An open-label trial of the long-term safety and tolerability of nintedanib per os, on top of standard of care, over at least 2 years, in children and adolescents with clinically significant fibrosing Interstitial Lung Disease (InPedILD®-ON)
- Conditions
- Respiratoire bronchiolitis-geassocieerde interstitiële longziekteinterstitial lung diseaselungfibrose
- Registration Number
- NL-OMON54130
- Lead Sponsor
- Boehringer Ingelheim
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 1
For new patients:
1. Children and adolescents 6 to 17 years old at Visit 2.
2. Signed and dated written informed consent and assent, where applicable, in
accordance
with ICH-GCP and local legislation prior to admission to the trial.
3. Male or female patients. Female of childbearing potential (WOCBP1) must
confirm that
sexual abstinence is standard practice and will be continued until 3 months
after last drug
intake, or be ready and able to use a highly effective method of birth control
per ICH M3
(R2) that results in a low failure rate of less than 1% per year when used
consistently and
correctly, in combination with one barrier method, from 28 days prior to
initiation of
study treatment, during treatment and until 3 months after last drug intake.
Sexual
abstinence is defined as abstinence from any sexual act that may result in
pregnancy. A
list of contraception methods meeting these criteria is provided in the parental
information and in CTP Section 4.2.2.3.
4. Patients with evidence of fibrosing ILD on HRCT within 12 months of Visit 1
as assessed
by the investigator and confirmed by central review.
5. Patients with FVC % predicted >=25% at Visit 2.
6. Patients with clinically significant disease at Visit 2, as assessed by the
investigator based
on any of the following:
* Fan score >=3, or
* Documented evidence of clinical progression over time based on either:
o a 5-10% relative decline in FVC% predicted accompanied by worsening
symptoms, or
o a >=10% relative decline in FVC % predicted, or
o increased fibrosis on HRCT, or
o other measures of clinical worsening attributed to progressive lung disease
(e.g.
increased oxygen requirement, decreased diffusion capacity).
For roll-over patients from the InPedILD® study:
Only criteria 2 and 3 listed for new patients are applicable with the following
additional
inclusion criterion:
7. Patients who completed the InPedILD® trial as planned and who did not
permanently
prematurely discontinue study treatment.
For patients who prematurely discontinued treatment permanently in 1199-0337
but are potentially
eligible and for completed patients from parent trial not able to roll-over
into the extension trial within 12 weeks following their End of Treatment visit:
Inclusion criteria for new patients are applicable except criteria 4, and 6 (as
eligibility for these criteria has been done in 1199-0337 and does not need to
be repeated) and also except inclusion criteria 1 for completed patients from
parent trial not able to roll over within 12 weeks following their End of
Treatment Visit in the parent trial.
See protocol section 3.3.2.
For new patients:
1. AST and/or ALT >1.5 x ULN at Visit 1.
2. Bilirubin >1.5 x ULN at Visit 1.
3. Estimated Glomerular Filtration Rate (eGFR) <30 mL/min /1.73m² at Visit 1.
4. Patients with underlying chronic liver disease (Child Pugh A, B or C hepatic
impairment) at Visit 1.
5. Other investigational therapy received within 1 month or 5 half-lives
(whichever is
shorter but >=1 week) prior to Visit 2 except investigational therapy received in
InPedILD® trial.
6. Significant pulmonary arterial hypertension (PAH)
7. In the opinion of the Investigator, other clinically significant pulmonary
abnormalities.
8. Cardiovascular diseases
9. Bleeding risk
10. History of thrombotic event within 12 months of Visit 1.
11. Known hypersensitivity to the trial medication or its components (i.e. soya
lecithin).
12. Patients with documented allergy to peanut or soya.
13. Other disease that may interfere with testing procedures or in the judgment
of the
investigator may interfere with trial participation or may put the patient at
risk when
participating in this trial.
14. Life expectancy for any concomitant disease other than ILD <2.5 years
(investigator
assessment).
15. Female patients who are pregnant, nursing, or who plan to become pregnant
while in
the trial.
16. Patients not able or willing to adhere to trial procedures, including
intake of study
medication.
17. Patients who must or wish to take any drug considered likely to interfere
with the safe
conduct of the trial according to investigator*s benefit-risk assessment for
the individual
patient.
18. Patients with any diagnosed growth disorder such as growth hormone
deficiency or any
genetic disorder that is associated with short stature and/or treatment with
growth hormone therapy
within 6 months before Visit 2.
19. Patients <13.5 kg of weight at Visit 1
For roll-over patients from the InPedILD® study:
Only criteria 11, 12, 13, 15, 16, 17 and 19, listed for new patients are
applicable with the
following additional exclusion criterion:
20. Patient not compliant in parent trial (InPedILD®), with trial medication or
trial visits,
according to investigator*s judgement.
Roll-over patients may qualify for participation even though other exclusion
criteria may
have been met during the participation in InPedILD®, if the investigator*s
benefit-risk
assessment for the individual patient remains favourable. This should be
discussed with
sponsor before the roll-over of patient.
For patients who prematurely discontinued treatment permanently in 1199-0337
but are potentially
eligible and for completed patients from parent trial not able to roll-over
into the extension trial within 12 weeks following their End of Treatment visit:
All exclusion criteria for new patients are applicable. In addition, following
additional exclusion
criterion is applicable for patients who prematurely discontinued treatment
permanently in 1199-0337:
21. Patients who experienced drug-related adverse events during parent trial
leading to
permanent study treatment discontinuation.
See protocol section 3.3.3.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is the incidence of treatment emergent adverse events over<br /><br>the whole trial.<br /><br><br /><br>See section 2.1 of the protocol.</p><br>
- Secondary Outcome Measures
Name Time Method <p>N/A</p><br>