An Open-Label Extension to Study the Long-Term Effect of Dexpramipexole in Participants with Severe Eosinophilic Asthma (EXHALE-5)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Areteia Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

14 Oct 2024 → ongoing

Decision date (initial)

2024-09-24

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Areteia Therapeutics Inc.

External identifiers

EU CT number

2024-510810-33-00

WHO UTN

U1111-1305-0829

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the long-term safety of dexpramipexole 150 mg BID in participants who completed either of the Phase III studies EXHALE-2 or EXHALE-3.

Secondary objectives 2

1. To assess the long-term efficacy of Dexpramipexole in participants with eosinophilic asthma who completed either of the Phase III studies EXHALE-2 or EXHALE-3.
2. To evaluate the effect of Dexpramipexole on blood eosinophils.

Conditions and MedDRA coding

Severe eosinophilic asthma

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003328-PIP01-22

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Participants who completed the double-blind study intervention treatment period during either of the Phase III studies EXHALE-2 or EXHALE-3.
2. Signed informed consent form (ICF) and assent form as appropriate.
3. Negative urine pregnancy test for women of childbearing potential (WOCBP; after menarche) at the Baseline Visit/Day 1.
4. WOCBP (after menarche) must use either of the following methods of birth control, from the Baseline Visit/Day 1 through the End of Study Visit: a. A highly effective form of birth control (confirmed by the investigator). Highly effective forms of birth control include: true sexual abstinence, a vasectomized sexual partner, Implanon, female sterilization by tubal occlusion, any effective intrauterine device (IUD), IUD/intrauterine system, levonorgestrel intrauterine system, or oral contraceptive. Or b. Two protocol acceptable methods of contraception in tandem. Women not of childbearing potential are defined as women who are either permanently sterilized (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy), or who are postmenopausal. Women will be considered postmenopausal if they have been amenorrheic for ≥12 months prior to the planned date of the Baseline Visit/Day 1 without an alternative medical cause. The following age specific requirements apply: c. Women <50 years old will be considered postmenopausal if they have been amenorrheic for 12 months or more following cessation of exogenous hormonal treatment and follicle stimulating hormone levels in the postmenopausal range. d. Women ≥50 years old will be considered postmenopausal if they have been amenorrheic for 12 months or more following cessation of all exogenous hormonal treatment.

Exclusion criteria 6

1. Clinically significant change in health status during either of the Phase III studies EXHALE-2 or EXHALE-3 which in the opinion of the investigator would make the participant unsuitable for participation in this study.
2. Participants with allergy/intolerance to dexpramipexole or any of its excipients.
3. Participants who met permanent discontinuation criteria or permanently discontinued study treatment for any reason in either of the Phase III studies EXHALE-2 or EXHALE-3.
4. Treatment with selected drugs known to have a substantial risk of neutropenia in the past 30 days
5. Pregnant or breastfeeding women.
6. Males who are unwilling to use an acceptable method of birth control during the entire study period (ie, condom with spermicide).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Assessment of treatment emergent AEs (TEAEs), clinical laboratory parameters, vital signs, and 12-lead ECGs.

Secondary endpoints 3

1. Annualized rate of severe asthma exacerbations over 52 weeks.
2. Asthma Control Questionnaire-6 (ACQ-6), change from baseline, at Weeks 16, 32, and 52.
3. Absolute eosinophil counts (AEC), change from baseline, at Weeks 16, 32, and 52.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Areteia Therapeutics Inc.

Sponsor organisation

Areteia Therapeutics Inc.

Address

101 Glen Lennox Drive Suite 300 Suite 3005

City

Chapel Hill

Postcode

27517-4086

Country

United States

Scientific contact point

Organisation

Areteia Therapeutics Inc.

Contact name

Andrew Friedman

Public contact point

Organisation

Areteia Therapeutics Inc.

Contact name

Andrew Friedman

Third parties 13

Locations

7 EU/EEA countries · 43 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

Documents 65 files

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

Application history

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