Find a clinical trial
- Study type Expanded access
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Cancer
Expanded Access Program to Provide STC-15 to Patients With Selected Advanced Cancer
This Expanded Access Program (EAP) is intended to provide access to STC-15 outside of a clinical study setting for adult and pediatric patients with serious or life-threatening malignancies who, in the opinion of their treating…
- Trial name
- NCT07906093
- Testing
- STC-15
- Sponsor
- STORM Therapeutics LTD
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.
Congenital Haemophilia
Compassionate Use of Concizumab if You Have Haemophilia
The compassionate use programme will give participants concizumab for free, even though it is not yet approved by health authorities. This is because participants need this medicine to treat their haemophilia properly.
- Trial name
- NCT04921956
- Testing
- Concizumab
- Where
- 25 sites in United States, Bulgaria and 1 more
- Sponsor
- Novo Nordisk A/S
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.
Pompe Disease Infantile-Onset
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
- Trial name
- NCT04327973
- Testing
- ATB200, AT2221
- Where
- 4 sites in United States, Italy and 1 more
- Sponsor
- Amicus Therapeutics
Lipodystrophy, Generalized Lipodystrophy (GLD)
Compassionate Use or Expanded Access of Mibavademab
Provide Expanded Access to REGN4461 for patients with diseases associated with deficient leptin signaling.
- Conditions
- Lipodystrophy, Generalized Lipodystrophy (GLD), Monogenic Obesity (MOB)
- Trial name
- NCT04710056
- Testing
- REGN4461
- Sponsor
- Regeneron Pharmaceuticals
Fabry Disease
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.
- Trial name
- NCT01476163
- Testing
- migalastat HCl 150 mg, migalastat HCl 20 mg
- Sponsor
- Amicus Therapeutics
Pompe Disease
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
- Trial name
- NCT03865836
- Testing
- ATB200, AT2221
- Sponsor
- Amicus Therapeutics
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
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
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
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.
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
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
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