encouraging results from the SACHA trial

Chicago, United States— In France, nearly half of children with cancers that have failed treatment cannot benefit from a medicine with marketing authorization or take part in a clinical trial.

In these cases, paediatricians, oncologists and haematologists ask the ANSM for compassionate access or treat off-label, using drugs authorized only for adults.

Since the tolerance and efficacy data for these prescriptions are only collected “anecdotally, without systematic declaration of serious adverse events in pharmacovigilance”, the French Society for the Fight against Childhood Cancer set up the observational study SACHA promoted by Gustave Roussy, who “systematically and prospectively collects data on the tolerance and efficacy of innovative therapies administered under these conditions on a national scale; real-life data.

The preliminary results of the SACHA study were presented at the congress of the American Society of Clinical Oncology (ASCO) June 6 by the Professor Gilles Vassalonco-pediatrician at Gustave Roussy[1,2].

“The results of the study must thus make it possible either to justify the cessation of certain individual prescriptions when they are poorly tolerated and/or ineffective, or to recommend the opening of a clinical trial in the event of an effective response rate. “, indicates a press release Gustave Roussy[1].

The results of the study must thus make it possible either to justify the discontinuation of certain individual prescriptions when they are poorly tolerated and/or ineffective, or to recommend the opening of a clinical trial in the event of an effective response rate.

First data

The SACHA study began in March 2020. In February 2022, 283 young patients had been included in 29 of the 31 French pediatric oncology centers (340 in April 2022). Their median age was 11.0 years (0.3-24.4 years).

The main types of cancer were central nervous system (CNS) tumors (47%), followed by non-CNS tumors (40%) and leukemias (13%).

The most prescribed advanced therapies were trametinib which was associated with the most side effects, trametinib/dabrafenib, pazopanib, cabozantinib, regorafenib and tazemetostat (see table).

“In total, data was collected on 52 different drugs, two-thirds of which were prescribed based on the molecular portrait of the tumour”, indicated Prof. Vassal for the review Pharmaceutiques before adding: “the program generates useful information. For example, we were able to document two hitherto unknown serious adverse events. Conversely, efficacy data collected in SACHA on a MET-inhibitor molecule led to the opening of a new arm in the Acsé-eSMART clinical trial program”.

The main innovative drugs prescribed off-label

Innovative medicines

NOT

n/evaluable patients

Patient with ≥ 1 AE / evaluable patients*

Severe AE*

Trametinib

64

9/40

30/64

3

Trametinib/Dabrafenib

36

11/20

2/33

2

Pazopanib

16

3/12

9/16

0

Cabozantinib

16

0/12

7/16

0

Regorafenib

15

0/9

7/15

0

Tazemetostat

14

1/14

4/14

1

*as of February 4, 2022.

© 2022 by American Society of Clinical Oncology

Building on its national success, the SACHA program should expand internationally before the end of the year with the participation of seven other European countries as well as Australia and New Zealand.

The SACHA study is funded by Imagine for Margo, Hubert Gouin “Childhood and Cancer” and the LEEM Foundation.

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