Find a clinical trial
Long Chain Fatty Acid Oxidation Disorders
Expanded Access to Triheptanoin
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
- Trial name
- NCT03773770
- Testing
- Triheptanoin
- Sponsor
- Ultragenyx Pharmaceutical Inc
Oral Health Promotion and Anti-inflammatory Effects of Fermented Lingonberry Juice Gel.
The aim of this clinical study is to investigate whether FLJ gel has effects on oral health, such as reducing BOP or plaque, and whether it affects the reduction of aMMP-8.
- Trial name
- NCT07737574
- Testing
- Fermented lingonberry juice gel
- Sponsor
- Hanna Lahteenmaki
MPS VII, Mucopolysaccharidosis VII
Expanded Access to Mepsevii
Individual patient expanded access requests may be considered for patients who have no other treatment options
- Conditions
- MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
- Trial name
- NCT03775174
- Testing
- Mepsevii
- Sponsor
- Ultragenyx Pharmaceutical Inc
Benign Neoplasm, Bone Marrow Transplantation Recipient
Bone Marrow Transplantation Using CD34-Selected Stem Cells From Related or Unrelated Donors in Treating Participants With Cancer or Other Disorders
This expanded access protocol studies bone marrow transplantation using CD34-selected stem cells from related or unrelated donors in treating participants with cancer or other disorders.
- Conditions
- Benign Neoplasm, Bone Marrow Transplantation Recipient, Hematopoietic Cell Transplantation Recipient, Malignant Neoplasm
- Trial name
- NCT03626285
- Testing
- Bone Marrow Transplantation, CliniMACS CD34 Reagent System, Peripheral Blood Stem Cell Transplantation
- Where
- United States
- Sponsor
- OHSU Knight Cancer Institute
Migraine
Fremanezumab Managed Access Program for Pediatric Patients
This is a Managed Access Program (MAP) based on a licensed physician's unsolicited request on a compassionate basis. Specifically, this MAP is for male and female pediatric patients 6 to 17 years with a diagnosis of either…
- Trial name
- NCT03539393
- Testing
- Fremanezumab
- Sponsor
- Teva Branded Pharmaceutical Products R&D LLC
Glioma, Recurrence
Vorasidenib Expanded Access Program
This is an expanded access program to provide vorasidenib for treatment of patients 12 years or older with IDH1- or IDH2-mutated glioma.
- Conditions
- Glioma, Recurrence, Disease Attributes, Pathologic Processes
- Trial name
- NCT05592743
- Testing
- Vorasidenib
- Sponsor
- Servier
Neuroblastoma
131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol
This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG).
- Trial name
- NCT01370330
- Testing
- Metaiodobenzylguanidine (MIBG)
- Where
- United States
- Sponsor
- Kieuhoa Vo
PDAC - Pancreatic Ductal Adenocarcinoma, Colorectal Cancer
Intermediate-size IND for Treatment of Patients With Advanced Cancer Using T Cells Engineered to Express TCR Targeting Mutant KRAS
The goal of this intermediate-size expanded access treatment program is to treat a subset of patients with advanced solid cancers, specifically pancreatic ductal adenocarcinoma (PDAC) and colorectal cancer (CRC), that express…
- Trial name
- NCT07614048
- Testing
- Cyclophosphamide, Fludarabine, Cell infusion
- Sponsor
- Providence Health & Services
Progressive Familial Intrahepatic Cholestasis
Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
To provide treatment access to patients with PFIC in the US who have pruritus and elevated serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following reasons: 1) Do not meet eligibility criteria for…
- Trial name
- NCT04483531
- Testing
- Odevixibat
- Where
- United States
- Sponsor
- Albireo
Hereditary Angioedema
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Hereditary Angioedema (HAE) pediatric (ages 2-11) post-trial and naïve…
- Trial name
- NCT07216378
- Testing
- Sebetralstat
- Where
- 6 sites in United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Anemia Severe
Expanded Access IND Administration of HBOC-201 in Patients With Severe Acute Anemia
HBOC-201 provides an oxygen treatment bridge and can be used to eliminate, delay, or reduce the need for red blood cell transfusions in anemic patients This is an expanded access IND protocol, and will provide treatment with…
- Trial name
- NCT03633604
- Testing
- HBOC-201
- Where
- United States
- Sponsor
- Michael Boisen
Rabies
An Expanded Access Program for SYN023 in Rabies Treatment
This sponsor-initiated Expanded Access Program provides compassionate-use SYN023, a dual monoclonal antibody cocktail (zamerovimab + mazorelvimab) targeting rabies virus glycoprotein, for US patients with confirmed or suspected…
- Trial name
- NCT07706907
- Testing
- Zamerovimab and Mazorelvimab Injection
- Sponsor
- Synermore Biologics (Suzhou) Co., Ltd.
Primary Immunodeficiency Diseases (PIDD)
Expanded Access to TAK-881 for People With Primary Immunodeficiency Diseases in the USA and Czech Republic
The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. This study will provide access to TAK-881 for participants with PIDD who have completed study TAK-881-3002…
- Trial name
- NCT06955793
- Testing
- TAK-881, SC Investigational Needle Sets
- Sponsor
- Takeda
Post-bariatric Hypoglycemia
An Expanded Access Protocol to Provide Avexitide in Patients With Post-Bariatric Hypoglycemia
The Expanded Access Program will provide access to avexitide for people with post-bariatric hypoglycemia (PBH) following Roux-en-Y gastric bypass (RYGB) who meet the eligibility criteria for this program.
- Trial name
- NCT07582874
- Testing
- Avexitide
- Where
- 4 sites in United States
- Sponsor
- Amylyx Pharmaceuticals Inc.
Systemic Mastocytoses, Indolent, Systemic Mastocytoses, Aggressive
Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis
The purpose of this expanded access program (EAP) protocol is to provide investigational bezuclastinib to patients with a diagnosis of nonadvanced systemic mastocytosis (NonAdvSM) or advanced systemic mastocytosis (AdvSM) who…
- Trial name
- NCT06915766
- Testing
- Bezuclastinib
- Where
- 23 sites in United States
- Sponsor
- Cogent Biosciences, Inc.
Gastrointestinal Neoplasms, Gastrointestinal Stromal Tumors
Expanded Access to Bezuclastinib to be Coadministered With Sunitinib for Patients With Gastrointestinal Stromal Tumors
The purpose of this expanded access program (EAP) is to provide investigational bezuclastinib so that it can be coadministered with sunitinib to patients with a diagnosis of gastrointestinal stromal tumors (GIST) with no…
- Trial name
- NCT06948955
- Testing
- Bezuclastinib, Sunitinib
- Where
- 26 sites in United States
- Sponsor
- Cogent Biosciences, Inc.
Pancreatic Ductal Adenocarcinoma, KRAS G12D
Expanded Access Protocol of ELI-002-102 in Subjects With KRAS/NRAS Mutated Pancreatic Ductal Adenocarcinoma
This is an open-label expanded access protocol (EAP) of ELI-002 7P immunotherapy (a lipid-conjugated immune-stimulatory oligonucleotide [Amph-CpG-7909] plus a mixture of lipid-conjugated peptide-based antigens [Amph-Peptides 7P])…
- Conditions
- Pancreatic Ductal Adenocarcinoma, KRAS G12D, KRAS G12V, KRAS G12S
- Trial name
- AMPLIFY-EAP (NCT07083479)
- Testing
- ELI-002 7P
- Sponsor
- Elicio Therapeutics
Chronic Graft Vs. Host Disease
Expanded Access of Axatilimab to Treat a Single Patient With Chronic Graft Versus Host Disease (GVHD)
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee and Single Patient IND approval.
- Trial name
- NCT05544032
- Testing
- axatilimab
- Sponsor
- Incyte Corporation
Lennox Gastaut Syndrome
An Extended Access Program (EAP) for Participants Who Have Completed Rufinamide Study E2080-G000-303
This is an extended access study for participants who have completed Rufinamide Study E2080-G000-303 to continue to have access to rufinamide until it becomes commercially available in Poland or until no participants remain in…
- Trial name
- NCT03778424
- Testing
- Rufinamide
- Where
- 2 sites in Poland
- Sponsor
- Eisai Inc.
Familial Adenomatous Polyposis (FAP)
Expanded Access to 0.5 mg eRapa for Familial Adenomatous Polyposis
This expanded access program provides 0.5 mg eRapa (encapsulated rapamycin) to patients with familial adenomatous polyposis (FAP) who have no satisfactory alternative treatment options and are not eligible to participate in a…
- Trial name
- eRapa-FAP (NCT07689942)
- Testing
- eRapa (encapsulated rapamycin)
- Sponsor
- Biodexa Pharmaceuticals