Table 1.
Ongoing Gene Therapy Clinical Trials for Primary Immunodeficiency
| Disease | Country | Sponsor | Vector | Outcome | Reference | Clinical Trials.gov ref no. |
|---|---|---|---|---|---|---|
| SCID-X1 | France UK US |
Assistance Publique—Hôpitaux de Paris, Great Ormond Street Hospital, Children's Hospital Boston | SIN γ-RV | 8/9 patients alive, immune recovery with clinical benefit | Hacein-Bey-Abina et al.19 |
NCT01410019 NCT01175239 NCT01129544 |
| SCID-X1 | US | St. Jude Children's Research Hospital, National Institute of Allergy and Infectious Diseases | SIN-LV | Two young adult patients, improved IgG production | De Ravin et al.30 |
NCT01512888 NCT01306019 |
| ADA-SCID | Italy | GlaxoSmithKline | γ-RV | 15/18 patients off ERT, clinical benefit | Aiuti et al.,54 Cicalese et al.117 | NCT00598481 |
| ADA- SCID | UK | Great Ormond Street Hospital | γ-RV | 4/6 patients off ERT, clinical benefit | Gaspar et al.55 | NCT01279720 |
| ADA- SCID | US | Donald B. Kohn, UCLA | γ-RV | 9/10 patients off ERT, clinical benefit | Candotti et al.,56 Carbonaro Sarracino et al.118 | NCT00794508 |
| ADA-SCID | UK US |
Great Ormond Street Hospital; Donald B. Kohn, UCLA |
SIN-LV | 5 patients, immune and metabolic recovery | Gaspar et al.119 |
NCT01380990 NCT01852071 |
| WAS | Italy | IRCCS San Raffaele | SIN-LV | 6 patients, immune, hematological and clinical improvement | Aiuti et al.,87 Scaramuzza et al.120 | NCT01515462 |
| WAS | France UK |
Genethon | SIN-LV | France: 4/5 patients alive, clinical improvement | Bosticardo et al.89 |
NCT01347346 NCT01347242 |
| WAS | US | Children's Hospital Boston | SIN-LV | 2 patients, immune and hematological improvement | Williams121 | NCT01410825 |
| CGD | Germany | Hubert Serve, Johann Wolfgang Goethe University Hospitals | SIN γ-RV | NCT01906541 | ||
| CGD | Germany Switzerland UK US |
Genethon UCLA |
SIN-LV | Kaufmann et al.109 |
NCT01855685 NCT02234934 |
ADA, adenosine deaminase; CGD, chronic granulomatous disease; ERT, enzyme replacement therapy; LV, lentiviral; RV, retroviral vector; SCID, severe combined immunodeficiency; SIN, self-inactivating; WAS, Wiskott–Aldrich syndrome.
Studies are classified as active and ongoing based on information retrieved from ClinicalTrials.gov. Updated information on recruitment status can be found on ClinicalTrials.gov.