Open-Label Extension of EryDex in A-T (OLE NEAT)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Quince Therapeutics S.p.A.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Nervous System Diseases [C10]

Trial duration

23 Dec 2024 → 31 Jan 2026

Decision date (initial)

2024-10-23

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Quince Therapeutics, S.p.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

Safety Objective: To evaluate the safety and tolerability of eDSP in participants with A-T.
Exploratory Objective: To evaluate the effects of eDSP on neurological symptoms in participants with A-T.

Conditions and MedDRA coding

Ataxia Telangiectasia (A-T)

Study design 1 period

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Males and females ≥ 6 years of age, with body weight ≥15 kg, who completed the IEDAT-04-2022 trial, including final efficacy assessments (Visit 9).
2. Participant’s body weight ≥15 kg.
3. Participant does not meet any of the criteria for treatment discontinuation and does not present safety contraindications for continuation of treatment as determined by the investigator.
4. In the investigator’s opinion, the potential benefit of receiving the investigational treatment in the OLE trial outweighs the potential risks of receiving the investigational treatment in the OLE trial.

Exclusion criteria 8

1. Females who are pregnant, or breastfeeding. Females of childbearing potential using adequate birth control as determined by their healthcare provider will be eligible.
2. Participant has clinically significant immune impairment that, in the opinion of the investigator, precludes further treatment with corticosteroids.
3. Participant has confirmed hemoglobinopathies, e.g., hemoglobin C disease, sickle cell anemia, hereditary spherocytosis, or thalassemia.
4. Participants with suicidal ideation.
5. Participant requires treatment with a systemic corticosteroid. Treatment with inhaled or intranasal corticosteroids for asthma or allergies, as well as use of topical corticosteroids will be permitted.
6. Participant requires any concomitant medication prohibited by the protocol, including strong inducers or inhibitors of cytochrome P450 3A4 (CYP3A4).
7. Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
8. Current participation in another clinical trial. Participation in observational, non-interventional studies is allowed as long as trial investigational endpoint raters can remain blinded to the assessments from other studies, and as long as the other trial participation does not interfere with participation in this trial.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety endpoint : assessment of treatment-emergent adverse events (TEAEs), including serious AEs, discontinuations due to AEs, changes in vital signs, laboratory parameters, physical and neurological examination findings, and the C-SSRS. Additionally, potential effects associated with the use of eDSP will be assessed by changes in cortisol, glucose, cluster of differentiation CD4+ lymphocyte count, bone mineral density obtained using energy x-ray absorptiometry (DEXA), and growth and development

Secondary endpoints 1

1. Exploratory Endpoint Assessments: Evaluation of the effects of eDSP will be assessed by the International Cooperative Ataxia Rating Scale (ICARS), the modified ICARS (mICARS), and the Rescored mICARS (RmICARS).

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Quince Therapeutics S.p.A.

Sponsor organisation

Quince Therapeutics S.p.A.

Address

Via Antonio Meucci 3

City

Bresso

Postcode

20091

Country

Italy

Scientific contact point

Organisation

Quince Therapeutics S.p.A.

Contact name

Dirk Thye

Public contact point

Organisation

Quince Therapeutics S.p.A.

Contact name

Dirk Thye

Third parties 1

Locations

6 EU/EEA countries · 11 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 115 files

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

Application history

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