Find a clinical trial
Amyotrophic Lateral Sclerosis
Early Access Program for Ulefnersen in Children, Adolescents and Adults With FUS-associated Amyotrophic Lateral Sclerosis (FUS-ALS)
This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria.
- Trial name
- NCT07862959
- Testing
- Ulefnersen
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Advanced Testicular Germ Cell Tumors, Metastatic Testicular Germ Cell Tumors
Medical Access Program for DS-9606a (DS-9606) in Patients With Advanced Testicular Germ Cell Tumors
DS-9606a demonstrates antitumor activity in patients with solid tumors known to express CLDN-6, primarily in patients with testicular GCT, with a manageable safety profile with appropriate risk minimization measures.
- Trial name
- NCT07863284
- Testing
- DS-9606a
- Sponsor
- Daiichi Sankyo
Relapsed or Refractory Multiple Myeloma
Pre-Approval Access Single Patient Request for Talquetamab in Relapsed or Refractory Multiple Myeloma
The purpose of this pre-approval access program is to provide talquetamab for the treatment of participants with relapsed or refractory multiple myeloma.
- Trial name
- NCT05503550
- Testing
- Talquetamab
- Sponsor
- Janssen Research & Development, LLC
Relapsed or Refractory Multiple Myeloma
Pre-Approval Access Program for Talquetamab in Relapsed or Refractory Multiple Myeloma
The purpose of this pre-approval access program is to give talquetamab monotherapy (treatment with single drug) to participants with relapsed or refractory multiple myeloma (a type of cancer that begins in plasma cells [white…
- Trial name
- NCT05757973
- Testing
- Talquetamab
- Sponsor
- Janssen Research & Development, LLC
Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome
Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
The goal of this program is to provide expanded access (i.e., before marketing authorization) to tiratricol as treatment for patients with monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as…
- Trial name
- NCT05911399
- Testing
- Tiratricol
- Where
- 17 sites in United States
- Sponsor
- Rare Thyroid Therapeutics International AB
SCLC, Extensive-stage Small Cell Lung Cancer
Medical Access Program for Ifinatamab Deruxtecan in 3L+ Pretreated Extensive-stage Small Cell Lung Cancer
The purpose of this Medical Access Program (also referred to as an Expanded Access Program in the USA) is to provide access to I-DXd for eligible patients with extensive-stage small cell lung cancer (ES-SCLC) after two or more…
- Trial name
- NCT07535359
- Testing
- Ifinatamab Deruxtecan
- Where
- 5 sites in United States
- Sponsor
- Daiichi Sankyo
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Expanded Access Program of Bitopertin For Participants With EPP or XLP
The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.
- Trial name
- NCT07603401
- Testing
- DISC-1459
- Where
- 7 sites in United States
- Sponsor
- Disc Medicine, Inc
Mucopolysaccharidosis Type IIIB, MPS IIIB (Sanfilippo B Syndrome)
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)
This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV) tralesinidase alfa (TA) for participants with mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo…
- Trial name
- EAP (NCT07733856)
- Testing
- Tralesinidase alfa (TA)
- Where
- United States
- Sponsor
- Spruce Biosciences
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm
Individual Patient Compassionate Use of Mirdametinib
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.
- Conditions
- Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
- Trial name
- NCT05028166
- Testing
- Mirdametinib (MEK Inhibitor)
- Where
- 2 sites in United States, Germany
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Desmoid Tumor, NOTCH Gene Mutation Positive Tumors
Individual Patient Compassionate Use of Nirogacestat
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.
- Trial name
- NCT05041036
- Testing
- Nirogacestat (Gamma Secretase Inhibitor)
- Where
- 2 sites in United States, Germany
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Retinitis Pigmentosa
Expanded Access Program for RP in Adults
This intermediate-size patient population expanded access program is to provide access to investigational product OCU400 for up to 75 patients with Retinitis Pigmentosa (RP) outside of the ongoing clinical trial Phase 3 program…
- Trial name
- NCT06574997
- Testing
- OCU400
- Where
- 5 sites in United States
- Sponsor
- Ocugen
Alexander Disease
Zilganersen Expanded Access Program for Individuals With Alexander Disease
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
- Trial name
- NCT07487389
- Testing
- zilganersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Total Parenteral Nutrition-induced Cholestasis
Expanded Access of Omegaven IV Fat Emulsion to Infants and Children With PNALD
This is an expanded access protocol for use of intravenous fish oil infusion, Omegaven, in infants and children with parenteral nutrition associated liver disease (PNALD) to decrease elevated liver enzymes and direct bilirubin.
- Trial name
- NCT02929303
- Testing
- Omegaven IV lipid emulsion
- Where
- United States
- Sponsor
- Wake Forest University Health Sciences
Ataxia-Telangiectasia (A-T)
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.
- Trial name
- NCT07380165
- Testing
- Levacetylleucine
- Sponsor
- IntraBio Inc
Multiple Sclerosis
Expanded Access to Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Progressive Multiple Sclerosis
To give expanded access to intrathecal autologous MSC-NP treatment to patients with progressive MS who do not meet the inclusion/exclusion criteria of our Phase II stem cell trial.
- Trial name
- NCT03822858
- Testing
- Intrathecal MSC-NP injection
- Where
- United States
- Sponsor
- Tisch Multiple Sclerosis Research Center of New York
KRAS G12V Mutant Advanced Epithelial Cancers
Intermediate-size IND for Treatment of KRAS G12V-mutant Tumors
This intermediate-size IND will treat participants with incurable, advanced or metastatic cancer, harboring KRAS G12V mutation and appropriate HLA class II match (HLA-DRB1*07:01).
- Trial name
- NCT05389514
- Testing
- Cyclophosphamide, Gemcitabine, Pembrolizumab
- Sponsor
- Providence Health & Services
Non-Muscle Invasive Bladder Neoplasms
Pre-Approval Expanded Access Study for TAR-200 Treating Physician Use
The purpose of this pre-approval access program is to provide access to TAR-200 prior to its marketing authorization for the treatment of participant(s) with high-risk non-muscle invasive bladder cancer (HR-NMIBC).
- Trial name
- NCT06877676
- Testing
- TAR-200
- Sponsor
- Janssen Research & Development, LLC
ALS (Amyotrophic Lateral Sclerosis)
Intermediate-size Patient Population Expanded Access Protocol
The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial.
- Trial name
- NCT07088159
- Testing
- Tazbentetol
- Where
- 16 sites in United States
- Sponsor
- Spinogenix
Recurrent Osteosarcoma
Single-Patient Expanded Access Protocol - RNA-LP in a Patient With Recurrent Osteosarcoma
Treatment of a patient with recurrent osteosarcoma with personalized pp65/tumor mRNA RNA-LP under an individual patient expanded access protocol.
- Trial name
- NCT07560709
- Testing
- pp65/tumor mRNA RNA-LP
- Where
- United States
- Sponsor
- University of Florida
Non Small Cell Lung Cancer, ALK-positive Non-small Cell Lung Cancer (NSCLC)
Expanded Access Program of Neladalkib (NVL-655) for Patients With Advanced ALK+ NSCLC or Other ALK+ Solid Tumors
The Expanded Access Program will provide an alternate mechanism for patients, who lack satisfactory therapeutic alternatives and cannot participate in a neladalkib clinical trial, to access investigational neladalkib.
- Trial name
- NVL-655-EAP (NCT06834074)
- Testing
- NVL-655
- Where
- 50 sites in United States, Australia and 10 more
- Sponsor
- Nuvalent Inc.