Paroxysmal Nocturnal HemoglobinuriaNCT06411626
Home Reported Outcomes in PNH
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO)… Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- PNH-relevant therapies
- How many people
- 128 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jun 2024to 15 Jun 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Individuals aged 18+ who have been diagnosed with PNH, regardless of symptom or treatment history, and are US-based with a proficient understanding of and ability to read the English language.
Who may be able to join
- Study participants eligible for inclusion in this study must meet all of the following criteria:
- Aged 18 or older
- US-based with a proficient understanding of and ability to read the English language
- Any patient with a diagnosis of PNH, regardless of symptom or treatment history
Who cannot join
- Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other exclusion criteria in order to meet the exploratory nature of the primary endpoint.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
United States1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
East Hanover, New Jersey, 07936
About this study
Official title: Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.
Read the detailed description
The study will be prospective and observational, conducted over an initial period of six months per individual from the point of study enrollment. Participants will utilize the Folia mobile app to enroll, consent, and complete all study activities. A hybrid recruitment method of clinic referrals and community referrals will be employed to identify participants, who will be asked to track routine treatment, symptoms, changes in treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins will be conducted to capture additional data inputs, with the possibility of integrating electronic health record (EHR) and/or claims data.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- PNH-relevant therapies
- Keywords
- Paroxysmal nocturnal hemoglobinuria, PNH, home reported outcomes, HRO, patient-reported outcome, PRO, real-world data, iptacopan
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PNH-relevant therapiesOther
This is an observational study. There is no treatment allocation. The decision to initiate PNH-relevant therapies (such as eculizumab, ravulizumab, pegcetacoplan, iptacopan, and others) will be based solely on clinical judgement.
| Study group | Role | What they receive |
|---|---|---|
| Participants with PNH | Participants with PNH Receives: Other: PNH-relevant therapies |
What is being measured
The primary outcome is the main question the study is designed to answer.
PNH symptom burden
Primary endpoint will be measured through longitudinal Home Reported Outcomes (HRO) tracking of self-selected symptoms at enrollment and associated changes in severity against self-reported baselines.
Measured: Baseline, month 6
Secondary outcomes (3)
Number of participants by treatment utilization and management of potential flare events
Treatment utilization and management of potential flare events tracked during the study period
Measured: Up to 6 months
Number of participants by reported treatment use and treatment switching
HRO-reported treatment use and treatment switching throughout the study duration, triangulated with symptoms reported throughout the study duration
Measured: Up to 6 months
Health-related quality of life (HRQoL)
HRQoL HRO tracking data:
- Mood \& behavioral health
- Productivity
- Reasons for discontinuing or changing a treatment
Measured: up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2024
- Study start11 Jun 2024
- Primary completion15 Jun 2026 (estimated)
- Study completion15 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 128 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06411626
- Sponsor's protocol ID
- CLNP023C1US01
- Status last verified
- Aug 2025
- First submitted
- 8 May 2024
- Last updated
- 27 Aug 2025
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US
- ClinicalTrials.gov ID
- NCT06411626
- Acronym / sponsor ID
- CLNP023C1US01
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Paroxysmal nocturnal hemoglobinuria, PNH, home reported outcomes, HRO, patient-reported outcome, PRO, real-world data, iptacopan
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 128 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 11 Jun 2024 (actual)
- Primary completion
- 15 Jun 2026 (estimated)
- Study completion
- 15 Jun 2026 (estimated)
- First posted
- 13 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.
Detailed description
The study will be prospective and observational, conducted over an initial period of six months per individual from the point of study enrollment. Participants will utilize the Folia mobile app to enroll, consent, and complete all study activities. A hybrid recruitment method of clinic referrals and community referrals will be employed to identify participants, who will be asked to track routine treatment, symptoms, changes in treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins will be conducted to capture additional data inputs, with the possibility of integrating electronic health record (EHR) and/or claims data.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with PNH | – | Other: PNH-relevant therapiesParticipants with PNH |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Individuals aged 18+ who have been diagnosed with PNH, regardless of symptom or treatment history, and are US-based with a proficient understanding of and ability to read the English language.
Key inclusion criteria (4 listed)
- Study participants eligible for inclusion in this study must meet all of the following criteria:
- Aged 18 or older
- US-based with a proficient understanding of and ability to read the English language
- Any patient with a diagnosis of PNH, regardless of symptom or treatment history
Key exclusion criteria (1 listed)
- Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other exclusion criteria in order to meet the exploratory nature of the primary endpoint.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PNH-relevant therapiesThis is an observational study. There is no treatment allocation. The decision to initiate PNH-relevant therapies (such as eculizumab, ravulizumab, pegcetacoplan, iptacopan, and others) will be based solely on clinical judgement. | Other | Participants with PNH |
Primary outcome
PNH symptom burden
Time frame: Baseline, month 6
Primary endpoint will be measured through longitudinal Home Reported Outcomes (HRO) tracking of self-selected symptoms at enrollment and associated changes in severity against self-reported baselines.
3 secondary outcomes
Number of participants by treatment utilization and management of potential flare events
Time frame: Up to 6 months
Number of participants by reported treatment use and treatment switching
Time frame: Up to 6 months
Health-related quality of life (HRQoL)
Time frame: up to 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 1 site in United States.
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 1 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: looking for participants now, as of the sponsor's last update on 27 Aug 2025. 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 United States. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PNH symptom burden (time frame: Baseline, month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Aug 2025.