A Long term Safety and Efficacy Study of Danicopan as an Add-on Therapy to C5i in Patients with PNH.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Alexion Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

5 Dec 2022 → ongoing

Decision date (initial)

2024-07-08

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Alexion Pharmaceuticals, Inc.

External identifiers

EU CT number

2023-504867-18-00

EudraCT number

2021-004253-22

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy

To characterize the long-term safety of treatment with danicopan as an add-on therapy to a complement component 5 inhibitor (C5i)

Secondary objectives 3

1. To characterize long-term efficacy of danicopan as an add-on therapy to a C5i
2. To characterize the long-term effect of treatment with danicopan as an add-on therapy to a C5i on Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue scores and on European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire-Core 30 Scale (EORTC-QLQ-C30) scores
3. To further characterize the safety of danicopan as an add-on therapy to a C5i

Conditions and MedDRA coding

Patients with Paroxysmal Nocturnal Hemoglobinuria

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment
2. Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.

Exclusion criteria 2

1. Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant.
2. Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs

Secondary endpoints 10

1. Change in hemoglobin (Hgb) values over time
2. Proportion of patients with Hgb increase of ≥ 2g/dL in the absence of transfusion over time
3. Change in absolute reticulocyte count over time
4. Change in lactate dehydrogenase (LDH) over time
5. Proportion of patients with LDH ≤ 1.5 × upper limit of normal (ULN) over time
6. Proportion of patients with transfusion avoidance (TA), defined as patients who remain transfusion free and do not require transfusion as per protocol specified guidelines
7. Change in FACIT Fatigue scores over time
8. Change in EORTC-QLQ-C30 scores over time
9. Change in safety laboratory parameters over time
10. TEAEs leading to discontinuation

Investigational products

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

Test 1

Auxiliary 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Alexion Pharmaceuticals Inc.

Sponsor organisation

Alexion Pharmaceuticals Inc.

Address

121 Seaport Boulevard

City

Boston

Postcode

02210-2050

Country

United States

Scientific contact point

Organisation

Alexion Pharmaceuticals Inc.

Contact name

European Clinical Trial Information

Public contact point

Organisation

Alexion Pharmaceuticals Inc.

Contact name

European Clinical Trial Information

Third parties 6

Locations

6 EU/EEA countries · 18 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 47 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