Find a clinical trial
Managed Access Program Allowing Access to Olorofim
This is a managed access programme for patients meeting the entry criteria eithe de novo or completing a clinical trial
- Trial name
- NCT07895264
- Testing
- Anti Fungal Drug, Olorofim is a novel antifungal drug
- Sponsor
- F2G Ltd
Brain Injury
Intermediate Size Patient Population Expanded Access Protocol to Evaluate HB-adMSCs for the Treatment of Patients With Traumatic or Nontraumatic Brain Injury
This expanded access protocol is part of IND 031942 to evaluate efficacy and safety of multiple intravenous administrations of allogeneic HB-adMSCs for the treatment of nontraumatic or traumatic brain injury for up to 8 patients…
- Trial name
- NCT07164482
- Testing
- HB- adMSCs (Hope Biosciences adipose derived mesenchymal stem cells)
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Cerebral Palsy, Hydrocephalus
Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries
This protocol is designed to enable access to intravenous infusions of banked umbilical cord blood (CB), that is thawed and not more than minimally manipulated, for children with various brain disorders.
- Conditions
- Cerebral Palsy, Hydrocephalus, Apraxia of Speech, Hypoxia Ischemia, Cerebral
- Trial name
- NCT03327467
- Testing
- Cord Blood Infusion
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD
Still's Disease, Including SJIA and AOSD, With MAS or ILD Complications
Managed Access Programs for MAS825, Arumakimig
The purpose of this registration is to list Managed Access Programs (MAPs) related to MAS825, arumakimig
- Trial name
- NCT07889310
- Testing
- Arumakimig
- Sponsor
- Novartis Pharmaceuticals
Epilepsies, Myoclonic, Drug Resistant Epilepsy
Extended Access Program With Lorcaserin For The Treatment of Dravet Syndrome and Other Refractory Epilepsies
The primary purpose of this study is to provide continued access of lorcaserin to participants with Dravet syndrome and other refractory epilepsies.
- Trial name
- NCT04457687
- Testing
- Lorcaserin
- Where
- 22 sites in United States, Canada
- Sponsor
- Eisai Inc.
Mitochondrial Diseases, Barth Syndrome
An Intermediate Size Expanded Access Protocol of Elamipretide
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor to learn more about this program.
- Trial name
- NCT04689360
- Testing
- elamipretide
- Sponsor
- Stealth BioTherapeutics Inc.
Tourettes Disorder, Tourette Disorder
Expanded Access Program for Treatment With Ecopipam for Tourette's Disorder
The goal of this expanded access study is to learn about the long-term effects of Ecopipam in participants with Tourette's Disorder (TD).
- Trial name
- EBS-101-TD-392 (NCT07093541)
- Testing
- ecopipam HCl ~2mg/kg/day
- Where
- 26 sites in United States
- Sponsor
- Emalex Biosciences Inc.
Hereditary Angioedema (HAE)
Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study
This protocol for post study access allows pediatric participants (2-11 years old at the time of consent) with HAE who have completed study CSL312_3003 (NCT05819775) to continue treatment with CSL312 for routine prevention of HAE…
- Trial name
- NCT07159464
- Testing
- CSL312
- Sponsor
- CSL Behring
Ebola Virus Disease
R3470-3471-3479 (REGN-EB3) Expanded Access Protocol (EAP) for Treatment of Ebola Virus Disease
Provide access to REGN-EB3 for the treatment of patients with Ebola Virus Disease (EVD).
- Trial name
- NCT03576690
- Testing
- REGN3470-3471-3479
- Sponsor
- Regeneron Pharmaceuticals
Relapsed or Refractory (R/R) Follicular Lymphoma (FL), Diffuse Large B-Cell Lymphoma (DLBCL)
A Compassionate Use (CU) Program of Odronextamab
Provide compassionate use of odronextamab
- Conditions
- Relapsed or Refractory (R/R) Follicular Lymphoma (FL), Diffuse Large B-Cell Lymphoma (DLBCL), B-Cell Non-Hodgkin Lymphoma (NHL), High-Grade B-Cell Lymphoma (HGBCL)
- Trial name
- NCT05619367
- Testing
- Odronextamab
- Sponsor
- Regeneron Pharmaceuticals
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.
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.
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
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
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
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
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