therapies (CAT) has issued a classification for this product: No D.3.11.4 Combination product that includes a device, but does not involve an Advanced Therapy: No D.3.11.5 Radiopharmaceutical medicinal product: No D.3.11.6 Immunological medicinal product (such as vaccine, allergen, immune serum): No D.3.11.7 Plasma derived medicinal product: No D.3.11.8 Extractive medicinal product: No D.3.11.9 Recombinant medicinal product: Yes D.3.11.10 Medicinal product containing genetically modified organisms: Yes D.3.11.11 Herbal medicinal product: No D.3.11.12 Homeopathic medicinal product: No D.3.11.13 Another type of medicinal product: No D.8 Information on Placebo D.8 Placebo: 1 D.8.1 Is a Placebo used in this Trial? Yes D.8.3 Pharmaceutical form of the placebo: Concentrate for solution for infusion D.8.4 Route of administration of the placebo: Intravenous use E. General Information on the Trial E.1 Medical condition or disease under investigation E.1.1 Medical condition(s) being investigated: FKRP-related limb-girdle muscular dystrophy (LGMD R9) E.1.1.1 Medical condition in easily understood language: FKRP-related limb-girdle muscular dystrophy (LGMD R9) E.1.1.2 Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] MedDRA Classification E.1.2 Medical condition or disease under investigation: E.1.2 Version: 20.0 E.1.2 Level: PT E.1.2 Classification code: 10028356 E.1.2 Term: Mu

Latest news:

Atamyo Therapeutics announced that the French National Medicines Health Agency (ANSM) granted a third authorization of a Clinical Trial Application (CTA) for ATA-100 in Europe . Two CTA approvals had previously been granted respectively by the United Kingdom Medicines & Healthcare products Regulatory Agency (MHRA) and by the Denmark Danish Medicines Agency (DKMA).
 
 

Is general: Yes