Trial · NCT02268552
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
NCT02268552, Phase 1, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2025-04-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA). Trials, record NCT02268552. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02268552
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
- NCT number
- NCT02268552
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 40 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Spinal Muscular Atrophy
- Interventions
- branaplam
- First posted
- 2014-10-20
- Last updated
- 2025-04-09
- Results first posted
- 2025-04-09
- Limitations and caveats
In alignment with the study objectives, safety and tolerability of branaplam irrespective of the dose, and protocol defined analyses, secondary efficacy endpoints were analyzed according to the treatment group that participants were assigned at baseline (Part 1\&3 and Part 2\&3) irrespective of study dose or route of administration. The fact that no dose limiting toxicity was observed in any of the doses and the lack of evidence for dose dependent safety findings further supports this strategy.
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Baseline up to 2 weeks for Part 1 | A DLT is defined as an adverse event or abnormal laboratory value assessed as unrelated to disease, disease progression, inter-current illness, or concomitant therapies that occurs within the first 14 days of treatment with LMI070 and meets any of the criteria for blood and lymphatic system disorders, gastrointestinal disorders, investigations and other toxicities considered clinically significant. |
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Baseline up to approximately 83 months | TEAEs are defined as adverse events starting on or after the first dose of study treatment that were absent pre-treatment, or events present prior to the first dose but increased in severity after the first dose. Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post last treatment. |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUCinf) After a Single Dose - Part 1 - Pharmacokinetics Analysis Set (PAS) | from 0 h to 168 h after first/single dose | The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity \[mass x time / volume\] |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUCinf) for All Observation Periods - Part 1 - PAS | from 0 h to 168 h after first/single dose | The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity \[mass x time / volume\] for all observations. AUC values used for comparison are combined from AUCinf values after single dose and AUC 0-168h values after repeated administration. |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax After a Single Dose - Part 1 - PAS | from 0 h to 168 h after first/single dose | The observed maximum plasma concentration following drug administration \[mass / volume). |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for All Observation Periods - Part 1 - PAS | from 0 h to 168 h after first/single dose | The observed maximum plasma (or serum or blood) concentration following drug administration \[mass / volume |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUC) After a Single Dose - Part 2 - PAS | from 0 h to 168 h after first/single dose | The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity \[mass x time / volume) |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUC) for All Observation Periods - Part 2 - PAS | from 0 h to 168 h after first/single dose | The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity \[mass x time / volume). AUC values used for comparison are combined from AUCinf values after single dose and AUC0-168h values after repeated administration. |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for a Single Dose - Part 2 - PAS | from 0 h to 168 h after first/single dose | The observed maximum plasma (or serum or blood) concentration following drug administration \[mass / volume) |
| Secondary | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for All Observation Periods - Part 2 - PAS | from 0 h to 168 h after first/single dose | The observed maximum plasma (or serum or blood) concentration following drug administration \[mass / volume) |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Baseline up to 2 weeks for Part 1 | Safety analysis set. No DLTs were observed, therefore maximum tolerated dose could not be determined. | Participants | NUMBER |
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Baseline up to approximately 83 months | Safety analysis set, all enrolled participants who received at least one dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUCinf) After a Single Dose - Part 1 - Pharmacokinetics Analysis Set (PAS) | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUCinf) for All Observation Periods - Part 1 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax After a Single Dose - Part 1 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for All Observation Periods - Part 1 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUC) After a Single Dose - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUC) for All Observation Periods - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for a Single Dose - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for All Observation Periods - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group122
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Part 1 LMI070 6mg/m2 | 0 | Participants | ||
| Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Part 1 LMI070 12mg/m2 | 0 | Participants | ||
| Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Part 1 LMI070 24mg/m2 | 0 | Participants | ||
| Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Part 1 LMI070 48mg/m2 | 0 | Participants | ||
| Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Part 1 LMI070 60mg/m2 | 0 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Part 1 LMI070 6mg/m2 | Participants with adverse events | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Part 1 LMI070 12mg/m2 | Participants with adverse events | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Part 1 LMI070 24mg/m2 | Participants with adverse events | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Part 1 LMI070 48mg/m2 | Participants with adverse events | 4 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Part 1 LMI070 60mg/m2 | Participants with adverse events | 3 | Participants |
1 to 10 of 122Source ClinicalTrials.gov
See all 122 results by groupParticipant flow63
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 | STARTED | Part 1 LMI070 6mg/m2 | 2 | |
| Part 1 | STARTED | Part 1 LMI070 12mg/m2 | 2 | |
| Part 1 | STARTED | Part 1 LMI070 24mg/m2 | 2 | |
| Part 1 | STARTED | Part 1 LMI070 48mg/m2 | 4 | |
| Part 1 | STARTED | Part 1 LMI070 60mg/m2 | 3 | |
| Part 1 | STARTED | Part 2 LMI070 0.625 mg/kg | 0 | |
| Part 1 | STARTED | Part 2 LMI070 2.5 mg/kg | 0 | |
| Part 1 | COMPLETED | Part 1 LMI070 6mg/m2 | 1 | |
| Part 1 | COMPLETED | Part 1 LMI070 12mg/m2 | 1 | |
| Part 1 | COMPLETED | Part 1 LMI070 24mg/m2 | 1 |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 participant flowAdverse events2,779
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Part 1 LMI070 6mg/m2 | 0 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Part 1 LMI070 12mg/m2 | 1 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Part 1 LMI070 24mg/m2 | 0 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Part 1 LMI070 48mg/m2 | 0 | 4 | |
| other | Anaemia | Blood and lymphatic system disorders | Part 1 LMI070 60mg/m2 | 1 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Part 2 LMI070 0.625 mg/kg | 0 | 10 | |
| other | Anaemia | Blood and lymphatic system disorders | Part 2 LMI070 2.5 mg/kg | 3 | 15 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Part 1 LMI070 6mg/m2 | 0 | 2 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Part 1 LMI070 12mg/m2 | 0 | 2 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Part 1 LMI070 24mg/m2 | 0 | 2 |
1 to 10 of 2,779Source ClinicalTrials.gov
See all 2,779 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 LMI070 6mg/m2 | 1 | 2 |
| Total, all-cause mortality | Part 1 LMI070 12mg/m2 | 2 | 2 |
| Total, all-cause mortality | Part 1 LMI070 24mg/m2 | 2 | 2 |
| Total, all-cause mortality | Part 1 LMI070 48mg/m2 | 3 | 4 |
| Total, all-cause mortality | Part 1 LMI070 60mg/m2 | 1 | 3 |
| Total, all-cause mortality | Part 2 LMI070 0.625 mg/kg | 1 | 10 |
| Total, all-cause mortality | Part 2 LMI070 2.5 mg/kg | 2 | 15 |
| Total, other adverse events | Part 1 LMI070 6mg/m2 | 2 | 2 |
| Total, other adverse events | Part 1 LMI070 12mg/m2 | 2 | 2 |
| Total, other adverse events | Part 1 LMI070 24mg/m2 | 2 | 2 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35241644 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02268552
GET https://conductscience.org/api/trials/v1/NCT02268552/registered?page=2