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NCT ID: NCT03847376 Approved for marketing - HIV Clinical Trials

Pre-Approval Access to Rilpivirine Long Acting (RPV LA) Injectable Suspension for the Treatment of a Participant With Human Immunodeficiency Virus (HIV)-1 Infection

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

The main purpose of this pre-approval access program (PAAP) is to provide rilpivirine Long Acting (RPV LA) injectable suspension for the treatment of human immunodeficiency virus (HIV)-1 infection as single patient request to individuals who have no available treatment alternatives and/or limited treatment options (for example, who are unable to participate in the Phase III clinical studies or do not qualify). RPV LA can only be provided if participants are also eligible for cabotegravir Long Acting injections.

NCT ID: NCT03862885 Approved for marketing - GIST Clinical Trials

Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic GIST

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

This is a US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.

NCT ID: NCT03895528 Approved for marketing - Progeria Clinical Trials

Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy

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

This treatment IND protocol will allow patients with HGPS and progeroid laminopathies access to lonafarnib, the only compound shown to have an effect on the HGPS disease process resulting in improved outcomes (Gordon et al, 2018). There are no approved treatments for HGPS and progeroid laminopathies.

NCT ID: NCT03924492 Approved for marketing - Clinical trials for Postpartum Depression

Expanded Access Protocol of ZULRESSO™ (Brexanolone) Injection for Adult Patients With Postpartum Depression

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

This is an expanded access program intended to provide access to ZULRESSO™ (brexanolone) injection for the treatment of a limited number of eligible women with postpartum depression during the period prior to commercial availability. Requests for access must be made by a healthcare provider for a specific patient based on a determination with patient that the benefits of treatment outweigh the risks and an assessment of eligibility based on program criteria. Sites with eligible patients must apply and meet requirements for participation in the expanded access program.

NCT ID: NCT03955679 Approved for marketing - Clinical trials for Spinal Muscular Atrophy

AveXis Managed Access Program Cohort for Access to AVXS-101

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

The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.

NCT ID: NCT04040894 Approved for marketing - Thyroid Eye Disease Clinical Trials

Expanded Access Protocol of Teprotumumab (HZN-001) for Patients With Active Thyroid Eye Disease

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

This is an expanded access protocol intended to provide access to teprotumumab for the treatment of up to 60 patients in the United States with active moderate to severe thyroid eye disease where there is no comparable or satisfactory alternative therapy for treatment.

NCT ID: NCT04056481 Approved for marketing - Clinical trials for Acute Hepatic Porphyria

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

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

The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).

NCT ID: NCT04058210 Approved for marketing - Cystic Fibrosis Clinical Trials

VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

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

The purpose of this program is to provide elexacaftor(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) mutation in response to unsolicited physician requests.

NCT ID: NCT04125472 Approved for marketing - Clinical trials for Primary Hyperoxaluria

Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

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

The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),

NCT ID: NCT04136808 Approved for marketing - Clinical trials for Locally Advanced or Metastatic Urothelial Carcinoma (UC)

An Expanded Access Treatment Protocol of Enfortumab Vedotin in Subjects With Locally Advanced or Metastatic Urothelial Carcinoma

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

The primary purpose of this expanded access program is to evaluate safety and tolerability of enfortumab vedotin (EV) in participants in the United States with locally advanced or metastatic urothelial carcinoma (UC) who have exhausted standard of care therapies and are not eligible to participate in an ongoing EV clinical study. This program will also evaluate the efficacy of EV.