Muscle biopsies in clinical trials

Part 1 - to be completed be the caregiver

On the 31st of January, a group of patients, patient organizations, clinicians, researchers and companies developing drugs for Duchenne came together for a workshop to discuss and evaluate the outcome measures that are currently used in clinical trials.

One of the sessions during the day involved a discussion on muscle biopsies. This session was led by the muscle biopsy working group which includes a patient, a representative from a patient organization, clinicians and researchers. During the discussions in that session it was felt that very little data had been collected on the patient perspective on muscle biopsies.

To address this the muscle biopsy working group decided to devise this questionnaire to allow us to better understand your views and look at how we can improve the patient and caregiver experience when carrying out muscle biopsies in clinical trials.

Please only complete the questionnaire if the child in your care has taken part in a clinical trial requiring an open muscle biospy. We want to know your feelings on open muscle biopsies (so not needle biopsies or skin biopsies). An open muscle biopsy is a surgical procedure where an incision is made in the skin and subcutaneous fat to reach the muscle and remove a part of it for analysis procedures.

We hope you will fill out this questionnaire to help us collect this important information.

There are three parts to this questionnaire. Parts 1 and 3 are to be completed by the caregiver, and Part 2 by the child with help from the caregiver.

Question Title

* 1. Did your child have a biopsy for diagnosis before the trial began?

Yes

Question Title

* 2. In which of the following clinical trials has your child participated?

Ataluren by PTC

PRO051 or Drisapersen by Prosensa-GSK-BioMarin (skipping exon 51)

PRO044 by Prosensa-BioMarin (skipping exon 44)

PRO045 by Prosensa-BioMarin (skipping exon 45)

PRO053 by Prosensa-BioMarin (skipping exon 53)

AVI 4658 by AVI (skipping exon 51 )

Eteplirsen or Exondys51 by Sarepta (skipping exon 51)

SRP-4045 by Sarepta (skipping exon 45)

SRP-4053 by Sarepta (skipping exon 53)

SRP-4045 and SRP-4053 by Sarepta (ESSENCE) (skipping exon 45 or 53)

Givinostat by Italfarmaco

SMT C1100 by Summit (Ezutromid) (utrophin upregulation)

Other (please specify)

Question Title

* 3. If your child has taken part in more than one clinical trial requiring biopsies, please state below which clinical trial you will evaluate in this questionnaire. We would appreciate you filling in the questionnaire again, evaluating the biopsies in the other trials you took part in. Please also indicate below whether this was the second, third etc trial the child took part in requiring biopsies.

Question Title

* 4. Please state in which month and year the clinical trial began?

Question Title

* 5. Please state the country where the clinical trial took place and the biopsies where taken?

Question Title

* 6. How many muscle biopsies were taken during the clinical trial? (Please do not include biopsies taken at diagnosis)

Other (please specify)

Question Title

* 7. From which muscle(s) were the biopsies taken?

Shin muscle – tibialis anterior

Calf muscle – gastrocnemius

Upper arm muscle - biceps

Shoulder muscle - deltoid

Foot muscle - EDB (extensor digitorum brevis)

Other (please specify)

Question Title

* 8. What type of anesthesia was used for the biopsies?

Local

General

I don’t know

Question Title

* 9. If your child had general anesthesia, do you know how they were put to sleep?

By a gas through a Mask

By a medicine via a cannula

Question Title

* 10. Did your child have any problems with the anesthesia?

Yes

Question Title

* 1. Did your child have a biopsy for diagnosis before the trial began?

Question Title

* 2. In which of the following clinical trials has your child participated?

Question Title

Question Title

* 4. Please state in which month and year the clinical trial began?

Question Title

* 5. Please state the country where the clinical trial took place and the biopsies where taken?

Question Title

* 6. How many muscle biopsies were taken during the clinical trial? (Please do not include biopsies taken at diagnosis)

Question Title

* 7. From which muscle(s) were the biopsies taken?

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* 8. What type of anesthesia was used for the biopsies?

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* 9. If your child had general anesthesia, do you know how they were put to sleep?

Question Title

* 10. Did your child have any problems with the anesthesia?

Question Title

* 11. If you answered yes above. Can you please explain here what problems they had?