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Retinitis PigmentosaPhase 1/2NCT03374657

A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa

A Phase 1/2 study testing CPK850 in people with Retinitis Pigmentosa, run by Novartis Pharmaceuticals at 1 site in Sweden.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study. This study will also evaluate the safety and potential efficacy of CPK850 on improving visual function in patients with decreased visual function… Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Retinitis Pigmentosa
What is being tested?
CPK850
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteSweden
Study dates
22 Aug 2018to 12 May 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male and female patients aged 18 to 70 years inclusive.
  • The visual acuity in the study eye at the screening 1 visit should be no better than 60 ETDRS letters.
  • Clinical diagnosis of Bothnia dystrophy, Newfoundland rod-cone dystrophy or other progressive retinitis pigmentosa phenotype with mutations in the RLBP1 gene verified by genetic testing.
  • Visible photoreceptor (outer nuclear) and Retinal Pigment Epithelium (RPE) layers on standard OCT scan in the study eye at the screening 1 visit.

Who cannot join

  • History of hypersensitivity to the study drug or to drugs of similar classes or to any of the medications required in the perioperative period.
  • Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints
  • Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, internist, or designee).
  • Women who are pregnant, or lactating or women of childbearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for two months after treatment

Trial locations and contacts

1 site in 1 country.

Sweden1 site

  • Novartis Investigative Site

    Stockholm, SE-112 82

Other trials in Sweden

About this study

Official title: An Open-label First-in-human Single Ascending Dose Study to Explore Safety, Tolerability and Efficacy of Subretinal Administration of CPK850 Gene Therapy in Patients With Retinitis Pigmentosa Due to Mutations in the Retinaldehyde Binding Protein 1 (RLBP1) Gene

The purpose of this first-in-human study is to explore the maximum tolerated dose (MTD) of CPK850 as determined by the single ascending dose ranging portion of the study. This study will also evaluate the safety and potential efficacy of CPK850 on improving visual function in patients with decreased visual function from RLBP1 retinitis pigmentosa due to biallelic mutations in the RLBP1 gene.

Read the detailed description

This study will potentially include 4 cohorts with a minimum of 3 patients per cohort. This trial design used a staggered patient enrollment with continuous data reviews to limit as much unforeseen risk as possible prior to enrolling each patient in each cohort or initiating another cohort. Only one eye (designated as the study or treated eye) will be dosed per patient. Each patient will be followed for 5 years after the subretinal injection of CPK850.

Being tested
CPK850
Keywords
Clinical trials, dark adaptation, gene therapy, RLBP1 mutation, retinitis pigmentosa.
Medical subject headings
Retinitis Pigmentosa

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CPK850Biological

    In one of 4 dose levels administered via subretinal injection under anesthesia

Study groups in NCT03374657
Study groupRoleWhat they receive
CPK Dose 1 (lowest dose)ExperimentalCPK850, one subretinal injection to the study eye
Receives: Biological: CPK850
CPK Dose 2 (next lowest dose)ExperimentalCPK850, one subretinal injection to the study eye
Receives: Biological: CPK850
CPK Dose 3 (third lowest dose)ExperimentalCPK850, one subretinal injection to the study eye
Receives: Biological: CPK850
CPK Dose 4 (highest dose)ExperimentalCPK850, one subretinal injection to the study eye
Receives: Biological: CPK850

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with adverse events (AEs), serious adverse events (SAEs) and deaths

    Safety events

    Measured: Up to Year 5

  2. Number of responders in dark adaptation

    A patient is considered a responder if sensitivity recovery values at 1 hour post-bleach are observed to be outside of the patient's prediction interval at ≥2 consecutive post-treatment visits within one year after treatment.

    Measured: Screening/baseline up to Year 1

Secondary outcomes (11)

  1. Number of responders with recovery of the cone system

    cone recovery during dark adaptation

    Measured: Screening/baseline up to Year 1

  2. Change from screening/baseline in Visual field perimetry mean deviation

    Assessed using automated static perimetry

    Measured: Screening/baseline up to Year 2

  3. Change from screening/baseline in Total contrast sensitivity score

    Contrast sensitivity (ie, the ability to detect relatively dim objects) will be assessed

    Measured: Screening/baseline up to Year 2

  4. Change from screening/baseline in Light-adapted microperimetry sensitivity

    Assessed using standard microperimetry equipment

    Measured: Screening/baseline up to Year 5

  5. Change from screening/baseline in the local electrical activity of the retina

    Assessed using a system designed to record multifocal electroretinogram (ERG) responses from a number of locations at one time

    Measured: Screening/baseline up to Year 2

  6. Change from screening/baseline in the electrical activity of the retina

    Assessed using a system designed to record full-field electroretinogram (ERG) responses with Ganzfeld stimulation.

    Measured: Screening/baseline up to Year 5

  7. Change from screening/baseline in Reading speed

    Assessed using standard reading speed charts

    Measured: Screening/baseline up to Year 2

  8. Change from screening/baseline in eye dominance

    Dominant eye for viewing targets at distance

    Measured: Screening/baseline up to Year 5

  9. Change from screening/baseline in Change from baseline in mobility test scores

    Assessed using a system designed to measure the ability to navigate obstacles in a maze-like environment under varying light conditions

    Measured: Screening/baseline up to Year 2

  10. Change from screening/baseline in the National Eye Institute - Visual function questionnaire 25 (NEI-VFQ 25) composite score

    Questionnaire completed by the participant to measure the influence of visual impairment on quality of life

    Measured: Screening/baseline up to Year 5

  11. Change from screening/baseline in the low luminance questionnaire (LLQ) responses

    Questionnaire completed by the participant to assess visual problems under low luminance conditions, including nighttime

    Measured: Screening/baseline up to Year 5

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Dec 2017
  2. Study start22 Aug 2018
  3. Primary completion12 May 2026
  4. Study completion12 May 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Model description
This is a non-confirmatory, open-label single ascending dose gene replacement-therapy study to assess safety, tolerability and efficacy of CPK850 in patients with RLBP1 retinitis pigmentosa due to biallelic mutations in the RLBP1 gene. The trial design uses a staggered patient enrollment.
Masking
Single blind
Who is masked
Outcomes assessor
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03374657
Sponsor's protocol ID
CCPK850X2202
Other IDs
2016-002696-10, 2016
Status last verified
Jun 2026
First submitted
11 Dec 2017
Last updated
11 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Publications

  • Kvanta A, Rangaswamy N, Holopigian K, Watters C, Jennings N, Liew MSH, Bigelow C, Grosskreutz C, Burstedt M, Venkataraman A, Westman S, Geirsdottir A, Stasi K, Andre H. Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial. Nat Commun. 2024 Sep 10;15(1):7438. doi: 10.1038/s41467-024-51575-4.

    PubMed 39256350

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CPK850 in people with Retinitis Pigmentosa, run by Novartis Pharmaceuticals at 1 site in Sweden.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with adverse events (AEs), serious adverse events (SAEs) and deaths (time frame: Up to Year 5). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03374657, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2026.

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