CC-90001-IPF-001 - safety and efficacy of CC90001 in patients with IPF
Research type
Research Study
Full title
A phase 2, 24-week, randomized, double-blind, placebo-controlled, multicenter study, followed by a 28-week treatment extension, to evaluate the efficacy and safety of CC-90001 in subjects with idiopathic pulmonary fibrosis.
IRAS ID
223856
Contact name
Gisli Jenkins
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2016-003473-17
Duration of Study in the UK
3 years, 2 months, 8 days
Research summary
Summary of Research
This is a Phase 2, multicentre, double-blind (where neither the participant nor the investigator knows which treatment the participant is receiving), randomised, placebo-controlled study evaluating the efficacy, safety, pharmacokinetics (PK – what the body does to the drug), quality of life and exploratory pharmacodynamics (PD – what the drug does to the body) of two treatment doses of CC-90001 (study drug) - 200 mg and 400 mg to be taken orally (PO) once daily(QD), compared with placebo (an inactive substance that contains no medicine), in participants with idiopathic pulmonary fibrosis (IPF).
IPF is a chronic, progressive disease that causes scarring (fibrosis) in the lungs. This scarring prevents the lungs from working properly.
Approximately 135 patients worldwide will take part in this research study. The total study duration is up to 61 weeks (Screening phase of up to 5 weeks, treatment phase of 24 weeks, extension phase of 28 weeks and follow-up phase of 4 weeks).
Following an initial screening at Visit 1, eligible participants will be randomised to receive either CC-90001 200 mg or 400 mg PO QD or matching placebo beginning on Day 1 of the study. Following the 24-week Double-blind Treatment Phase, participants will continue the same blinded treatment assignment for 28 weeks in the Treatment Extension Phase. During the 28-week Treatment Extension Phase, all participants will have the opportunity, if deemed appropriate by the Investigator, to receive standard of care treatment (SOC). The time at which to add SOC and the choice of SOC will be at the Investigator’s discretion.
All participants who complete the study treatment phases and those participants who discontinue study drug prior to the completion of the study will participate in the 4-week Post-treatment Observational Follow-up Phase.Summary of Results
This summary shows the results from this one study of CC-90001. Other
studies of CC-90001 may show different results. At the time of this report,
the sponsor does not plan to do further studies of CC-90001 for IPF. If you
have questions about the study or CC-90001, contact your study doctor or
study team.
Did the participants with IPF have improved lung
function after taking CC-90001 for 24 weeks?
To answer this question, researchers reviewed the participants’ percent
predicted forced vital capacity (also called FVC). FVC is the amount of air
that can be forcibly breathed out from the lungs after taking the deepest
breath possible. FVC is one way to test lung function or how well the lungs
work.
The study doctors checked how the percent predicted FVC changed after
participants took the study medicine for 24 weeks compared to before
starting the study medicine (at baseline). Then, they compared the results of
participants who took CC-90001 with those who took a placebo.
•
A percent predicted FVC higher than baseline means the lung
function improved.
• A percent predicted FVC equal to or lower than baseline means
the lung function did not improve or had worsened.
What was the answer to the researchers’ question?
The sponsor decided to end the study earlier than planned before
researchers could gather enough information to find out if CC-90001 could
improve the lung function of participants with IPF.
The sponsor decided to close the study early to focus on the development of
other Celgene medicines. It was not because of any observed or expected
results or safety findings with CC-90001.Because the sponsor ended the study early, this study did not
reach the planned number of participants, and the collected
information was not enough for researchers to carry out the
planned analysis.
As a result, it was not possible for researchers to find out if taking
CC-90001 for 24 weeks could improve the participants’ lung
function compared to taking a placebo.
The sponsor reviewed the information collected when the study ended in
December 2021.How did the percent predicted FVC change after
24 weeks of taking 200 mg of CC-90001?
On average:• Participants who took 200 mg of CC-90001 had percent predicted
FVC higher than baseline after 4 weeks to 8 weeks of treatment.
Then, percent predicted FVC was lower than baseline after 12 weeks
to 24 weeks of treatment.
• After 24 weeks of taking 200 mg of CC-90001, percent predicted
FVC was lower than baseline by about 2%How did the percent predicted FVC change after
24 weeks of taking 400 mg of CC-90001?
On average:• Participants who took 400 mg of CC-90001 had percent predicted
FVC higher than baseline after 4 weeks and through 20 weeks of
treatment. Then, percent predicted FVC was lower than baseline
after 24 weeks of treatment.
• After 24 weeks of taking 400 mg of CC-90001, percent predicted
FVC was lower than baseline by 1%How did the percent predicted FVC change after
24 weeks of taking a placebo?
On average:• Participants who took a placebo had a percent predicted FVC lower
than baseline throughout the 24-week treatment period.
• After 24 weeks of taking a placebo, percent predicted FVC was lower
than baseline by about 3%.Overall:
It was not possible for researchers to carry out the planned analysis for the
results described above. This is because the sponsor ended the study early,
and this study did not reach the planned number of participants. That means
any differences in the results do not show if taking CC-90001 for 24 weeks
could improve the participants’ lung function compared to taking a placebo.
The results described do not mean that everyone in this study had these
results. The charts in Figures 1, 2, and 3 show the average change in
percent predicted FVC of participants and are the main results of the study.
This summary does not show the individual results of participants.Why was this study done?
Researchers were looking for a different way to treat idiopathic pulmonary
fibrosis (IPF).
IPF is a disease that causes scar tissue (fibrosis) to develop in
the lungs. Scarring causes stiffness in the lungs and makes it
hard to breathe.
IPF starts slowly and gets worse over time. About half of the
people with IPF either die or have a lung transplant within 3 to
5 years of diagnosis. The cause of IPF is unknown.CC-90001 is a medicine that was studied for the treatment of IPF. It can
block a protein called c-Jun N-terminal kinase (also called JNK). JNK is
involved in processes that lead to swelling and scarring in some body
tissues.
Researchers wanted to find out if CC-90001 helped improve lung function in
participants with IPF. Researchers also wanted to learn what side effects
participants may have experienced while taking CC-90001.
How was this study done?
This study had 2 parts.
In both parts, none of the participants, study doctors, or
researchers were told which treatment was given until the
study had finished. This is called a double-blind study.Part 1: Participants were assigned to receive either CC-90001 or a placebo
by chance. The doses of CC-90001 were in milligrams (also called mg). This
part was also known as the “24-week treatment periodâ€.CC-90001 (either 200 or 400 mg) was compared with a
placebo treatment in Part 1.
The placebo treatment looked the same as CC-90001, but it
did not have any “active ingredient†in it.A computer program randomly selected who could take
CC-90001 (200 or 400 mg) and who could take a placebo.Some participants who finished Part 1 joined Part 2.
Part 2: Participants took CC-90001 for the rest of the study.Participants took CC-90001 (either 200 or 400 mg) in
Part 2.• Participants who were assigned to take CC-90001 (200 or
400 mg) in Part 1 continued taking the same dose in
Part 2.
• For participants who were assigned to take placebo in
Part 1, a computer program randomly selected who could
take 200 or 400 mg of CC-90001 in Part 2.What treatments were studied?
Part 1: Participants took either CC-90001 (200 or 400 mg) or placebo
tablets by mouth once daily for up to 24 weeks.
Some participants in the placebo group took a placebo for more than
24 weeks because they signed up during an earlier version of the study plan.
Part 2: Participants took CC-90001 (either 200 or 400 mg) for the rest of
the study.Where can I learn more about this study?
This document provides a summary of the main results of the study
(participants with IPF). It includes the answer to the main question that
researchers had and information about the side effects experienced by
participants with IPF when treated with CC-90001.This study also signed up 23 participants with progressive pulmonary
fibrosis (PPF) in Part 1, and 21 of these participants continued to
Part 2. The study plan and the assigned study medicines were the
same as described in this summary for the IPF group.
Researchers checked how many participants with PPF had a side
effect that study doctors thought may be related to the study
medicine. Overall:
No participant in Part 1 or Part 2 had a serious side effect.
During Part 2, 1 participant who took 400 mg of CC-90001
died due to complications from a lung illness.
• A total of 4 out of 23 participants (17%) in Part 1 and
2 out of 21 participants (10%) in Part 2 had at least 1 side
effect. Nausea was the most common side effect in the PPF
group for Part 1 and Part 2REC name
East of England - Cambridge South Research Ethics Committee
REC reference
17/EE/0221
Date of REC Opinion
14 Sep 2017
REC opinion
Further Information Favourable Opinion