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A study to investigate the long-term safety and efficacy of a new drug, NI-0501, in children with a disease that is called Haemophagocytic Lymphohistiocytosis.

Phase 1
Conditions
Haemophagocytic lymphohistiocytosis
MedDRA version: 21.1 Level: PT Classification code 10071583 Term: Haemophagocytic lymphohistiocytosis System Organ Class: 10021428 - Immune system disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Registration Number
EUCTR2012-005753-23-GB
Lead Sponsor
Swedish Orphan Biovitrum AG
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
Not specified
Target Recruitment
55
Inclusion Criteria

1. Having received at least one dose of NI-0501 during a previous NI-0501 study or under a CU treatment protocol
2. Informed Consent by the patient or the patient’s legal representative(s), as applicable, with the assent of patients who are legally capable of providing it
3. Having agreed to continue using adequate methods of birth control until 6 months after the last administered dose of NI-0501, whenever relevant. Males with partners(s) of child-bearing potential must agree to take appropriate precautions to avoid fathering a child until 6 months after receiving last dose of NI-0501.

Are the trial subjects under 18? yes
Number of subjects for this age range: 55
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

None

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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