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NCT ID: NCT03153462 Approved for marketing - Clinical trials for Relapsed/Refractory Diffuse Large B Cell Lymphoma

Axicabtagene Ciloleucel Expanded Access Study

ZUMA-9
Start date: n/a
Phase:
Study type: Expanded Access

A multicenter, open-label expanded access protocol for the treatment of subjects with relapsed/refractory large B-cell lymphoma. Subjects who received an infusion of axicabtagene ciloleucel will complete the remainder of the 15 year follow-up assessments in a separate long-term follow-up study, KT-US-982-5968

NCT ID: NCT03154437 Approved for marketing - Hemophilia A Clinical Trials

An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors

Start date: n/a
Phase:
Study type: Expanded Access

This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.

NCT ID: NCT03245424 Approved for marketing - Clinical trials for Acute Myeloid Leukemia

Ivosidenib Expanded Access Program in Relapsed/Refractory AML With an IDH1 Mutation

Start date: n/a
Phase:
Study type: Expanded Access

To provide access to ivosidenib monotherapy to patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation.

NCT ID: NCT03245528 Approved for marketing - Sepsis Clinical Trials

Expanded Access for LJPC-501

Start date: n/a
Phase: N/A
Study type: Expanded Access

The primary objective of the study is to provide access to LJPC-501 for distributive shock patients who remain hypotensive despite receiving fluid and vasopressor therapy.

NCT ID: NCT03259633 Approved for marketing - Clinical trials for NF type1 With Inoperable Plexiform Neurofibromas

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

Start date: n/a
Phase:
Study type: Expanded Access

This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit. Approximately 100 patients in the US will be treated as part of this protocol

NCT ID: NCT03278314 Approved for marketing - Cystic Fibrosis Clinical Trials

Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

Start date: n/a
Phase:
Study type: Expanded Access

To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719). To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of age and older, homozygous for F508del.

NCT ID: NCT03400098 Approved for marketing - Clinical trials for Amyloidosis, Hereditary

ATTR Expanded Access Program (EAP) by Ionis

Start date: n/a
Phase:
Study type: Expanded Access

The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).

NCT ID: NCT03501615 Approved for marketing - Clinical trials for Relapsed/Refractory Hairy Cell Leukemia

An Early Access Programme for Moxetumomab Pasudotox in Relapsed/Refractory Hairy Cell Leukemia

MOXE
Start date: n/a
Phase:
Study type: Expanded Access

Early Access Programme to provide treatment access to moxetumomab pasudotox for eligible patients with relapsed/refractory hairy cell leukemia

NCT ID: NCT03533361 Approved for marketing - Thyroid Cancer Clinical Trials

Expanded Access Program With Lenvatinib for the Treatment of Differentiated Thyroid Cancer in Brazil

Start date: n/a
Phase:
Study type: Expanded Access

This is an Expanded Access Program to make lenvatinib available to participants with radioiodine-refractory differentiated thyroid cancer in Brazil. Participants who have no other treatment options available, and who, in the opinion and clinical judgment of the treating physician, would benefit from treatment with lenvatinib will be enrolled. This is a multicenter, open-label program consisting of 2 phases: a 28-day pretreatment phase (including screening) and a treatment phase. Treatment will be provided as long as there is a clinical benefit based on tumor assessments performed according to the center's standard of care and the judgment of the participant's treating physician.

NCT ID: NCT03561194 Approved for marketing - Clinical trials for Parenteral Nutrition Associated Liver Disease

Omegaven Protocol:Intermediate Size Patient Population

Start date: n/a
Phase:
Study type: Expanded Access

Children requiring prolonged courses of PN are at risk for developing PN associated liver disease. We hypothesize that although omega-6 fatty acid emulsions prevent fatty acid deficiency, they are not cleared in a manner similar to enteral chylomicrons and therefore accumulate in the liver and resulting in steatotic liver injury. We further hypothesize that a fat emulsion comprised of omega-3 fatty acids (i.e., fish oil) such as Omegaven® would be beneficial in the management of steatotic liver injury by its inhibition of de novo lipogenesis, the reduction of arachidonic acid-derived inflammatory mediators, prevention of essential fatty acid deficiency through the presence of small amounts of arachidonic acid, and improved clearance of lipids from the serum.