Find a clinical trial
Parkinson Disease
Compassionate Use for BIA 28-6156 in Patients With Parkinson's Disease (PD) Who Have a Pathogenic Variant in the GBA1 Gene (GBA-PD) and Completed the ACTIVATE Study
This Program is designed to provide continued access BIA 28-6156, free and compassionately for an eligible patient previously enrolled in the BIA 28-6156-201 (ACTIVATE) clinical study and that had completed the study.
- Trial name
- NCT07522606
- Testing
- BIA 28-6156
- Where
- 36 sites in United States, Italy and 3 more
- Sponsor
- Bial R&D Investments, S.A.
Neuroblastoma, Pheochromocytoma
A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma
This is an expanded access protocol/compassionate use single institution study designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with progressive neuroblastoma and metastatic pheochromocytoma who…
- Trial name
- NCT03015844
- Testing
- 131-I-meta-iodobenzylguanidine
- Where
- United States
- Sponsor
- Northwell Health
Glioblastoma, Respiratory Distress Syndrom
NuvOx Expanded Access
NanO₂ is an investigational drug being studied by NuvOx Therapeutics. It has not been approved by the U.S. Food and Drug Administration (FDA) for any use.
- Conditions
- Glioblastoma, Respiratory Distress Syndrom, Ischemic Stroke, Hypoxic Conditions
- Trial name
- NCT07748299
- Testing
- NanO₂
- Sponsor
- NuvOx LLC
Bipolar 1 Disorder, Schizophrenia Disorder
An Expanded Access Program (EAP) for OLZ/SAM in Pediatric Participants With Schizophrenia or Bipolar I Disorder (ALKS 3831-315)
To provide continued access to OLZ/SAM for pediatric participants with schizophrenia or bipolar I disorder who have completed participation in the ALKS 3831-A313 long-term safety extension study.
- Trial name
- NCT07744113
- Testing
- OLZ/SAM
- Where
- United States
- Sponsor
- Alkermes, Inc.
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
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
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.
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
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
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
Peripheral Nerve Transections, Acute or Planned in Upper Extremity and Facial Nerves
NTX-001 to Repair Peripheral Nerve Transection(s)
The proposed use of NTX-001 for transections of upper extremity and facial peripheral nerves, acutely or planned.
- Trial name
- NCT05565846
- Testing
- NTX-001
- Where
- 2 sites in United States
- Sponsor
- Neuraptive Therapeutics 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
Leukemia, Bone Marrow Failure Syndrome
Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC
The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT).
- Conditions
- Leukemia, Bone Marrow Failure Syndrome, Immunodeficiencies
- Trial name
- NCT03145545
- Testing
- Apha/beta T and CD19+ cell depletion using CliniMACS device
- Where
- United States
- Sponsor
- Children's Hospital of Philadelphia
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.
Smallpox, Monkeypox
Tecovirimat (ST-246) Treatment for Orthopox Virus Exposure
The purpose of this clinical protocol is to treat individuals with Tecovirimat after exposure to orthopox viruses.
- Trial name
- NCT02080767
- Testing
- Tecovirimat
- Sponsor
- U.S. Army Medical Research and Development Command