As of October 2008, the Website clinicaltrials.gov listed 79 investigative drugs made available through expanded-access programs. This total excludes non-drug-related procedures as well as investigational medical devices. Of the 79 programs, 19 of them are in the area of HIV infection and 29 are in the area of cancer.
Of these 79 expanded-access programs, six products have been approved for marketing. Programs approved for marketing include Alimta alone or with Cisplatin in mesothelioma, sponsored by Eli Lilly and Co. (lilly.com); three programs for Humira for rheumatoid arthritis, sponsored by Abbott Laboratories (abbott.com); Isentress for HIV infection, sponsored by Merck & Co. (merck.com); sapropterin for phenylketonuria, sponsored by BioMarin Pharmaceutical Inc. (biomarinpharm.com); Soliris for paraxysmal hemoglobinuria, sponsored by Alexion Pharmaceutical Inc. (alexionpharm.com); and Sprycel for leukemia, sponsored by Bristol-Myers Squibb Co. (bms.com).
| Investigative drug |
Program status |
Sponsor |
Condition |
| 3, 4 DAP |
Available |
University of California, Davis / Jacobus Pharmaceutical Co. |
Lambert-Eaton myasthenic syndrome |
| Adefovir + Levocarnitine |
Completed |
Gilead Sciences Inc. (gilead.com) |
HIV infection |
| Alimta alone or with Cisplatin |
Approved for marketing |
Eli Lilly and Co. (lilly.com) |
Mesothelioma |
| AMD3100 + G-CSF |
Available |
Genzyme Corp. (genzyme.com) |
Autologous stem cell transplant |
| AMN107 |
Active, not recruiting |
Novartis (novartis.com) |
Chronic myeloid leukemia |
| Ampligen |
Recruiting |
Hemispherx Biopharma (hemispherx.net) |
Chronic fatigue syndrome |
| Aptivus (capsules/oral solution) |
Available |
Boehringer Ingelheim GmbH (boehringer-ingelheim.com) |
HIV infections |
| Aztreonam Lysine |
Available |
Gilead Sciences Inc. (gilead.com) |
Cystic fibrosis; Pseudomonas aeruginosa airway infection |
| Baraclude |
Available |
Bristol-Myers Squibb Co. (bms.com) |
Hepatitis B |
| Bexxar (Tositumomab) |
No longer available |
GlaxoSmithKline (gsk.com) |
Non-Hodgkin's lymphoma |
| Bexxar |
Active, not recruiting |
University of Nebraska |
Lymphoma |
| Biological (HIV-1 Immunogen) |
Completed |
The Immune Response Corp., Carlsbad Calif. |
HIV infection |
| Casodex |
No longer available |
AstraZeneca Plc. (astrazeneca-us.com) |
Adenocarcinoma of the prostate |
| CEOP/IMVP-Dexa chemotherapy |
No longer available |
Arbeitsgemeinschaft medikamentoese Tumortherapie (agmt.at) |
Lymphoma |
| Cyclosporine Inhalation Solution |
Available |
APT Pharmaceuticals Inc. (aptbio.com) |
Lung transplant |
| Cymbalta |
No longer available |
Eli Lilly and Co. (lilly.com) |
Major depressive disorder; fibromyalgia; diabetic peripheral neuropathic pain; generalized anxiety disorder |
| Daptomycin |
Completed |
Cubist Pharmaceuticals Inc. (cubist.com) |
Gram-positive bacterial infections |
| Decitabine |
Completed |
MGI Pharma Inc. (mgipharma.com) |
Acute myelogenous leukemia; myelodysplastic syndrome |
| Deferasirox |
Active, not recruiting |
Novartis S.A. (novartis.com) |
Thalassemia; sickle cell disease; Diamond Blackfan anemia; myelofibrosis |
| Defibrotide |
Available |
St. Jude Children's Research Hospital (stjude.org) |
Hepatic veno-occlusive disease |
| Dexamethasone + Velcade |
No longer available |
Millennium Pharmaceuticals Inc. (mlnm.com) |
Multiple myeloma |
| Erwinia L-asparaginase |
Available |
Phoenix Children's Hospital / Fisher Bioservices (fisherbioservices.com) / EUSA Pharma Inc. (eusapharma.com) |
Acute lymphoblastic leukemia; acute lymphoid leukemia |
| Etravirine |
Available |
University of South Alabama / Tibotec Pharmaceutical Ltd. (tibotec.com) |
HIV infection |
| Everolimus |
Available |
Novartis S.A. (novartis.com) |
Metastatic renal cell cancer |
| Ferriprox |
Available |
Children's Hospital of Philadelphia (chop.edu); ApoPharma |
Iron overload |
| Fuzeon + Optimized ARV |
Active, not recruiting |
Roche (roche.com) / Trimeris Inc. (trimeris.com) |
HIV infection |
| Humira |
Approved for marketing |
Abbott Laboratories (abbott.com) |
Rheumatoid arthritis |
| Humira |
Approved for marketing |
Abbott Laboratories (abbott.com) |
Rheumatoid arthritis |
| Humira |
Approved for marketing |
Abbott Laboratories (abbott.com) |
Rheumatoid arthritis |
| Iobenguane |
Available |
GE Healthcare (gehealthcare.com) |
Neuroblastoma |
| Iodine-131 Anti-B1 Antibody |
Active, not recruiting |
Corixa Corp. / GlaxoSmithKline (gsk.com) |
Non-Hodgkin's lymphoma |
| Iressa |
Completed |
AstraZeneca (astrazeneca-us.com) |
Carcinoma; non-small-cell lung cancer; metastases; neoplasm |
| Isentress |
Approved for marketing |
Merck & Co. (merck.com) |
HIV infections |
| lapatinib + capecitabine |
Recruiting |
GlaxoSmithKline (gsk.com) |
Breast cancer |
| Lenalidomide + dexamethasone |
Active, not recruiting |
Celgene Corp. (celgene.com) |
Relapsed or refractory multiple myeloma |
| Lenalidomide + dexamethasone |
Recruiting |
Celgene Corp. (celgene.com) / Prologue Research International (procro.com) |
Multiple myeloma |
| MabThera |
Active, not recruiting |
Roche (roche.com) |
Rheumatoid arthritis |
| Maraviroc |
Recruiting |
Pfizer Inc. (pfizer.com) |
HIV infection |
| Metvix |
Available |
Galderma S.A. (galderma.com) |
Field actinic keratoses; basal cell carcinoma; Bowen's disease |
| Miralax |
Available |
University of Texas Southwestern Medical Center (utsouthwestern.edu) |
Urge syndrome |
| Mirapex |
Temporarily not available |
James P. Bennett Jr., M.D.,Ph.D.; University of Pittsburgh (pitt.edu); University of Nebraska (unl.edu) |
Amyotrophic lateral sclerosis |
| MK0831 |
Completed |
Merck & Co. (merck.com) |
HIV infections |
| Myozyme |
Available |
Genzyme (genzyme.com) |
Glycogen storage disease type II; late-onset Pompe disease; acid maltase deficiency disease; glycogenosis 2 |
| Myozyme |
Completed |
Genzyme (genzyme.com) |
Glycogen Storage Disease Type II; Glycogenosis 2 |
| Myozyme |
Completed |
Genzyme (genzyme.com) |
Glycogen storage disease type II; glycogenosis 2 |
| NRTI, NNRTI, or PI |
Completed |
Rajavithi Hospital / Clinical Molecular Biological Center, Department of Education and Research, Rajavithi hospital in Thailand |
HIV infection; AIDS; neurological complications |
| Oral Liquid Efavirenz |
Available |
Bristol-Myers Squibb Co. (bms.com) |
HIV infection |
| Oral Liquid Efavirenz: Canada |
Available |
Bristol-Myers Squibb Co. (bms.com) |
HIV infection |
| Oral Satraplatin |
Active, not recruiting |
GPC Biotech (gpc-biotech.com) |
Prostate cancer |
| Paromomycin IM Injection |
Recruiting |
Institute for OneWorld Health (oneworldhealth.org) |
Visceral leishmaniasis |
| Pentostam |
Available |
University of California, San Francisco (ucsf.edu) |
Cutaneous and mucocutaneous leishmaniasis |
| Pergolide |
Available |
Valeant Pharmaceuticals North America (valeant.com) |
Parkinson’s disease |
| Plerixafor |
Available |
Genzyme Corp. (genzyme.com) |
Non-Hodgkin's lymphoma; Hodgkin's disease; multiple myeloma |
| Prochymal |
Available |
Osiris Therapeutics Inc. (osiristx.com) / Quintiles Transnational Corp. (quintiles.com) |
Graft vs. host disease |
| Raltegravir |
Recruiting |
Community Research Initiative of New England (crine.org) |
HIV infection |
| Remune |
Completed |
The Immune Response Corp. (orchestratherapeutics.com) |
HIV infection |
| Retaane |
No longer available |
University of Iowa (uiowa.edu) |
Macular degeneration |
| Reyataz |
No longer available |
Bristol-Myers Squibb Co. (bms.com) |
HIV infections |
| Sapropterin |
Approved for marketing |
BioMarin Pharmaceutical Inc. (biomarinpharm.com) |
Phenylketonuria |
| Soliris |
Approved for marketing |
Alexion Pharmaceuticals Inc. (alexionpharm.com) |
Paroxysmal hemoglobinuria |
| Sprycel |
Approved for marketing |
Bristol-Myers Squibb Co. (bms.com) |
Leukemia |
| SU011248 |
Recruiting |
King Faisal Specialist Hospital & Research Center (kfshrc.edu.sa) |
Metastatic renal cell carcinoma |
| Sustiva + Videx + Epivir |
Completed |
French National Agency for Research on AIDS and Viral Hepatitis |
HIV infection |
| Tasigna + Glivec/Gleevec |
Active, not recruiting |
King Faisal Specialist Hospital & Research Center (kfshrc.edu.sa) |
Chronic myeloid leukemia |
| Tasigna + Glivec/Gleevec |
Recruiting |
King Faisal Specialist Hospital & Research Center (kfshrc.edu.sa) |
Chronic myeloid leukemia |
| Tasigna |
Available |
Novartis S.A. (novartis.com) |
Chronic myelogenous leukemia |
| Tegaserod |
No longer available |
Novartis S.A. (novartis.com) |
Irritable bowel syndrome with constipation; chronic idiopathic constipation |
| Telzir |
No longer available |
GlaxoSmithKline (gsk.com) |
HIV infections |
| Tenofovir |
Active, not recruiting |
Gilead Sciences Inc. (gilead.com) |
HIV infection |
| Tipranavir/Ritonavir |
Completed |
Boehringer Ingelheim GmbH (boehringer-ingelheim.com) |
HIV infection |
| Trasylol |
Available |
Bayer Corp. (bayer.com) |
Postoperative hemorrhage |
| Tremelimumab |
No longer available |
Pfizer Inc. (pfizer.com) |
Advanced unresectable melanoma |
| Vandetanib |
Recruiting |
AstraZeneca Plc. (astrazeneca-us.com) |
Hepatocellular carcinoma |
| VariZIG |
Available |
Cangene Corp. (cangene.com) / Centers for Disease Control and Prevention (cdc.gov) |
Varicella |
| Velcade |
Completed |
Janssen-Cilag International NV (janssen-cilag.com) |
Multiple myeloma |
| Viracept |
Completed |
Agouron Pharmaceuticals Inc. |
HIV infection |
| Visudyne + Lucentis + Decadron |
Available |
Vitreous - Retina - Macula Consultants of New York / QLT Inc. (qltinc.com) |
Age-related macular degeneration |
| Voraxaze |
Available |
Protherics Plc (protherics.com) / AAIPharma (aaipharma.com) |
Neoplasms |
| ZD1839 |
Active, not recruiting |
AstraZeneca (astrazeneca-us.com) |
Non-small cell lung cancer; cancer of the head and neck |
| Source: Clinicaltrials.gov |
